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Trial record 1 of 1 for:    WVE-003-001
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Study of WVE-003 in Patients With Huntington's Disease

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ClinicalTrials.gov Identifier: NCT05032196
Recruitment Status : Recruiting
First Posted : September 2, 2021
Last Update Posted : December 14, 2021
Sponsor:
Information provided by (Responsible Party):
Wave Life Sciences Ltd.

Tracking Information
First Submitted Date  ICMJE August 18, 2021
First Posted Date  ICMJE September 2, 2021
Last Update Posted Date December 14, 2021
Actual Study Start Date  ICMJE September 6, 2021
Estimated Primary Completion Date December 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 26, 2021)
Safety: Proportion of patients with adverse events (AEs) [ Time Frame: Day 1 through end of study (minimum of 36 weeks) ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 26, 2021)
  • Maximum concentration (Cmax) of WVE-003 in plasma [ Time Frame: Day 1 through end of study (minimum of 36 weeks) ]
  • 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) ]
  • Terminal half-life (t1/2) of WVE-003 in cerebrospinal fluid (CSF) [ Time Frame: Day 1 through end of study (minimum of 36 weeks) ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of WVE-003 in Patients With Huntington's Disease
Official Title  ICMJE A Multicenter, Randomized, Double-blind, Placebo Controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington's Disease
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Huntington Disease
Intervention  ICMJE Drug: WVE-003
WVE-003 is a stereopure antisense oligonucleotide (ASO)
Study Arms  ICMJE
  • Experimental: WVE-003 (Dose A) or placebo
    Intervention: Drug: WVE-003
  • Experimental: WVE-003 (Dose B) or placebo
    Intervention: Drug: WVE-003
  • Experimental: WVE-003 (Dose C) or placebo
    Intervention: Drug: WVE-003
  • Experimental: WVE-003 (Dose D) or placebo
    Intervention: Drug: WVE-003
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 26, 2021)
36
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2022
Estimated Primary Completion Date December 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 25 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Operations 855-215-4687 clinicaltrials@wavelifesci.com
Listed Location Countries  ICMJE Australia,   Canada,   Denmark,   France,   Germany,   Poland,   Spain,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05032196
Other Study ID Numbers  ICMJE WVE-003-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Wave Life Sciences Ltd.
Study Sponsor  ICMJE Wave Life Sciences Ltd.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director, MD Wave Life Sciences
PRS Account Wave Life Sciences Ltd.
Verification Date December 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP