SNPMiner Trials by Shray Alag


SNPMiner Trials: Clinical Trial Report


Report for Clinical Trial NCT03225846

Developed by Shray Alag, 2019.
SNP Clinical Trial Gene

A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-120102 Administered Intrathecally in Patients With Huntington's Disease

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).

NCT03225846 Huntington's Disease
MeSH: Huntington Disease

2 Interventions

Name: WVE-120102

Description: WVE-120102 is a stereopure antisense oligonucleotide (ASO)

Type: Drug

WVE-120102 (Dose A) or placebo WVE-120102 (Dose B) or placebo WVE-120102 (Dose C) or placebo WVE-120102 (Dose D) or placebo

Name: Placebo

Description: 0.9% Sodium Chloride

Type: Drug

WVE-120102 (Dose A) or placebo WVE-120102 (Dose B) or placebo WVE-120102 (Dose C) or placebo WVE-120102 (Dose D) or placebo


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-120102 in plasma

Measure: Pharmacokinetics (PK): Maximum observed concentration (Cmax)

Time: Day 1 to Day 196

Description: tmax of WVE-120102 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-120102 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-120102 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

Purpose: Treatment

Allocation: Randomized

Sequential Assignment


There is one SNP

SNPs


1 rs362331

Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease 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).



HPO Nodes