SNPMiner Trials by Shray Alag


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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 196

Description: tmax of WVE-120101 in plasma

Measure: PK: Time of occurrence of Cmax (tmax)

Time: Day 1 to Day 196

Description: 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 196

Description: Elimination rate of WVE-120101 from plasma

Measure: PK: Terminal elimination rate constant

Time: Day 1 to Day 196

Description: Concentration of mutant huntingtin (mHTT) protein in CSF

Measure: Pharmacodynamics (PD)

Time: Day 1 to Day 196

Description: 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 140

Other 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 140

Description: 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 140

Description: Changes from baseline MRI of the brain.

Measure: Magnetic Resonance Imaging

Time: Screening to Day 140


HPO Nodes