Study of WVE-003 in Patients With Huntington's Disease

Phase 1

Completed

• Conditions: Huntington Disease
• Interventions: Drug: WVE-003

• Registration Number: NCT05032196
• Lead Sponsor: Wave Life Sciences Ltd.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-08-18
• Study First Submitted QC: 2021-08-26
• Study First Posted: 2021-09-02
• Study Start: 2021-09-06
• Status Verified: 2023-11
• Primary Completion: 2024-05-24
• Study Completion: 2024-05-24
• Last Update Submitted: 2024-07-17
• Last Update Posted: 2024-07-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 47

Inclusion Criteria

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

Exclusion Criteria

1. Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years

2. 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

3. Implantable CNS device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan

4. Inability to undergo brain MRI (with or without sedation)

5. Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture

6. Previously received tominersen

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
WVE-003 (Dose B) or placebo	WVE-003	-
WVE-003 (Dose A) or placebo	WVE-003	-
WVE-003 (Dose C) or placebo	WVE-003	-

Primary Outcome Measures

Name	Time	Method
Safety: Proportion of patients with adverse events (AEs)	Day 1 through end of study (minimum of 36 weeks)

Secondary Outcome Measures

Name	Time	Method
Area under the plasma concentration time curve for WVE-003 from time 0 to last quantifiable concentration (AUC0-t)	Day 1 through end of study (minimum of 36 weeks)
Terminal half-life (t1/2) of WVE-003 in cerebrospinal fluid (CSF)	Day 1 through end of study (minimum of 36 weeks)
Maximum concentration (Cmax) of WVE-003 in plasma	Day 1 through end of study (minimum of 36 weeks)

Trial Locations

Locations (23)

Westmead Hospital; 🇦🇺 Westmead, New South Wales, Australia

Monash Health; 🇦🇺 Clayton, Victoria, Australia

Royal Melbourne Hospital; 🇦🇺 Melbourne, Victoria, Australia

University of Alberta Hospital; 🇨🇦 Edmonton, Alberta, Canada

The Ottawa Hospital; 🇨🇦 Ottawa, Ontario, Canada

Centre Hospitalier de l-Universite de Montreal; 🇨🇦 Montréal, Quebec, Canada

Rigshospitalet; 🇩🇰 Copenhagen, Denmark

Hopital Henri Mondor - Hospital; 🇫🇷 Créteil, France

Institut du Cerveau et de la Moelle Epiniere; 🇫🇷 Paris, France

Katholisches Klinikum Bochum gGmbH; 🇩🇪 Bochum, Germany

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