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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05032196
Recruitment Status : Recruiting
First Posted : September 2, 2021
Last Update Posted : January 6, 2023
Sponsor:
Information provided by (Responsible Party):
Wave Life Sciences Ltd.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Huntington Disease Drug: WVE-003 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
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)
Actual Study Start Date : September 6, 2021
Estimated Primary Completion Date : June 2023
Estimated Study Completion Date : June 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: WVE-003 (Dose A) or placebo Drug: WVE-003
WVE-003 is a stereopure antisense oligonucleotide (ASO)
Experimental: WVE-003 (Dose B) or placebo Drug: WVE-003
WVE-003 is a stereopure antisense oligonucleotide (ASO)
Experimental: WVE-003 (Dose C) or placebo Drug: WVE-003
WVE-003 is a stereopure antisense oligonucleotide (ASO)
Experimental: WVE-003 (Dose D) or placebo Drug: WVE-003
WVE-003 is a stereopure antisense oligonucleotide (ASO)


Outcome Measures
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Primary Outcome Measures :
  1. Safety: Proportion of patients with adverse events (AEs) [ Time Frame: Day 1 through end of study (minimum of 36 weeks) ]

Secondary Outcome Measures :
  1. Maximum concentration (Cmax) of WVE-003 in plasma [ Time Frame: Day 1 through end of study (minimum of 36 weeks) ]
  2. Area under the plasma concentration time curve for WVE-003 from time 0 to last quantifiable concentration (AUC0-t) [ Time Frame: Day 1 through end of study (minimum of 36 weeks) ]
  3. Terminal half-life (t1/2) of WVE-003 in cerebrospinal fluid (CSF) [ Time Frame: Day 1 through end of study (minimum of 36 weeks) ]

Eligibility Criteria
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Ages Eligible for Study:   25 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05032196


Contacts
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Contact: Clinical Operations 855-215-4687 clinicaltrials@wavelifesci.com

Locations
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Sponsors and Collaborators
Wave Life Sciences Ltd.
Investigators
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Study Director: Medical Director, MD Wave Life Sciences
More Information
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Responsible Party: Wave Life Sciences Ltd.
ClinicalTrials.gov Identifier: NCT05032196    
Other Study ID Numbers: WVE-003-001
First Posted: September 2, 2021    Key Record Dates
Last Update Posted: January 6, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Chorea
Dyskinesias
 Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Neurocognitive Disorders
Mental Disorders