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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03761849




Registration number
NCT03761849
Ethics application status
Date submitted
30/11/2018
Date registered
3/12/2018
Date last updated
1/08/2019

Titles & IDs
Public title
A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Patients With Manifest Huntington's Disease
Scientific title
A Randomized, Multicenter, Double-Blind, Placebo-Controlled, Phase III Clinical Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Patients With Manifest Huntington's Disease
Secondary ID [1] 0 0
GENERATION HD1
Secondary ID [2] 0 0
BN40423
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Huntingtons Disease 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - RO7234292
Treatment: Drugs - Placebo

Experimental: RO7234292 Q8W - RO4234292 is administered intrathecally every 8 weeks.

Experimental: RO7234292 Q16W - RO7234292 is administered intrathecally every 16 weeks. Participants in this arm will also receive placebo at alternate weeks to keep the blind.

Placebo Comparator: Placebo - Placebo will be administered every 8 weeks by IT injection.


Treatment: Drugs: RO7234292
Intrathecal injection

Treatment: Drugs: Placebo
Intrathecal injection

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS)
Timepoint [1] 0 0
Baselined, Week 101
Primary outcome [2] 0 0
Change From Baseline in the TFC Score
Timepoint [2] 0 0
Baseline, Week 101
Secondary outcome [1] 0 0
Change From Baseline in Total Motor Score (TMS)
Timepoint [1] 0 0
Baseline, Week 101
Secondary outcome [2] 0 0
Change From Baseline in Symbol Digit Modalities Test (SDMT)
Timepoint [2] 0 0
Baseline, Week 101
Secondary outcome [3] 0 0
Change From Baseline in Stroop Word Reading (SWR) Test
Timepoint [3] 0 0
Baseline, Week 101
Secondary outcome [4] 0 0
Change From Baseline in the Clinical Global Impression, Severity Scale (CGI-S)
Timepoint [4] 0 0
Baseline, Week 101
Secondary outcome [5] 0 0
Percentage of Patients with a Decrease From Baseline of >=1 point on the TFC
Timepoint [5] 0 0
Baseline, Week101
Secondary outcome [6] 0 0
Percentage of Patients With a Decline From Baseline of >=1.2 Points on the cUHDRS
Timepoint [6] 0 0
Baseline, Week 101
Secondary outcome [7] 0 0
Percentage of Patients With an Unchanged or Improved Score on the Clinical Global Impression, Change Scale (CGI-C) Score
Timepoint [7] 0 0
Baseline to Week 101
Secondary outcome [8] 0 0
Percentage of Participants with Adverse Events
Timepoint [8] 0 0
Up to 117 Weeks (29 months)
Secondary outcome [9] 0 0
Change From Baseline in Montreal Cognitive Assessment (MoCA)
Timepoint [9] 0 0
Screening, Baseline, Weeks 5, 21, 37, 53, 69, 85 and 101 and early treatment termination
Secondary outcome [10] 0 0
Percentage of Participants With Suicidal Ideation or Behavior, as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Score
Timepoint [10] 0 0
Screening, Baseline, Weeks 5, 13, 21, 29, 37, 45, 53, 61, 69, 77, 85, 93, 101, 117, end of treatment for early treatment termination and at early treatment termination
Secondary outcome [11] 0 0
Concentration of RO7234292 in Plasma
Timepoint [11] 0 0
Baseline, Weeks 13, 21, 37, 53, 69, 85, and 101 and early treatment termination
Secondary outcome [12] 0 0
Trough Concentration of RO7234292 in CSF
Timepoint [12] 0 0
Baseline, Weeks 13, 21, 37, 53, 69, 85, and 101 and early treatment termination
Secondary outcome [13] 0 0
Incidence of Anti-Drug Antibodies (ADAs)
Timepoint [13] 0 0
Baseline, Weeks 13, 21, 37, 53, 69, 85, and 101 and early treatment termination and safety follow-up visit
Secondary outcome [14] 0 0
Titer and Antibody Subtype, determined if ADAs are Identified
Timepoint [14] 0 0
Baseline, Weeks 13, 21, 37, 53, 69, 85, and 101 and early treatment termination and safety follow-up visit
Secondary outcome [15] 0 0
Change From Baseline in CSF mHTT Protein Level
Timepoint [15] 0 0
Baseline, Week 101
Secondary outcome [16] 0 0
Change From Baseline in Whole and Regional Brain Volumes, as detrmined by structural magnetic resonance imaging (MRI)
Timepoint [16] 0 0
Baseline, Week 101
Secondary outcome [17] 0 0
Change From Baseline in CSF Neurofilament Light Chain (NfL) Proteint Level
Timepoint [17] 0 0
Baseline, Week 101

Eligibility
Key inclusion criteria
- Manifest HD diagnosis, defined as a DCL score of 4

- Independence Scale (IS) score >= 70

- Genetically confirmed disease by direct DNA testing with a CAP score >400

- Clinical assessment to ensure individual has intact functional independence at
baseline to maintain self-care and core activities of daily living (ADLs).
Minimum age
25 Years
Maximum age
65 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Any serious medical condition or clinically significant laboratory, or vital sign
abnormality or claustrophobia at screening that, in the investigator's judgment,
precludes the patient's safe participation in and completion of the study

- Pregnant or breastfeeding, or intending to become pregnant during the study or within
5 months after the final dose of study drug

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
WESTMEAD HOSPITAL; Deparment of Neurology - Westmead
Recruitment hospital [2] 0 0
Monash Medical Centre - Clayton
Recruitment hospital [3] 0 0
Royal Melbourne Hospital; Department of Neurology - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
3168 - Clayton
Recruitment postcode(s) [3] 0 0
3050 - Parkville
Recruitment outside Australia
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United States of America
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Alabama
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Arizona
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California
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Colorado
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District of Columbia
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Florida
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Illinois
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Massachusetts
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Missouri
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Pennsylvania
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Tennessee
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Texas
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Utah
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Washington
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Argentina
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Caba
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Argentina
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Capital Federal
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Argentina
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Ciudad Autonoma Buenos Aires
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Austria
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Innsbruck
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Austria
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Salzburg
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Canada
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Alberta
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Canada
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British Columbia
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Canada
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Nova Scotia
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Ontario
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Quebec
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Denmark
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Aarhus N
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Denmark
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København Ø
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France
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Amiens Cedex1
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France
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Angers Cedex 9
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France
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Creteil
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Lille
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Bochum
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Germany
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Bonn
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Germany
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Erlangen
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Germany
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Lübeck
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Germany
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Münster
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Germany
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Ulm
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Italy
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Emilia-Romagna
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Lazio
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Italy
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Liguria
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Puglia
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Mie
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Miyagi
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Niigata
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Auckland
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Christchurch
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Wellington
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Gdansk
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Krakow
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Warszawa
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Russian Federation
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Krasnojarsk
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Russian Federation
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Moskovskaja Oblast
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Tatarstan
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Vizcaya
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Basel
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Gümligen
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Aberdeen
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Birmingham
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Cambridge
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Cardiff
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Oxford
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Sheffield
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United Kingdom
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Southhampton

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Hoffmann-La Roche
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study will evaluate the efficacy, safety, and biomarker effects of RO7234292 (RG6042)
compared with placebo in patients with manifest Huntington's disease (HD).
Trial website
https://clinicaltrials.gov/show/NCT03761849
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Trials
Address 0 0
Hoffmann-La Roche
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Reference Study ID Number: BN40423 www.roche.com/about_roche/roche_worldwide.htm
Address 0 0
Country 0 0
Phone 0 0
888-662-6728 (U.S. and Canada)
Fax 0 0
Email 0 0
global-roche-genentech-trials@gene.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03761849