PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD
Purpose
This study will evaluate the efficacy and safety of pridopidine 45mg twice daily (BID) in patients with early stage manifest Huntington Disease (HD).
Condition
- Huntington Disease
Eligibility
- Eligible Ages
- Over 25 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
MAIN STUDY 1. Diagnosis of HD based on clinical features and the presence of ≥36 CAG repeats in the huntingtin gene 2. Diagnostic confidence level (DCL) of 4 3. Adult-onset HD with onset of signs and symptoms ≥18 years of age 4. Stage 1 or Stage 2 HD, defined as a UHDRS-TFC score of ≥7, at screening
Exclusion Criteria
- Use of pridopidine within 12 months before the baseline visit. 2. Gene therapy at any time 3. Any serious medical condition or clinically significant laboratory, or vital sign abnormality that precludes the patient's safe participation in and completion of the study e.g. significant heart disease within 12 weeks before baseline or history of certain cardiac arrhythmias 4. History of epilepsy or seizures within the last 5 years 5. Pregnant or breastfeeding, or intention to become pregnant during the study
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Experimental Pridopidine |
45 mg pridopidine twice daily (BID) |
|
Placebo Comparator Placebo |
Matching placebo |
|
More Details
- Status
- Active, not recruiting
- Sponsor
- Prilenia
Study Contact
Detailed Description
This is a phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of pridopidine 45 mg BID in patients with early stage HD. Eligible patients who completed the Main Study (65 to 78 weeks) will have the option to enroll into an open-label extension.