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

Prilenia

Status and phase: Completed; Phase 3

Conditions: Huntington Disease

Treatments: Drug: Placebo; Drug: Pridopidine

Study type: Interventional

Funder types: Industry

Identifiers

NCT04556656

PL101-HD301

2020-002822-10 (EudraCT Number)

Details and patient eligibility

About

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

Full 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.

Enrollment: 499 patients

Sex: All

Ages: 25+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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499 participants in 2 patient groups, including a placebo group

Pridopidine

Experimental group

Description:

45 mg pridopidine twice daily (BID)

Treatment:

Drug: Pridopidine

Placebo

Placebo Comparator group

Description:

Matching placebo

Treatment:

Drug: Placebo