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

Huntington Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05032196
• Other study ID #: WVE-003-001
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: September 6, 2021
• Est. completion date: December 2024

Study information

• Verified date: November 2023
• Source: Wave Life Sciences Ltd.
• Contact: Clinical Operations
• Phone: 855-215-4687
• Email: clinicaltrials[@]wavelifesci.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 54
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 25 Years to 60 Years

Eligibility

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

Related Conditions & MeSH terms

• Huntington Disease

Intervention

Drug:
• WVE-003
WVE-003 is a stereopure antisense oligonucleotide (ASO)

Locations

Country	Name	City	State
Australia	Monash Health	Clayton	Victoria
Australia	Royal Melbourne Hospital	Melbourne	Victoria
Australia	Westmead Hospital	Westmead	New South Wales
Canada	University of Alberta Hospital	Edmonton	Alberta
Canada	Centre Hospitalier de l-Universite de Montreal	Montréal	Quebec
Canada	The Ottawa Hospital	Ottawa	Ontario
Denmark	Rigshospitalet	Copenhagen
France	Hopital Henri Mondor - Hospital	Créteil
France	Institut du Cerveau et de la Moelle Epiniere	Paris
Germany	Katholisches Klinikum Bochum gGmbH	Bochum
Germany	George-Huntington-Institut GmbH	Muenster
Germany	kbo-Isar-Amper-Klinikum Taufkirchen (Vils)	Taufkirchen
Italy	Centro Ricerche Cliniche Di Verona	Verona
Netherlands	Leiden University Medical Center	Leiden
Netherlands	Maastricht University Medical Center	Maastricht
Poland	Szpital Sw. Wojciecha	Gdansk
Poland	Instytut Psychiatrii I Neurologii	Warsaw
Spain	Hospital de la Sanata Creu i Sant Pau	Barcelona
Spain	Hospital Universitario Ramón y Cajal	Madrid
United Kingdom	Cardiff University, Schools of Medicine and Biosciences	Cardiff	Wales
United Kingdom	Royal Devon and Exeter Hospital NHS Trust	Exeter	Devon
United Kingdom	Royal Hospital for Children, Pharmacy Aseptic Unit	Glasgow	Glasgow City
United Kingdom	Royal Liverpool University Hospital	Liverpool

Sponsors (1)

Lead Sponsor	Collaborator
Wave Life Sciences Ltd.

Countries where clinical trial is conducted

Australia,  Canada,  Denmark,  France,  Germany,  Italy,  Netherlands,  Poland,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety: Proportion of patients with adverse events (AEs)		Day 1 through end of study (minimum of 36 weeks)
Secondary	Maximum concentration (Cmax) of WVE-003 in plasma		Day 1 through end of study (minimum of 36 weeks)
Secondary	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)
Secondary	Terminal half-life (t1/2) of WVE-003 in cerebrospinal fluid (CSF)		Day 1 through end of study (minimum of 36 weeks)