A Multicenter, Randomized, Double-blind, Placebo Controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington's Disease (SELECT-HD)

Wave Life Sciences Ltd.23 sites in 10 countries47 target enrollmentSeptember 6, 2021

ConditionsHuntington Disease

DrugsSAD: 30mg WVE-003 SAD: 60mg WVE-003 SAD: 90mg WVE-003 SAD: Pooled Placebo MD: 30mg WVE-003 MD: Placebo

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Huntington Disease
Sponsor: Wave Life Sciences Ltd.
Enrollment: 47
Locations: 23
Primary Endpoint: Safety: Proportion of Patients With Treatment Emergent Adverse Events (TEAEs) Related to Study Drug
Status: Completed
Last Updated: 8 months ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of WVE-003 in adult patients with early-manifest HD who carry the targeted single nucleotide polymorphism (SNP) - SNP3.

Registry

clinicaltrials.gov

Start Date

September 6, 2021

End Date

May 24, 2024

Last Updated

8 months ago

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

Wave Life Sciences Ltd.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion
•Ambulatory, male or female patients aged ≥25 to ≤60 years
•Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4
•UHDRS Total Functional Capacity Scores ≥9 and ≤13

Exclusion Criteria

•Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years
•Received any other study drug, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer, with the exception of the following:
•a. Received WVE-120101 or WVE-120102 within the last 3 months
•Implantable CNS device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan
•Inability to undergo brain MRI (with or without sedation)
•Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture
•Previously received tominersen

Outcomes

Primary Outcomes

Safety: Proportion of Patients With Treatment Emergent Adverse Events (TEAEs) Related to Study Drug

Time Frame: Day 1 through Week 24 (single ascending dose Period 1); Day 1 through Week 28 (multi dose Period 2)

The primary outcome for this study was safety and is reported as the proportion of patients with TEAEs related to study drug.

Secondary Outcomes

Pharmacokinetics of WVE-003 in Plasma(Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2))
Concentration of WVE-003 in Cerebrospinal Fluid (CSF)(28 days post-dose during Period 1 (P1:Day29); 28 days post last dose during Period 2 (P2: Day141))