Pridopidine for Huntington's Disease: a 1-year, randomized, placebo-controlled study

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 3, randomized, double-blind, placebo-controlled study followed by a 2-year open-label extension. Adults with Huntington's disease (ages 23-65) who are not using antidopaminergic medications at screening will receive pridopidine 45 mg twice daily or placebo for 12 months, with a small titration period at start. The study aims to measure overall clinical progression and changes in functional capacity, cognition, motor function, quality of life, and speech. Participants will have six in-clinic visits during the placebo-controlled period, and an open-label extension will follow to evaluate longer-term effects. Lead sponsor is Prilenia, with collaborators Ferrer Internacional, at sites including UAB, UCSD, and UCLA.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Adult-onset HD (onset of signs and symptoms and a clinical diagnosis at ≥21 years of age).
* A diagnosis based on clinical features and the presence of ≥40 CAG repeats in the huntingtin (HTT) gene confirmed by historical laboratory quanitified results or by a diagnostic test at Screening.
* Diagnostic confidence level (DCL) of 4 (DCL=4 unequivocal motor signs, ≥99% confidence) on the standardized motor exam Total Motor Score (TMS).
* Total Functional Capacity (TFC) score of ≥7 at Screening and Baseline.
* Cytosine-Adenine-Guanine (CAG)-Age Product (CAP)100 score ≥95 at Screening.
* Independence Scale (IS) score ≤90% at Screening.
* Total Motor Score (TMS) of ≥20 at Screening and Baseline.
* Not using ADMs (VMAT2i and neuroleptics/antipsychotics) for at least 6 months prior to Screening visit. Importantly, it is not encouraged to discontinue the participants' ADM treatment solely for enrollment in the current trial.

Exclusion Criteria:

* Clinically significant cardiovascular disease (e.g. QTcF \>450 msec \[males\] or \>470 msec \[females\], arrhythmias, uncontrolled atrial fibrillation, or congenital long QT syndrome), seizure history ≤5 years, significant neurological disorders (e.g. intracranial pathology or cerebrovascular events), active/recent malignancy (unless localized and resolved), or any serious or uncontrolled systemic disease (e.g. hepatic, renal, respiratory, endocrine, infectious \[HBV, HCV, HIV\], or psychiatric) that may pose safety risk or interfere with participation.
* Severe hepatic or renal impairment.
* Any mutant huntingtin (mHTT) lowering therapy in the past year.
* Medications that prolong QT interval, taken within 4 weeks of the baseline visit.
* Use of pridopidine within 6 weeks or 5 half-lives before the screening visit.
* Previous participation in intracranial gene therapy study.
* Laboratory values that fall outside of the central laboratory's reference range at Screening and are considered clinically significantly abnormal by the Investigator and affect the participant's suitability to participate in the study or put the participant at risk if he/she enters the study in the Investigator's opinion.
* Female participants who are pregnant, planning to become pregnant or breastfeeding.

Where it's running

• The University of Alabama at Birmingham
  Birmingham, Alabama, United States Recruiting
• University of California, San Diego
  La Jolla, California, United States Recruiting
• Hereditary Neurological Disease Centre
  Wichita, Kansas, United States Recruiting
• Struthers Parkinson's Center / Health Partners Institute
  Golden Valley, Minnesota, United States Recruiting
• Columbia University
  New York, New York, United States Recruiting
• Duke University
  Durham, North Carolina, United States Recruiting

+16 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Prilenia Medical Information
• Email: MedInfo@prilenia.com
• Phone: 018575745755

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.