Title:

A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants with Prodromal and Early Manifest Huntington’s Disease

EUCT number:

2023-503928-10-00

Protocol code:

BN42489

Table of Contents

1 Summary

1.1 Trial Information

Medical condition(s):

Prodromal and Early Manifest Huntington’s Disease

Trial Phase:

Therapeutic exploratory (Phase II)

Transition Trial:

Yes

Sponsor:

F. Hoffmann-La Roche AG

Participants type:

Patients

Age range:

18-64 years

Locations:

Poland, Germany, Italy, Denmark, Portugal, Austria, Spain, France

Main objective (English):

To evaluate the safety of tominersen compared with placebo on the basis of incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale, change from baseline in clinical laboratory results [cerebrospinal fluid (CSF) white blood cell (WBC) and protein], and safety magnetic resonance imaging (MRI) (DB Period) To evaluate CSF mutant huntingtin (mHTT) protein levels in response to tominersen compared with placebo at 9 months (DB Period) To evaluate the efficacy of tominersen compared with placebo on the basis of change from baseline in composite unified huntington's disease rating scale (cUHDRS) (non-U.S.) and total functional capacity (TFC) (U.S.) at 16 months (DB Period) To evaluate the safety of long-term tominersen administration on the basis incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale, Change over time in clinical laboratory results (CSF, WBC and protein), and safety MRI (OLE Period)

1.2 Overall Trial status

Overall trial status:

Ongoing, recruitment ended

Start of Trial:

2023-02-01

End of trial:

Global end of trial:

Overall Trial Status:

Ongoing, recruitment ended

Application Trial Status:

Member State Application Trial Status Decision Date
Poland Ongoing, recruitment ended 2024-05-27
Germany Ongoing, recruitment ended 2024-05-22
Italy Ongoing, recruitment ended 2024-05-22
Denmark Ongoing, recruitment ended 2024-05-20
Portugal Ongoing, recruitment ended 2024-05-17
Austria Ongoing, recruitment ended 2024-05-22
Spain Ongoing, recruitment ended 2024-05-22
France Ongoing, recruitment ended 2024-05-24

1.3 Trial Notifications

1.3.1 Austria

Start of trial:

2023-09-05

Restart trial:

End of trial:

Early termination:

Reason for early termination:

1.3.2 Denmark

Start of trial:

2023-03-16

Restart trial:

End of trial:

Early termination:

Reason for early termination:

1.3.3 France

Start of trial:

2023-07-20

Restart trial:

End of trial:

Early termination:

Reason for early termination:

1.3.4 Germany

Start of trial:

2023-04-03

Restart trial:

End of trial:

Early termination:

Reason for early termination:

1.3.5 Italy

Start of trial:

2023-05-02

Restart trial:

End of trial:

Early termination:

Reason for early termination:

1.3.6 Spain

Start of trial:

2023-02-01

Restart trial:

End of trial:

Early termination:

Reason for early termination:

1.3.7 Poland

Start of trial:

2023-03-23

Restart trial:

End of trial:

Early termination:

Reason for early termination:

1.3.8 Portugal

Start of trial:

2023-05-29

Restart trial:

End of trial:

Early termination:

Reason for early termination:

1.4 Recruitment Notifications

1.4.1 Austria

Start of recruitment:

2023-10-24

Restart of recruitment:

End of recruitment:

2025-02-18

1.4.2 Denmark

Start of recruitment:

2023-05-02

Restart of recruitment:

End of recruitment:

2025-02-18

1.4.3 France

Start of recruitment:

2023-08-28

Restart of recruitment:

End of recruitment:

2025-02-18

1.4.4 Germany

Start of recruitment:

2023-04-25

Restart of recruitment:

End of recruitment:

2025-02-18

1.4.5 Italy

Start of recruitment:

2023-05-08

Restart of recruitment:

End of recruitment:

2025-02-18

1.4.6 Spain

Start of recruitment:

2023-02-03

Restart of recruitment:

End of recruitment:

2025-02-18

1.4.7 Poland

Start of recruitment:

2023-04-04

Restart of recruitment:

End of recruitment:

2025-02-18

1.4.8 Portugal

Start of recruitment:

2023-07-31

Restart of recruitment:

End of recruitment:

2025-02-18

1.5 Trial duration

Estimated recruitment start date in EU/EEA:

2023-03-03

Estimated end of trial date in EU/EEA:

2027-04-14

Estimated global end date of the trial:

2027-04-14

1.5.1 Source of monetary or financial support

Organisation name:

F. Hoffmann-La Roche Ltd

1.6 Serious Breaches

1.6.1 Serious Breach SB-81093

MSCs:

Austria, Denmark, France, Germany, Italy, Spain, Poland, Portugal

Sponsor internal identifier:

QE-158514

Business key:

SB-81093

Publication date:

2025-05-07

Last modified:

2025-05-07

Justification:

Date of becoming aware of the serious breach:

2025-04-30

Date of serious breach:

2025-04-30

Affected ocuntries:

United States

Breach category:

Protocol

Areas impacted:

Data reliability or robustness, Subject rights

Description of serious breach and impacts on trial:

Persistent level of GCP non-compliance and lack of PI Oversight with no clear commitment from the site to ameliorate the problem -A persistent pattern of GCP and protocol non-compliance. -Unresolved critical documentation deficiencies. -Lack of timely or effective corrective action implementation. -No demonstrated commitment from the site or PI to address deficiencies.

Actions taken and planned (including timelines) to investigate and correct the breach and to prevent the reoccurence of that or a similar breach:

Since issues first identified in 2024, sponsor medical monitor, MSL and operations team has engaged with the PI and ensure appropriate re-training Site was then placed on a screening hold in September 2024 due to inadequate source documentation practices. Following multiple retraining sessions and site visits, a formal CAPA was implemented in February 2025. However, the site failed to meet the CAPA action deadline of March 12th, and several major protocol deviations related to the use of wrong needle remain unreported to the IRB as of today (which raises concerns on subject safety). A final site visit by Roche is scheduled for May 1st to review the CAPA and discuss the possibility of site closure.

Has the occurrence of the serious breach impacted subjects safety and/or benefit-risk balance?:

No

Sites

Site Name Site Address Site Postcode Site City Country Type of Organisation Other Type of Organisation
Inland Northwest Research Address 99202 Washington United States Clinical investigator

1.6.2 Serious Breach SB-71765

MSCs:

Austria, Denmark, France, Germany, Italy, Spain, Poland, Portugal

Sponsor internal identifier:

QE-150814

Business key:

SB-71765

Publication date:

2025-02-26

Last modified:

2025-02-26

Justification:

Date of becoming aware of the serious breach:

2025-02-20

Date of serious breach:

2025-02-20

Affected ocuntries:

Australia

Breach category:

Protocol

Areas impacted:

Subject rights, Subject safety

Description of serious breach and impacts on trial:

During an onsite monitoring visit, the CRA identified that Subject 20148’s neurologic examination was not performed at subject's Month 9 visit on 10th Sep 2024. Neurologic examinations are required per the protocol at Month 9 visit prior to administering the study drug via intrathecal injection. This is to identify any neurological findings, in particular raised intracranial pressure, which would pose a risk to the patient if the intrathecal injection was to go ahead. Therefore, the missing neurologic examination at month 9 has potential to significantly impact the safety of the patient. The study coordinator advised that the neuro exam was missed as proformas for source collection had not been updated from protocol v2 requirements. The patient has not had any AEs reported during their time on the study.

Actions taken and planned (including timelines) to investigate and correct the breach and to prevent the reoccurence of that or a similar breach:

Immediate actions taken: - A major protocol deviation was reported on 24Jan2025 - Site has been advised to update proforma/source documentation to align with current protocol requirements. - Report SNC to the Australian HREC and site Research Governance Office (RGO).

Has the occurrence of the serious breach impacted subjects safety and/or benefit-risk balance?:

No

Sites

Site Name Site Address Site Postcode Site City Country Type of Organisation Other Type of Organisation
Royal Melbourne Hospital Address 3052 Parkville Australia Clinical investigator

1.7 Unexpected Events

1.8 Urgent Safety Measures

1.8.1 Urgent Safety Measure US-79119

MSCs:

Austria, Denmark, France, Germany, Italy, Spain, Poland, Portugal

Sponsor internal identifier:

CLIN-2025-00391

Business key:

US-79119

Publication date:

2025-04-14

Last modified:

2025-04-14

Justification:

Date of implementation of the urgent safety measure:

2025-04-14

Is the urgent safety measure in response to a reported unexpected event or SUSAR?:

Other

World Wide Unique Identifier:

Unexpected Event Unique Identifier:

Description of the event that triggered the urgent safety measure:

The iDMC recently reviewed safety data and conducted an interim analysis. They recommended that Study BN42489 should continue. No concerns regarding safety or signs of clinical worsening in any arm were raised. The 100mg Q16W dose was found to be more likely to result in clinical benefit than the 60mg dose. The study will be modified to test only the 100mg dose versus placebo for the remainder of the study. The 60mg dose will be discontinued. Participants assigned the 60mg dose will be switched to the 100mg dose.

Description of the urgent safety measures taken:

Dose modification in BN42489: 100mg Q16W & placebo continue, 60mg dose discontinued and participants switched to 100mg

1.9 Temporary Halts

1.10 Corrective Measures

1.11 Applications

1.11.1 IN

Application type:

INITIAL

Submission date:

2024-04-08

1.11.1.1 Assessment Part I

Reference Member State:

Denmark

Final conclusion:

Acceptable

Conclusion reporting date:

2024-05-17

1.11.1.2 Assessment Part II

Member state Final conclusion Conclusion reporting date
Austria Acceptable 2024-05-06
Denmark Acceptable 2024-05-02
France Acceptable 2024-05-23
Germany Acceptable 2024-05-02
Italy Acceptable 2024-05-13
Spain Acceptable 2024-05-06
Poland Acceptable 2024-05-24
Portugal Acceptable 2024-05-03

1.11.1.3 Decision

Member state Decision Decision date Decision type
Poland Authorised
2024-05-27
Decision
Germany Authorised
2024-05-22
Decision
Portugal Authorised
2024-05-17
Decision
Denmark Authorised
2024-05-20
Decision
France Authorised
2024-05-24
Decision
Austria Authorised
2024-05-22
Decision
Italy Authorised
2024-05-22
Decision
Spain Authorised
2024-05-22
Decision

1.11.2 SM-1

Application type:

SUBSTANTIAL MODIFICATION

Submission date:

2024-07-25

1.11.2.1 Assessment Part I

Reference Member State:

Final conclusion:

Acceptable

Conclusion reporting date:

1.11.2.2 Assessment Part II

Member state Final conclusion Conclusion reporting date
Italy Acceptable 2024-09-02

1.11.2.3 Decision

Member state Decision Decision date Decision type
Italy Authorised
2024-09-09
Decision

1.11.3 SM-2

Application type:

SUBSTANTIAL MODIFICATION

Submission date:

2024-08-08

1.11.3.1 Assessment Part I

Reference Member State:

Final conclusion:

Acceptable

Conclusion reporting date:

1.11.3.2 Assessment Part II

Member state Final conclusion Conclusion reporting date
France Acceptable 2024-09-11

1.11.3.3 Decision

Member state Decision Decision date Decision type
France Authorised
2024-09-13
Decision

1.11.4 SM-3

Application type:

SUBSTANTIAL MODIFICATION

Submission date:

2024-07-25

1.11.4.1 Assessment Part I

Reference Member State:

Final conclusion:

Acceptable

Conclusion reporting date:

1.11.4.2 Assessment Part II

Member state Final conclusion Conclusion reporting date
Poland Acceptable 2024-09-02

1.11.4.3 Decision

Member state Decision Decision date Decision type
Poland Authorised
2024-09-04
Decision

1.11.5 SM-4

Application type:

SUBSTANTIAL MODIFICATION

Submission date:

2024-07-25

1.11.5.1 Assessment Part I

Reference Member State:

Final conclusion:

Acceptable

Conclusion reporting date:

1.11.5.2 Assessment Part II

Member state Final conclusion Conclusion reporting date
Portugal Acceptable 2024-08-29

1.11.5.3 Decision

Member state Decision Decision date Decision type
Portugal Authorised
2024-09-02
Decision

1.11.6 NSM-1

Application type:

NON SUBSTANTIAL MODIFICATION

Submission date:

2024-10-04

1.11.6.1 Assessment Part I

Reference Member State:

Denmark

Final conclusion:

Acceptable

Conclusion reporting date:

1.11.6.2 Assessment Part II

Member state Final conclusion Conclusion reporting date

1.11.6.3 Decision

Member state Decision Decision date Decision type
Austria Authorised
2024-10-04
Decision
Denmark Authorised
2024-10-04
Decision
Germany Authorised
2024-10-04
Decision
Spain Authorised
2024-10-04
Decision
Italy Authorised
2024-10-04
Decision
France Authorised
2024-10-04
Decision
Poland Authorised
2024-10-04
Decision
Portugal Authorised
2024-10-04
Decision

1.11.7 NSM-2

Application type:

NON SUBSTANTIAL MODIFICATION

Submission date:

2024-10-11

1.11.7.1 Assessment Part I

Reference Member State:

Denmark

Final conclusion:

Acceptable

Conclusion reporting date:

1.11.7.2 Assessment Part II

Member state Final conclusion Conclusion reporting date

1.11.7.3 Decision

Member state Decision Decision date Decision type
Austria Authorised
2024-10-11
Decision
Denmark Authorised
2024-10-11
Decision
Germany Authorised
2024-10-11
Decision
Spain Authorised
2024-10-11
Decision
Italy Authorised
2024-10-11
Decision
France Authorised
2024-10-11
Decision
Poland Authorised
2024-10-11
Decision
Portugal Authorised
2024-10-11
Decision

1.11.8 NSM-3

Application type:

NON SUBSTANTIAL MODIFICATION

Submission date:

2024-10-28

1.11.8.1 Assessment Part I

Reference Member State:

Denmark

Final conclusion:

Acceptable

Conclusion reporting date:

1.11.8.2 Assessment Part II

Member state Final conclusion Conclusion reporting date

1.11.8.3 Decision

Member state Decision Decision date Decision type
Austria Authorised
2024-10-28
Decision
Denmark Authorised
2024-10-28
Decision
Germany Authorised
2024-10-28
Decision
Spain Authorised
2024-10-28
Decision
Italy Authorised
2024-10-28
Decision
France Authorised
2024-10-28
Decision
Poland Authorised
2024-10-28
Decision
Portugal Authorised
2024-10-28
Decision

1.11.9 SM-7

Application type:

SUBSTANTIAL MODIFICATION

Submission date:

2025-01-30

1.11.9.1 Assessment Part I

Reference Member State:

Denmark

Final conclusion:

Acceptable

Conclusion reporting date:

2025-03-26

1.11.9.2 Assessment Part II

Member state Final conclusion Conclusion reporting date

1.11.9.3 Decision

Member state Decision Decision date Decision type
Denmark Authorised
2025-03-26
Decision
Italy Authorised
2025-03-28
Decision
Portugal Authorised
2025-03-27
Decision
Austria Authorised
2025-03-31
Decision
France Authorised
2025-03-27
Decision
Germany Authorised
2025-03-27
Decision
Poland Authorised
2025-03-31
Decision
Spain Authorised
2025-03-26
Decision

1.11.10 SM-8

Application type:

SUBSTANTIAL MODIFICATION

Submission date:

2025-04-28

1.11.10.1 Assessment Part I

Reference Member State:

Denmark

Final conclusion:

Acceptable

Conclusion reporting date:

2025-07-18

1.11.10.2 Assessment Part II

Member state Final conclusion Conclusion reporting date
Austria Acceptable 2025-06-30
Germany Acceptable 2025-07-03
Portugal Acceptable 2025-06-17
Denmark Acceptable 2025-06-23
Poland Acceptable 2025-06-30
Spain Acceptable 2025-06-25
France Acceptable 2025-06-11
Italy Acceptable 2025-08-04

1.11.10.3 Decision

Member state Decision Decision date Decision type
France Authorised
2025-07-18
Decision
Italy Authorised
2025-08-06
Decision
Austria Authorised
2025-07-22
Decision
Poland Authorised
2025-07-23
Decision
Spain Authorised
2025-07-22
Decision
Portugal Authorised
2025-07-18
Decision
Denmark Authorised
2025-07-21
Decision
Germany Authorised
2025-07-22
Decision

1.11.11 SM-9

Application type:

SUBSTANTIAL MODIFICATION

Submission date:

2025-08-14

1.11.11.1 Assessment Part I

Reference Member State:

Denmark

Final conclusion:

Acceptable

Conclusion reporting date:

2025-10-10

1.11.11.2 Assessment Part II

Member state Final conclusion Conclusion reporting date
Portugal Acceptable 2025-10-07

1.11.11.3 Decision

Member state Decision Decision date Decision type
Denmark Authorised
2025-10-13
Decision
Italy Authorised
2025-10-14
Decision
Austria Authorised
2025-10-13
Decision
Spain Authorised
2025-10-15
Decision
Germany Authorised
2025-10-13
Decision
Poland Authorised
2025-10-15
Decision
France Authorised
2025-10-15
Decision
Portugal Authorised
2025-10-14
Decision

1.11.12 SM-10

Application type:

SUBSTANTIAL MODIFICATION

Submission date:

2026-02-06

1.11.12.1 Assessment Part I

Reference Member State:

Denmark

Final conclusion:

Acceptable

Conclusion reporting date:

2026-03-27

1.11.12.2 Assessment Part II

Member state Final conclusion Conclusion reporting date
France Acceptable 2026-03-20
Poland Acceptable 2026-03-27
Germany Acceptable 2026-02-20
Italy Acceptable 2026-03-30
Denmark Acceptable 2026-03-05
Spain Acceptable 2026-04-14
Portugal Acceptable 2026-03-10
Austria Acceptable 2026-04-03

1.11.12.3 Decision

Member state Decision Decision date Decision type
Spain Authorised
2026-04-20
Decision
Austria Authorised
2026-04-07
Decision
Italy Authorised
2026-04-01
Decision
Denmark Authorised
2026-03-27
Decision
Poland Authorised
2026-03-31
Decision
Germany Authorised
2026-04-01
Decision
France Authorised
2026-03-27
Decision
Portugal Authorised
2026-03-30
Decision

1.11.13 NSM-4

Application type:

NON SUBSTANTIAL MODIFICATION

Submission date:

2026-04-30

1.11.13.1 Assessment Part I

Reference Member State:

Final conclusion:

Acceptable

Conclusion reporting date:

2026-03-27

1.11.13.2 Assessment Part II

Member state Final conclusion Conclusion reporting date
Spain

1.11.13.3 Decision

Member state Decision Decision date Decision type
Spain Authorised
2026-04-30
Decision

1.11.14 NSM-5

Application type:

NON SUBSTANTIAL MODIFICATION

Submission date:

2026-07-13

1.11.14.1 Assessment Part I

Reference Member State:

Denmark

Final conclusion:

Acceptable

Conclusion reporting date:

2026-03-27

1.11.14.2 Assessment Part II

Member state Final conclusion Conclusion reporting date

1.11.14.3 Decision

Member state Decision Decision date Decision type
France Authorised
2026-07-13
Decision
Poland Authorised
2026-07-13
Decision
Germany Authorised
2026-07-13
Decision
Italy Authorised
2026-07-13
Decision
Denmark Authorised
2026-07-13
Decision
Portugal Authorised
2026-07-13
Decision
Austria Authorised
2026-07-13
Decision
Spain Authorised
2026-07-13
Decision

1.11.15 SM-11

Application type:

SUBSTANTIAL MODIFICATION

Submission date:

2026-07-22

1.11.15.1 Assessment Part I

Reference Member State:

Final conclusion:

Acceptable

Conclusion reporting date:

1.11.15.2 Assessment Part II

Member state Final conclusion Conclusion reporting date
France Acceptable 2026-08-30

1.11.15.3 Decision

Member state Decision Decision date Decision type
France Authorised
2026-09-01
Decision

1.11.16 NSM-6

Application type:

NON SUBSTANTIAL MODIFICATION

Submission date:

2026-09-11

1.11.16.1 Assessment Part I

Reference Member State:

Denmark

Final conclusion:

Acceptable

Conclusion reporting date:

2026-03-27

1.11.16.2 Assessment Part II

Member state Final conclusion Conclusion reporting date

1.11.16.3 Decision

Member state Decision Decision date Decision type
Poland Authorised
2026-09-11
Decision
Germany Authorised
2026-09-11
Decision
Italy Authorised
2026-09-11
Decision
Denmark Authorised
2026-09-11
Decision
Portugal Authorised
2026-09-11
Decision
Austria Authorised
2026-09-11
Decision
Spain Authorised
2026-09-11
Decision
France Authorised
2026-09-11
Decision

2 Full trial information (Part I)

2.1 Trial details

2.1.1 Trial identifiers

2.1.1.1 Clinical trial identifiers

EU trial number:

2023-503928-10-00

Full title (English):

A Phase II, Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen in Individuals with Prodromal and Early Manifest Huntington’s Disease

Public title (English):

A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants with Prodromal and Early Manifest Huntington’s Disease

Protocol code:

BN42489

2.1.1.2 Secondary identifying numbers

WHO universal trial number (UTN):

ClinicalTrials.gov identifier (NCT number):

ISRCTN number:

2.1.1.3 Additional registries

2.1.2 Trial Information

2.1.2.1 Transition Trial

EudraCT number:

2022-001991-32

2.1.2.2 Trial Category

Trial phase:

Therapeutic exploratory (Phase II)

Trial category:

2

Justification for trial category:

Deferral is selected because this is a confirmatory/registrational trial and the development program for the molecule is ongoing

2.1.2.3 Medical Conditions

Medical condition(s) (English):

Prodromal and Early Manifest Huntington’s Disease

Is the medical condition considered to be a rare disease:

No

Therapeutic area:

Diseases [C] - Nervous System Diseases [C10], Not possible to specify

2.1.2.4 Medical condition(s) MedDRA information

Version Level Classification code Term name System organ class
20.0 PT 10070668 Huntington's disease 100000004850

2.1.2.5 Main objective

Trial scope:

Safety, Other, Efficacy

Main objective (English):

To evaluate the safety of tominersen compared with placebo on the basis of incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale, change from baseline in clinical laboratory results [cerebrospinal fluid (CSF) white blood cell (WBC) and protein], and safety magnetic resonance imaging (MRI) (DB Period) To evaluate CSF mutant huntingtin (mHTT) protein levels in response to tominersen compared with placebo at 9 months (DB Period) To evaluate the efficacy of tominersen compared with placebo on the basis of change from baseline in composite unified huntington's disease rating scale (cUHDRS) (non-U.S.) and total functional capacity (TFC) (U.S.) at 16 months (DB Period) To evaluate the safety of long-term tominersen administration on the basis incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale, Change over time in clinical laboratory results (CSF, WBC and protein), and safety MRI (OLE Period)

2.1.2.6 Secondary objective

Secondary objective number Secondary objective (English)
1 To evaluate the safety of tominersen compared with placebo on the basis of change from baseline in vital signs, electrocardiogram (ECG) parameters, plasma clinical laboratory results, montreal cognitive assessment (MoCA), and proportion of participants with suicidal ideation or behavior as assessed by columbia-suicide severity rating scale (C-SSRS) score at each visit (DB Period) To evaluate the safety of long-term tominersen on the basis of change over time in vital signs, electrocardiogram (ECG) parameters, plasma clinical laboratory results, montreal cognitive assessment (MoCA), and proportion of participants with suicidal ideation or behavior as assessed by columbia-suicide severity rating scale (C-SSRS) score at each visit (OLE Period)
2 To evaluate the efficacy of tominersen compared with placebo on the basis of change from baseline at 16 months for the assessments of TFC (non-U.S.)/cUHDRS (U.S.), symbol digit modalities test (SDMT) stroop word reading (SWR) and total motor score (TMS) (DB Period) To evaluate the efficacy of long-term tominersen on the basis of change over time for the assessments of cUHDRS, TFC, symbol digit modalities test (SDMT) stroop word reading (SWR) and total motor score (TMS) (OLE Period)
3 To evaluate change from baseline in CSF neurofilament light chain (NfL) in response to tominersen compared with placebo at 16 months (DB Period)
4 To evaluate the immune response to tominersen (DB Period) To evaluate the immune response to long-term tominersen administration (OLE Period)

2.1.2.7 Principal inclusion criteria

Inclusion criteria number Principal inclusion criteria (English)
1 Huntington’s disease (HD) gene expansion mutation carrier status with a CAP score of 400-500 inclusive
2 Either: Prodromal HD (defined as DCL 2 to 3, Independence Scale (IS) ⩾70, and ⩾TFC8); or Early manifest HD (defined as DCL 4, Independence Scale (IS) ⩾70, and ⩾TFC8)
3 Estimated glomerular filtration rate ≥ 60 mL/min/1.73 m2 in at least one out of two maximum screening samples
4 Total body weight > 40 kg and a body mass index within the range of 18-32 kg/m2
5 Age 25- 50 years, inclusive, at the time of signing the Informed Consent Form
6 Study Companion

2.1.2.8 Principal exclusion criteria

Exclusion criteria number Principal exclusion criteria (English)
1 History of attempted suicide or suicidal ideation with plan that required hospital visit and/or change in level of care within 12 months prior to screening
2 Current or previous use of an ASO (including small interfering RNA) or any HTT lowering therapy (including tominersen)
3 Anti-platelet or anticoagulant therapy
4 Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 5 months after the final dose of study drug
5 History of gene therapy, cell transplantation, or brain surgery
6 Hydrocephalus

2.1.2.9 Primary end points

End point criteria number Primary end point (English)
1 Incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale (DB Period)
2 Change from baseline in clinical laboratory results (CSF WBC and protein) (DB Period)
3 Safety MRI (DB Period)
4 Percentage change from baseline in geometric means of CSF mHTT protein levels at Month 9 (DB Period)
5 Change from baseline in cUHDRS (non-U.S.) and TFC (U.S.) at 16 months (DB Period)
6 Incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale (OLE Period)
7 Change over time in clinical laboratory results (CSF, WBC, and protein) (OLE period)
8 Safety MRI (OLE period)

2.1.2.10 Secondary end points

Secondary end point number Secondary end point (English)
1 Change from baseline in vital signs (DB period)
2 Change from baseline in ECG parameters (DB period)
3 Change from baseline in plasma clinical laboratory results (DB period)
4 Change from baseline in MoCA (DB period)
5 Proportion of participants with suicidal ideation or behavior as assessed by C-SSRS score at each visit, including detailed focus on any individual cases identified as having severe ideation or behavior during the study conduct (DB period)
6 Change from baseline at 16 months for the assessments of TFC (non-U.S.)/cUHDRS (U.S.), SDMT, TMS, and SWR (DB period)
7 Change from baseline in CSF NfL levels at 16 months (DB period)
8 Incidence of anti-drug antibodies (ADAs) at specified timepoints relative to the prevalence of ADAs at baseline (DB period)
9 Titers will be determined if ADAs are identified (DB period)
10 Change over time in cUHDRS, TFC, SDMT, TMS, SWR (OLE period)
11 Change over time in vital signs (OLE period)
12 Change over time in ECG parameters (OLE period)
13 Change over time in plasma clinical laboratory results (OLE period)
14 Change over time in MoCA (OLE period)
15 Proportion of participants with suicidal ideation or behavior as assessed by C-SSRS score at each visit, including detailed focus on any individual cases identified as having severe ideation or behavior during the study conduct (OLE period)
16 Incidence of ADAs at specified timepoints (OLE period)

2.1.2.11 Individual Participant Data (IPD) Sharing statement

Plan to share IPD:

Undecided

Plan description:

NA

2.1.2.12 Participants

Gender:

Male and Female

Age range:

18-64 years

Age range secondary identifier:

Clinical trial group:

Patients

Vulnerable population:

Yes

2.1.3 Protocol information

2.1.3.1 Study design

Period details:

Number Period title Period description Allocation method Blinding used Roles blinded Blinding implementation details Arm details
1 Evaluate the safety, biomarkers, and efficacy of two doses of tominersen compared with placebo. This study is a Phase II, randomized, double-blind, placebo-controlled, dose-finding study evaluating the safety, biomarkers, and efficacy of two doses of tominersen compared with placebo in patients with prodromal and early manifest HD. The study consists of a screening period of up to 4 weeks, a minimum double-blind treatment period of 16 months with a common close design, a safety follow-up period of 5 months, and an optional open-label extension (OLE) period. Upon completion of the screening period, eligible participants will be randomly allocated in a 1:1:1 ratio to receive 60 mg tominersen, 100 mg tominersen, or placebo administered intrathecally Q16W for 16 months. Randomised Controlled Double Investigator, Subject

2.1.4 Scientific advice and paediatric investigation plan (PIP)

Competent authorities that have provided scientific advice:

European Medicines Agency

EMA paediatric investigation number:

EMEA-002546-PIP01-19

2.1.5 Associated clinical trials

Associated EU CTA number Full title Sponsor for associated clinical trial

2.1.7 References

Reference to publication:

Reference link to publication:

2.2 Products

2.2.1 Role: Test Name: RO7234292

2.2.1.1 Product: RO7234292

Type

Product

Excluded MSCs

2.2.1.1.1 Product details

Medicinal product name:

RO7234292

EU medicinal product number/medicinal product unique ID:

PRD10384563

Pharmaceutical form:

SOLUTION FOR INJECTION

Strength:

Medicinal product other name:

Tominersen

Is this a specific paediatric formulation:

No

Product authorisation status:

Not Authorised

Medicinal product role in trial:

Test

Sponsors product code:

RO 723-4292/F02-01

2.2.1.1.2 Products characteristics

Medicinal product characteristics:

Other

Other medicinal product:

antisense oligonucleotide

2.2.1.1.3 Dosage and administration Details

Route of administration:

I.T. BOLUS INJECTION TO THE INTRATHECAL SPACE

Maximum duration of treatment:

1 Day(s)

Maximum daily dose allowed:

0

Daily dose unit of measure:

DF dosage form

Maximum total dose allowed:

0

Total dose unit of measure:

DF dosage form

2.2.1.1.4 Information about the modification of the medicinal product

Has the medicinal product been modified in relation to its Marketing Authorisation:

No

Description of the modification:

2.2.1.1.5 Product classification

Anatomical Therapeutic Chemical (ATC) Codes:

ATC name:

ATC level:

2.2.1.1.6 Product authorisation details

MA holder

F. HOFFMANN-LA ROCHE LTD

MA authorisation country

Marketing authorisation number

Centralised procedure/MRP/DCP/registration procedure number

2.2.1.1.7 Orphan designation

Does this product have an orphan drug designation:

No

Designation number for orphan drug:

2.2.1.1.8 Active substance

Classification:

Nucleic Acid

Active Substance name:

TOMINERSEN

Active substance name synonyms:

Active Substance other descriptive name:

EU Active Substance Code:

SUB198185

Strength:

Status:

Not Authorised

2.2.1.1.9 Advanced therapy medicinal product
2.2.1.1.10 Device associated with medicinal product
Product used in combination with a device Product ID Device trade name Description of the device Type of device Device has CE mark Device notified body

2.2.1.1 Compliance with (GMP) for the medicinal product

Authorisation number of manufacturing and import:

2.2.2 Role: Placebo Name: RO7234292 Placebo

2.2.2.1 Product: RO7234292 Placebo

Type

Product

Excluded MSCs

2.2.2.1.1 Product details

Medicinal product name:

RO7234292 Placebo

EU medicinal product number/medicinal product unique ID:

N/A

Pharmaceutical form:

N/A

Strength:

Medicinal product other name:

N/A

Is this a specific paediatric formulation:

No

Product authorisation status:

Medicinal product role in trial:

Placebo

Sponsors product code:

2.2.2.1.2 Products characteristics

Medicinal product characteristics:

Other medicinal product:

2.2.2.1.3 Dosage and administration Details

Route of administration:

Maximum duration of treatment:

undefined Week(s)

Maximum daily dose allowed:

Daily dose unit of measure:

Maximum total dose allowed:

Total dose unit of measure:

2.2.2.1.4 Information about the modification of the medicinal product

Has the medicinal product been modified in relation to its Marketing Authorisation:

No

Description of the modification:

2.2.2.1.5 Product classification

Anatomical Therapeutic Chemical (ATC) Codes:

N/A

ATC name:

N/A

ATC level:

N/A

2.2.2.1.6 Product authorisation details

MA holder

N/A

MA authorisation country

Iceland

Marketing authorisation number

N/A

Centralised procedure/MRP/DCP/registration procedure number

2.2.2.1.7 Orphan designation

Does this product have an orphan drug designation:

No

Designation number for orphan drug:

2.2.2.1.8 Active substance

Classification:

Active Substance name:

Active substance name synonyms:

Active Substance other descriptive name:

N/A

EU Active Substance Code:

N/A

Strength:

Status:

2.2.2.1.9 Advanced therapy medicinal product
2.2.2.1.10 Device associated with medicinal product
Product used in combination with a device Product ID Device trade name Description of the device Type of device Device has CE mark Device notified body

2.2.2.1 Compliance with (GMP) for the medicinal product

Authorisation number of manufacturing and import:

3 Trial Results

3.1 Summaries of Results

3.2 Layperson Summaries of Results

3.3 Clinical Study Reports

4 Locations and contact points

4.1 Locations

4.1.1 Poland - Ongoing, recruitment ended

Planned number of subjects:

20

4.1.1.1 Site: Wojskowy Instytut Medycyny Lotniczej

OMS ID:

ORG-100052267

Department name:

Klinika Neurologii

Site location:

Ul. Zygmunta Krasinskiego 54/56

Site street address:

Ul. Zygmunta Krasinskiego 54/56

Site city:

Warsaw

Site post code:

01-755

Site country:

Poland

First name:

Grzegorz

Last name:

Witkowski

Title:

Dr.

Telephone number:

+48261852928

Email:

gwitkowski@wiml.waw.pl

4.1.1.2 Site: Krakowska Akademia Neurologii Sp. z o.o.

OMS ID:

ORG-100046735

Department name:

Centrum Neurologii Klinicznej

Site location:

Ul. Arianska 7/3

Site street address:

Ul. Arianska 7/3

Site city:

Cracow

Site post code:

31-505

Site country:

Poland

First name:

Monika

Last name:

Rudzińska-Bar

Title:

Prof.

Telephone number:

+48601070994

Email:

mrudzinska@afm.edu.pl

4.1.1.3 Site: Copernicus Podmiot Leczniczy Sp. z o.o.

OMS ID:

ORG-100043332

Department name:

Szpital Św. Wojciecha, Oddział Neurologiczny

Site location:

Al. Jana Pawla II 50

Site street address:

Al. Jana Pawla II 50

Site city:

Gdansk

Site post code:

80-462

Site country:

Poland

First name:

Jarosław

Last name:

Sławek

Title:

Prof.

Telephone number:

+48587684661

Email:

jaroslawek@gumed.edu.pl

4.1.2 Germany - Ongoing, recruitment ended

Planned number of subjects:

40

4.1.2.1 Site: Universitaetsklinikum Ulm AöR

OMS ID:

ORG-100006370

Department name:

Department of Neurology

Site location:

Oberer Eselsberg 45, Eselsberg

Site street address:

Oberer Eselsberg 45

Site city:

Ulm

Site post code:

89081

Site country:

Germany

First name:

Bernhard

Last name:

Landwehrmeyer

Title:

Prof.

Telephone number:

+4973150063080

Email:

bernhard.landwehrmeyer@uni-ulm.de

4.1.2.2 Site: Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.

OMS ID:

ORG-100043714

Department name:

Klinik für Neurodegenerative Erkrankungen

Site location:

Venusberg-Campus 1/99, Venusberg

Site street address:

Venusberg-Campus 1/99

Site city:

Bonn

Site post code:

53127

Site country:

Germany

First name:

Patrick

Last name:

Weydt

Title:

Dr.

Telephone number:

+4922843302849

Email:

Patrick.Weydt@ukbonn.de

4.1.2.3 Site: Katholisches Klinikum Bochum gGmbH

OMS ID:

ORG-100033643

Department name:

Neurologische Klinik der Ruhr-Universitaet Bochum

Site location:

Gudrunstrasse 56, Grumme

Site street address:

Gudrunstrasse 56

Site city:

Bochum

Site post code:

44791

Site country:

Germany

First name:

Carsten

Last name:

Saft

Title:

Prof.

Telephone number:

+492345092703

Email:

Carsten.Saft@ruhr-uni-bochum.de

4.1.2.4 Site: Universitaetsklinikum Erlangen AöR

OMS ID:

ORG-100006207

Department name:

Molekulare Neurologie

Site location:

Schwabachanlage 6, Innenstadt

Site street address:

Schwabachanlage 6

Site city:

Erlangen

Site post code:

91054

Site country:

Germany

First name:

Martin

Last name:

Regensburger

Title:

Dr.

Telephone number:

+4991318539324

Email:

martin.regensburger@uk-erlangen.de

4.1.2.5 Site: Kbo Isar-Amper-Klinikum Taufkirchen (Vils)

OMS ID:

ORL-000005986

Department name:

Huntington-Zentrum-Sued

Site location:

Braeuhausstrasse 5

Site street address:

Braeuhausstrasse 5

Site city:

Taufkirchen/Vils

Site post code:

84416

Site country:

Germany

First name:

Alzbeta

Last name:

Muehlbaeck

Title:

Dr.

Telephone number:

+498084934212

Email:

alzbeta.muehlbaeck@kbo.de

4.1.2.6 Site: Universitaetsklinikum Schleswig-Holstein AöR

OMS ID:

ORG-100023619

Department name:

Zentrum für Seltene Erkrankungen

Site location:

Ratzeburger Allee 160

Site street address:

Ratzeburger Allee 160

Site city:

Luebeck

Site post code:

23538

Site country:

Germany

First name:

Alexander

Last name:

Münchau

Title:

Prof.

Telephone number:

+4945131018215

Email:

alexander.muenchau@uni-luebeck.de

4.1.2.7 Site: Charite Universitaetsmedizin Berlin KöR

OMS ID:

ORG-100008480

Department name:

Klinik für Psychiatrie und Psychotherapie, CCM

Site location:

Chariteplatz 1, Mitte

Site street address:

Chariteplatz 1

Site city:

Berlin

Site post code:

10117

Site country:

Germany

First name:

Josef

Last name:

Priller

Title:

Prof.

Telephone number:

+4930450617209

Email:

Josef.Priller@charite.de

4.1.2.8 Site: Universitaetsklinikum Aachen AöR

OMS ID:

ORG-100023618

Department name:

Klinik für Neurologie

Site location:

Pauwelsstrasse 30

Site street address:

Pauwelsstrasse 30

Site city:

Aachen

Site post code:

52074

Site country:

Germany

First name:

Kathrin

Last name:

Reetz

Title:

Prof.

Telephone number:

+492418085522

Email:

kreetz@ukaachen.de

4.1.3 Italy - Ongoing, recruitment ended

Planned number of subjects:

14

4.1.3.1 Site: IRCCS Foundation Istituto Neurologico Carlo Besta

OMS ID:

ORG-100006637

Department name:

UOC Genetica Medica - Neurogenetica

Site location:

Via Giovanni Celoria 11

Site street address:

Via Giovanni Celoria 11

Site city:

Milan

Site post code:

20133

Site country:

Italy

First name:

Caterina

Last name:

Mariotti

Title:

Dr.

Telephone number:

+390223942766

Email:

caterina.mariotti@istituto-besta.it

4.1.3.2 Site: Azienda Unita Sanitaria Locale Di Bologna

OMS ID:

ORG-100010199

Department name:

UOC Clinica Neurologica Metropolitana (NeuroMet)

Site location:

Via Altura 3

Site street address:

Via Altura 3

Site city:

Bologna

Site post code:

40139

Site country:

Italy

First name:

Cesa Lorella Maria

Last name:

Scaglione

Title:

Dr.

Telephone number:

+390514966919

Email:

cesa.scaglione@isnb.it

4.1.4 Denmark - Ongoing, recruitment ended

Planned number of subjects:

3

4.1.4.1 Site: Rigshospitalet

OMS ID:

ORG-100002431

Department name:

Hukommelsesklinikken

Site location:

Blegdamsvej 9

Site street address:

Blegdamsvej 9

Site city:

Copenhagen Oe

Site post code:

2100

Site country:

Denmark

First name:

Lena

Last name:

Hjermind

Title:

Dr.

Telephone number:

35458025

Email:

lena.elisabeth.hjermind.01@regionh.dk

4.1.5 Portugal - Ongoing, recruitment ended

Planned number of subjects:

10

4.1.5.1 Site: Hospital De Santa Maria E.P.E.

OMS ID:

ORG-100028629

Department name:

Neurociências e Saúde Mental

Site location:

Avenida Professor Egas Moniz Piso 3

Site street address:

Avenida Professor Egas Moniz Piso 3

Site city:

Lisbon

Site post code:

1649-028

Site country:

Portugal

First name:

Leonor

Last name:

Guedes

Title:

Prof.

Telephone number:

+351210405814

Email:

leonor.guedes@ulssm.min-saude.pt

4.1.5.2 Site: CNS Saude Lda.

OMS ID:

ORG-100044820

Department name:

Clínica Médica

Site location:

Bairro De Santo Antonio 47

Site street address:

Bairro De Santo Antonio 47

Site city:

Torres Vedras

Site post code:

2560-280

Site country:

Portugal

First name:

Joaquim

Last name:

Ferreira

Title:

Prof.

Telephone number:

+351261330702

Email:

science@cnscampus.com

4.1.6 Austria - Ongoing, recruitment ended

Planned number of subjects:

5

4.1.6.1 Site: Medizinische Universitaet Innsbruck

OMS ID:

ORG-100007200

Department name:

University clinic for neurology

Site location:

Anichstrasse 35

Site street address:

Anichstrasse 35

Site city:

Innsbruck

Site post code:

6020

Site country:

Austria

First name:

Klaus

Last name:

Seppi

Title:

Prof.

Telephone number:

+4351250425810

Email:

lki.ne.parkinson@tirol-kliniken.at

4.1.7 Spain - Ongoing, recruitment ended

Planned number of subjects:

43

4.1.7.1 Site: Hospital Universitario De Burgos

OMS ID:

ORG-100047051

Department name:

Neurología

Site location:

Avenida De Las Islas Baleares 3

Site street address:

Avenida De Las Islas Baleares 3

Site city:

Burgos

Site post code:

09006

Site country:

Spain

First name:

Esther

Last name:

Cubo Delgado

Title:

Dr.

Telephone number:

947256533

Email:

mcubo@saludcastillayleon.es

4.1.7.2 Site: Hospital Universitario Virgen De La Macarena

OMS ID:

ORG-100041183

Department name:

Neurología

Site location:

Avenida Del Doctor Fedriani 3

Site street address:

Avenida Del Doctor Fedriani 3

Site city:

Sevilla

Site post code:

41009

Site country:

Spain

First name:

Rafael

Last name:

Perez Noguera

Title:

Dr.

Telephone number:

955006627

Email:

rafapereznoguera@gmail.com

4.1.7.3 Site: Hospital Universitario De Badajoz

OMS ID:

ORG-100031808

Department name:

Neurología

Site location:

Avenida De 9 De Junio 2

Site street address:

Avenida De 9 De Junio 2

Site city:

Parla

Site post code:

28981

Site country:

Spain

First name:

David Jesus

Last name:

Ceberino Muñoz

Title:

Dr.

Telephone number:

924218100

Email:

david.ceberino@salud-juntaex.es

4.1.7.4 Site: Hospital Universitario Ramon Y Cajal

OMS ID:

ORG-100028538

Department name:

Neurología

Site location:

Carretera Del Colmenar Viejo Km 9 100, Por El Pardo

Site street address:

Carretera Del Colmenar Viejo Km 9 100

Site city:

Madrid

Site post code:

28034

Site country:

Spain

First name:

Jose Luis

Last name:

López-Sendón Moreno

Title:

Dr.

Telephone number:

913368821

Email:

jlsendonmoreno@salud.madrid.org

4.1.7.5 Site: Hospital Universitario De Cruces

OMS ID:

ORG-100028621

Department name:

Neurología

Site location:

Cruces Plaza S/n

Site street address:

Cruces Plaza S/n

Site city:

Barakaldo

Site post code:

48903

Site country:

Spain

First name:

Tamara

Last name:

Fernández Valle

Title:

Dr.

Telephone number:

946006363

Email:

Tamara.fernandezvalle@osakidetza.eus

4.1.7.6 Site: Hospital Universitario Y Politecnico La Fe

OMS ID:

ORG-100029610

Department name:

Neurología

Site location:

Avenida De Fernando Abril Martorell 106

Site street address:

Avenida De Fernando Abril Martorell 106

Site city:

Valencia

Site post code:

46026

Site country:

Spain

First name:

Carmen

Last name:

Peiró Vilaplana

Title:

Dr.

Telephone number:

+34682893185

Email:

peiro_marvil@gva.es

4.1.7.7 Site: Hospital De La Santa Creu I Sant Pau

OMS ID:

ORG-100028622

Department name:

Neurología

Site location:

Carrer De San Quinti 89

Site street address:

Carrer De San Quinti 89

Site city:

Barcelona

Site post code:

08041

Site country:

Spain

First name:

Jaime

Last name:

Kulisevsky Bojarski

Title:

Dr.

Telephone number:

649142360

Email:

jkulisevsky@santpau.cat

4.1.8 France - Ongoing, recruitment ended

Planned number of subjects:

27

4.1.8.1 Site: Les Hopitaux Universitaires De Strasbourg

OMS ID:

ORG-100006709

Department name:

Neurologie

Site location:

1 Avenue Moliere, Bp 49

Site street address:

1 Avenue Moliere

Site city:

Strasbourg Cedex 2

Site post code:

67098

Site country:

France

First name:

Thomas

Last name:

Wirth

Title:

Prof.

Telephone number:

0388128919

Email:

thomas.wirth@chru-strasbourg.fr

4.1.8.2 Site: Assistance Publique Hopitaux De Paris

OMS ID:

ORG-100004082

Department name:

Neurologie

Site location:

51 Av Du Mal De Lattre De Tassigny

Site street address:

51 Av Du Mal De Lattre De Tassigny

Site city:

Creteil

Site post code:

94000

Site country:

France

First name:

Katia

Last name:

Youssov

Title:

Prof.

Telephone number:

0149814301

Email:

katia.youssov@aphp.fr

4.1.8.3 Site: Centre Hospitalier Universitaire De Lille

OMS ID:

ORG-100006742

Department name:

Neurologie

Site location:

Rue Emile Laine

Site street address:

Rue Emile Laine

Site city:

Lille Cedex

Site post code:

59037

Site country:

France

First name:

Clémence

Last name:

Simonin

Title:

Dr.

Telephone number:

0320446752

Email:

clemence.simonin@chu-lille.fr

4.1.8.4 Site: Centre Hospitalier Universitaire De Toulouse

OMS ID:

ORG-100011252

Department name:

Neurologie

Site location:

1 Place Du Docteur Joseph Baylac

Site street address:

1 Place Du Docteur Joseph Baylac

Site city:

Toulouse

Site post code:

31300

Site country:

France

First name:

Jérémie

Last name:

Pariente

Title:

Prof.

Telephone number:

0561777686

Email:

jeremie.pariente@inserm.fr

4.1.8.5 Site: Centre Hospitalier Universitaire D'Angers

OMS ID:

ORG-100009206

Department name:

Neurologie

Site location:

4 Rue Larrey

Site street address:

4 Rue Larrey

Site city:

Angers

Site post code:

49100

Site country:

France

First name:

Christophe

Last name:

Verny

Title:

Prof.

Telephone number:

0241357855

Email:

chverny@chu-angers.fr

4.1.8.6 Site: Centre Hospitalier Universitaire De Bordeaux

OMS ID:

ORG-100008602

Department name:

Service de Génétique Médicale

Site location:

Place Amelie Raba Leon

Site street address:

Place Amelie Raba Leon

Site city:

Bordeaux

Site post code:

33000

Site country:

France

First name:

Cyril

Last name:

Goizet

Title:

Prof.

Telephone number:

0556795952

Email:

cyril.goizet@chu-bordeaux.fr

4.1.8.7 Site: Assistance Publique Hopitaux De Marseille

OMS ID:

ORG-100008698

Department name:

Neurologie

Site location:

264 Rue Saint Pierre

Site street address:

264 Rue Saint Pierre

Site city:

Marseille

Site post code:

13005

Site country:

France

First name:

Jean-Philippe

Last name:

Azulay

Title:

Prof.

Telephone number:

0491384333

Email:

Jean-philippe.AZULAY@ap-hm.fr

4.1.8.8 Site: Centre Hospitalier Universitaire De Montpellier

OMS ID:

ORG-100011148

Department name:

Neurologie

Site location:

80 Avenue Augustin Fliche

Site street address:

80 Avenue Augustin Fliche

Site city:

Montpellier Cedex 5

Site post code:

34295

Site country:

France

First name:

Cecilia

Last name:

Marelli

Title:

Dr.

Telephone number:

0467336029

Email:

c-marelli@chu-montpellier.fr

4.1.9 Countries outside of the European Economic Area

Countries outside of the European Economic Area:

United States, Australia, Argentina, Switzerland, United Kingdom, Canada, New Zealand

Participants in the rest of the world:

138

4.2 Sponsors

4.2.1 Sponsor:

F. Hoffmann-La Roche AG

4.2.1.1 Sponsor details

ID:

ORG-100001445

Name of sponsor organisation:

F. Hoffmann-La Roche AG

Address:

Grenzacherstrasse 124

Town/City:

Basel

Post code:

4058

Country:

Switzerland

Phone:

Email address:

4.2.1.2 Scientific contact point

Name of organisation:

F. Hoffmann-La Roche AG

Functional contact point name:

Trial Information System - TISL

Phone:

41616881111

Email address:

global.eudract@roche.com

4.2.1.3 Public contact point

Name of organisation:

F. Hoffmann-La Roche AG

Functional contact point name:

Trial Information System - TISL

Phone:

41616881111

Email address:

global.eudract@roche.com

4.2.1.4 Third parties associated with the trial

ID Organisation Name Address City Postcode Country Phone Email Duties
ORG-100011524 Labcorp Central Laboratory Services SARL Rue Moise-Marcinhes 7 Meyrin 1217 Switzerland +41588227901 ctasubmissions@labcorp.com Laboratory analysis
ORG-100044665 Publicis Healthcare Communications Group Limited Pembroke Building, Kensington Village, Avonmore Road London W14 8DG United Kingdom 441753833348 graham.robinson@publicislangland.com Other
ORG-100013039 Eresearchtechnology Inc. 1818 Market Street Ste 2600 Philadelphia 19103-3600 United States 18003092111 customercare@clario.com Other
ORG-100041455 Q2q Communications Limited 3 And 4 Floor, 25e The Quadrant Richmond TW9 1DJ United Kingdom 4402085699444 roche-genentech-rfp@q2q.co.uk Other
ORG-100041621 Greenphire LLC 1018 West 9th Avenue Suite 200 King Of Prussia 19406-1225 United States +12156094365 ctis_inquiry@greenphire.com Other
ORG-100011560 PPD Development LP 2244 Dabney Road Richmond 23230-3323 United States +18047997030 BioanalyticalQAManagement@ppd.com Laboratory analysis
ORG-100006268 Pharmaceutical Research Associates Group B.V. Amerikaweg 18 Assen 9407 TK Netherlands +31504022222 BA_Submissions@iconplc.com Other, Laboratory analysis
ORL-000000245 Charles River Laboratories International 22022 Transcandienne Senneville Quebec H9X 3R3 Canada 15146308200 regulatory-affairs@crl.com Other, Laboratory analysis
ORG-100042142 Ixico Technologies Limited 4th Floor, Griffin Court, 15 Long Lane London EC1A 9PN United Kingdom 4402037637490 info@ixico.com Other
ORG-100042527 Iqvia Laboratories Limited The Alba Campus, Rosebank Livingston EH54 7EG United Kingdom 4401506818094 John.mcilquham@iqvia.com Other, Laboratory analysis
ORG-100040604 Signant Health Global LLC 785 Arbor Way Blue Bell 19422-1986 United States +18887940122 IRTHelpdesk@signanthealth.com Other
ORG-100031937 MicroCoat Biotechnologie GmbH Am Neuland 3 Bernried Am Starnberger See 82347 Germany 490815899810 info@microcoat.de Laboratory analysis

4.2.2 Responsibilities of the sponsor

Sponsor(s) responsible for compliance:

Sponsor(s) responsible for being a contact point:

Sponsor(s) responsible for implementing the measures taken in accordance with article 77: