PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Arm, Multicenter Study Evaluating the Efficacy and Safety of Pridopidine in Patients With Early Stage of Huntington Disease

Sponsors

Lead Sponsor: Prilenia

Source Prilenia
Brief Summary

This study will evaluate the efficacy and safety of pridopidine 45mg twice daily (BID) in patients with early stage manifest Huntington Disease (HD).

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.

Overall Status Not yet recruiting
Start Date October 15, 2020
Completion Date April 7, 2023
Primary Completion Date March 3, 2023
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Change from baseline in the Unified Huntington Disease Rating Scale-Total functional capacity (UHDRS-TFC) score Baseline, 65 weeks
Enrollment 480
Condition
Intervention

Intervention Type: Drug

Intervention Name: Pridopidine

Description: Pridopidine hard gelatin capsule

Arm Group Label: Pridopidine

Intervention Type: Drug

Intervention Name: Placebo

Description: Pridopidine-matching placebo hard gelatin capsule

Arm Group Label: Placebo

Eligibility

Criteria:

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 1. 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

Gender: All

Minimum Age: 25 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Contact

Last Name: Kristin Strazdins

Phone: 1-585-242-0967

Email: [email protected]

Verification Date

October 2020

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Pridopidine

Type: Experimental

Description: 45 mg pridopidine twice daily (BID)

Label: Placebo

Type: Placebo Comparator

Description: Matching placebo

Patient Data No
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Source: ClinicalTrials.gov