- 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.
PRECISE-HD
Not yet recruiting
Official Title: A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Huntington’s Disease (HD)
A previous study called PROOF-HD tested pridopidine in about 500 people with Huntington’s disease. The overall result was disappointing because the drug did not clearly outperform placebo in the entire group. However, researchers noticed something interesting: people who were not taking certain HD medications (antipsychotics or anti-chorea drugs) appeared to decline more slowly when taking pridopidine.
PRECISE-HD is a “second chance” Phase 3 trial designed to find out whether pridopidine can genuinely slow HD progression after promising signals were seen in a subgroup of patients in the earlier PROOF-HD study.
This Phase 3 study consists of two study periods, a randomized, double-blind, placebo1-year study period and a 2-year open-label extension (OLE) period .
The study looks at the effect of pridopidine 45 mg twice daily on HD in participants who are not using antidopaminergic medications (ADMs) at study start.
Reseachers then track:
- Ability to perform daily activities
- Thinking and memory
- Movement problems
- Overall disease progression
- Safety and side effects
Phase: 3
Subtype: Interventional Therapeutic
Primary Purpose: Treatment
Intervention Type: small molecule.
Primary Intervention: Drug: Pridopidine
Duration: 12 months
Ages Eligible
for Study:
23 Years to 65 Years
Sexes Eligible
for Study:
All
Accepts Healthy Volunteers:
No
- 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.
Ages Eligible for Study
23 Years to 65 Years (Adult, Older Adult )
Sexes Eligible for Study
All
Accepts Healthy Volunteers
No
LOCATIONS
To be confirmed
