Active clinical trials for "Huntington Disease"

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Descriptive analysis of N- homocysteinylated Huntingtin in 3 groups of human fibroblasts: presymptomatic HD individuals with UHDRS motor ≤ 5 (Mutated Huntingtin), symptomatic HD individuals with motor UHDRS > 5 (Mutated Huntingtin) human control cell lines, unmutated Huntingtin

Intro Huntington's disease (HD) patients suffer from motor, cognitive and behavioral impairments, with heterogeneous phenotypes and variable time course. This leads to a high variance of HD markers, none of which is currently sensitive enough to 1) measure disease progression from small cohort data, 2) predict disease entry in carriers of the HD mutation (during the prodromal phase or in patients considered asymptomatic: pre-HD patients), and 3) measure a significant evolution of the state of pre-HD patients over a time window compatible with the realization of clinical trials (about 2/3 years). Moreover, the markers of HD do not allow a fine stratification of the patients. Hypothesis/Objective Our objectives are 1) to evaluate the sensitivity of new markers and assessment tools for symptomatic (HD) and presymptomatic (pre-HD) patients, 2) to define a model of disease progression, and 3) to establish an enrichment strategy to improve patient selection for future therapeutic trials. Method We will evaluate newly developed cognitive tests, multimodal imaging techniques, biological markers and use innovative statistical approaches. We will follow 60 patients with the mutation responsible for MH (40 presymptomatic pre-MH patients, 20 symptomatic MH patients) and 20 healthy volunteers (controls) over a 24-month period.

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of BN82451B versus placebo after oral administration twice daily (bid) for 28 days in patients with Huntington's Disease (HD).

Phase A: Recruit 50 patients with HD, and their caregivers, to complete a neuropsychiatric and quality of life battery of scales at baseline. Have these 50 patients complete a formal psychiatric assessment with a psychiatrist within 2 weeks of this clinical battery, and the results of these 2 types of assessments will be compared to establish the level of agreement between clinical rating scales and formal psychiatric assessment. Phase B: Continue to follow Phase A cohort longitudinally and administer neuropsychiatric and quality of life battery at 6 months, 12 months, and 18 months form baseline. Recruit an additional 50 patients, administer the same neuropsychiatric and quality of life battery at baseline, implement medication and counseling intervention according to a standard of care protocol, and follow up with the same neuropsychiatric and quality of life battery at 6, 12, and 18 months.

WVE-HDSNP1-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120101 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism, rs362307 (SNP1). To participate in the study, patients must have completed the Phase 1b/2a clinical study WVE-HDSNP1-001.

PRECISION-HD2 is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of WVE-120102 in adult patients with early manifest Huntington's disease (HD) who carry a targeted single nucleotide polymorphism (SNP) rs362331 (SNP2).

To assess the safety and tolerability of five doses of PBF-999 (160 mg and 320mg) after repeated (8 days) single daily oral dose administration in young male and female healthy subjects.

WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism, rs362331 (SNP2). To participate in the study, patients must have completed the Phase 1b/2a clinical study WVE-HDSNP2-001.

The purpose of the study is to collect and assess long term data on the safety, tolerability, and efficacy of pridopidine in patients with Huntington's disease (HD).