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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03761849
Ethics application status
Date submitted
30/11/2018
Date registered
3/12/2018
Date last updated
1/03/2024

Titles & IDs
Public title
A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants 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) Score-Z Score
Timepoint [1] 0 0
Weeks 21 for ODC and 69 for NDC
Primary outcome [2] 0 0
Change From Baseline in the Total Functional Capacity (TFC) Score
Timepoint [2] 0 0
Weeks 21 for ODC and 69 for NDC
Secondary outcome [1] 0 0
Change From Baseline in Total Motor Score (TMS)
Timepoint [1] 0 0
Weeks 21 for ODC and 69 for NDC
Secondary outcome [2] 0 0
Change From Baseline in Symbol Digit Modalities Test (SDMT) Scores' Least Squares Mean Values
Timepoint [2] 0 0
Weeks 21 for ODC and 69 NDC
Secondary outcome [3] 0 0
Change From Baseline in Stroop Word Reading (SWR) Test Scores' Least Squares Mean Values
Timepoint [3] 0 0
Weeks 21 for ODC and 69 for NDC
Secondary outcome [4] 0 0
Change From Baseline in the Clinical Global Impression, Severity Scale (CGI-S) Scores' Least Squares Mean Values
Timepoint [4] 0 0
Week 69 for NDC Only
Secondary outcome [5] 0 0
Percentage of Patients With a Decrease From Baseline of >=1 Point on the Total Functional Capacity (TFC) Score
Timepoint [5] 0 0
Week 69 for NDC only
Secondary outcome [6] 0 0
Percentage of Patients With a Decline From Baseline of >=1.2 Points on the Composite Unified Huntington's Disease Rating Scale-cUHDRS Score
Timepoint [6] 0 0
Week 69 for NDC Only
Secondary outcome [7] 0 0
Percentage of Patients With an Unchanged or Improved Score on the Clinical Global Impression, Change Scale Score
Timepoint [7] 0 0
Weeks 53 and 69 NDC only
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
Up to Week 21 for ODC, Up to Week 69 for NDC
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
For ODC at 13th Month, for NDC at Week 101
Secondary outcome [11] 0 0
Concentration of RO7234292 in Plasma
Timepoint [11] 0 0
Week 21 for ODC and Week 69 for NDC
Secondary outcome [12] 0 0
Trough Concentration of RO7234292 in Cerebrospinal Fluid (CSF)
Timepoint [12] 0 0
Week 21 for ODC and Week 69 for NDC
Secondary outcome [13] 0 0
Incidence of Anti-Drug Antibodies (ADAs).
Timepoint [13] 0 0
Week 21 for ODC and Week 69 for NDC
Secondary outcome [14] 0 0
Titer and Antibody Subtype, Determined if ADAs Are Identified
Timepoint [14] 0 0
Week 21 for ODC and Week 69 for NDC
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
Week 13 for ODC
Secondary outcome [17] 0 0
Change From Baseline in CSF Neurofilament Light Chain (NfL) Proteint Level
Timepoint [17] 0 0
Week 21 for ODC, Weeks 21 and 69 for NDC

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
Sex
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
Completed
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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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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British Columbia
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Nova Scotia
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Chile
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Aarhus N
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København Ø
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Lübeck
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Ulm
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Puglia
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Mie
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Groningen
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Christchurch
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Wellington
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Gdansk
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Moskovskaja Oblast
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Gümligen
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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
Trial website
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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.