SNPMiner Trials (Home Page)
Report for SNP rs362307
Developed by Shray Alag, 2020.
SNP Clinical Trial Gene
There is one clinical trial.
Clinical Trials
1 A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-120101 Administered Intrathecally in Patients With Huntington's Disease
PRECISION-HD1 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-120101 in adult patients with early manifest Huntington's disease (HD) who carry a targeted single nucleotide polymorphism (SNP) rs362307 (SNP1).
NCT03225833 Huntington's Disease Drug: WVE-120101 Drug: Placebo MeSH:Huntington Disease
Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease PRECISION-HD1 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-120101 in adult patients with early manifest Huntington's disease (HD) who carry a targeted single nucleotide polymorphism (SNP) rs362307 (SNP1).
Primary Outcomes
Description: All AEs reported or observed during the study, including AEs resulting from concurrent illnesses, reactions to concurrent medications, or progression of disease states
Measure: Safety: Number of patients with adverse events (AEs) Time: Day 1 to Day 210 (end of study)Description: Severity will be evaluated using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
Measure: Safety: Severity of AEs Time: Day 1 to Day 210 (end of study)Description: An SAE is defined as any event that results in death, is immediately life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or is a congenital anomaly/birth defect not present at Prescreening.
Measure: Safety: Number of patients with serious AEs (SAEs) Time: Day 1 to Day 210 (end of study)Measure: Safety and Tolerability: Number of patients who withdraw due to AEs Time: Day 1 to Day 210 (end of study)
Secondary Outcomes
Description: Cmax of WVE-120101 in plasma
Measure: Pharmacokinetics (PK): Maximum observed concentration (Cmax) Time: Day 1 to Day 196Description: tmax of WVE-120101 in plasma
Measure: PK: Time of occurrence of Cmax (tmax) Time: Day 1 to Day 196Description: AUC 0-t from time zero to the last quantifiable concentration of WVE-120101 in plasma
Measure: PK: Area under the plasma concentration-time curve (AUC 0-t) Time: Day 1 to Day 196Description: Elimination rate of WVE-120101 from plasma
Measure: PK: Terminal elimination rate constant Time: Day 1 to Day 196Description: Concentration of mutant huntingtin (mHTT) protein in CSF
Measure: Pharmacodynamics (PD) Time: Day 1 to Day 196Description: Change from baseline to the last measured time point (Day 140) and difference from placebo in the TFC, administered as part of the Unified Huntington's Disease Rating Scale (UHDRS).
Measure: Clinical Effects: Total Functional Capacity (TFC) Time: Day 1 to Day 140Other Outcomes
Description: Change from baseline to the last measured time point (Day 140) and difference from placebo in the UHDRS
Measure: UHDRS Time: Day 1 to Day 140Description: Change from baseline to the last measured time point (Day 140) and difference from placebo in the PBA-s
Measure: Short Problems Behavior Assessment (PBA-s) Time: Day 1 to Day 140Description: Changes from baseline MRI of the brain.
Measure: Magnetic Resonance Imaging Time: Screening to Day 140