Huntington's Disease Clinical Trials

VX15/2503-N-131 is a Phase 2, multi-center, randomized, double-blind, placebo controlled study of VX15/2503 in subjects with late prodromal and early manifest Huntington's disease. The primary objective is to evaluate the safety and tolerability of monthly IV administration of a single dosage of VX15/2503 (or placebo). Efficacy endpoints include determining the ...

This clinical trial is a phase 2, double-blind dose escalation regimen of once-weekly OPK-88003 in subjects with T2DM. The trial consists of four phases: a screening/baseline phase (up to 2 weeks prior to first dose), a 30-week treatment period consisting of a dose escalation phase (8 weeks) and a target ...

In the study the investigators plan to include 100 early affected HD patients (5 UHDRS 40) in a randomized, double-blind, controlled study in 2 centers (France and the Netherlands). Patients will receive either triheptanoin at 1g/kg of body weight per day (n = 50), or a control oil (n = ...

This will be a 12-week, randomized, double-blind, placebo-controlled, 4-arm, parallel group, multi-center study to evaluate the safety and efficacy of LGD-6972 in subjects with T2DM inadequately controlled on metformin monotherapy (a stable [≥12 weeks], daily dose of ≥1000mg at randomization). Subjects with T2DM will be treated with one of 3 ...

The purpose of this study is to assess the efficacy, safety, and tolerability of multiple doses of gemcabene 600 mg QD compared to placebo in patients with hypercholesterolemia not adequately controlled on high-intensity or moderate-intensity stable statin therapy. Patients with HeFH, ASCVD, or otherwise uncontrolled, may be included with baseline ...

The primary objective of this study is the proof of mechanism and support of dose finding, together with the safety evaluation in patients with clinical evidence of NASH. To gain further insight into clinical effects of AOC3 inhibition on NASH further exploratory analyses of biomarkers related to NASH and liver ...

The purpose of this research study is to study the safety and efficacy of fenofibrate, an FDA-approved drug for high cholesterol and/or elevated triglyerides (fats), as a treatment for Huntington's disease (HD). Subjects who meet the entry criteria will be randomized (3:1) to either 145mg of fenofibrate or placebo.

This is a Phase 2, randomized, active-controlled, open-label study with a 3-arm parallel design. Healthy 2-month old infants (42 to 98 days of age) with no history of pneumococcal vaccination will be randomized in a 1:1:1 ratio to receive a 4-dose series of: multivalent pneumococcal conjugate vaccine coadministered with Prevnar ...

This is a phase II dose-response study in which participants with HD will receive three intravenous injections of the investigational product or placebo (one every month for three months) a total of three cycles. The subjects will be randomized in 2: 2: 1 ratio for the groups G1: lower dose ...

This study will test the safety, tolerability, pharmacokinetics and pharmacodynamics of IONIS-HTTRx administered intrathecally to adult patients with Huntington's Disease.