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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 : November 1, 2021
Sponsor:
Information provided by (Responsible Party):
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.

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

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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
Actual Study Start Date : September 6, 2021
Estimated Primary Completion Date : December 2022
Estimated Study Completion Date : December 2022

Resource links provided by the National Library of Medicine


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)




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) ]


Information from the National Library of Medicine

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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

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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Australia, Victoria
Monash Health Recruiting
Clayton, Victoria, Australia, 3168
Principal Investigator: Yenni Lie, MD         
Royal Melbourne Hospital Recruiting
Melbourne, Victoria, Australia, 3050
Principal Investigator: Dennis Velakoulis, MBBS MMed DMedSci         
Australia, Western Australia
North Metropolitan Health Service Recruiting
Perth, Western Australia, Australia, 6910
Principal Investigator: Carolyn Orr, MD         
Canada, Quebec
Centre Hospitalier de l-Universite de Montreal Recruiting
Montréal, Quebec, Canada, H2X019
Principal Investigator: Sylvain Chouinard, MD         
Denmark
Rigshospitalet Recruiting
Copenhagen, Denmark, 2100
Principal Investigator: Lena Hjermind, MD         
France
Institut du Cerveau et de la Moelle Epiniere Recruiting
Paris, France, 75646
Principal Investigator: Alexandra Durr, MD, PhD         
Germany
Katholisches Klinikum Bochum gGmbH Recruiting
Bochum, Germany, 44791
Principal Investigator: Carsten Saft, MD         
George-Huntington-Institut GmbH Recruiting
Muenster, Germany, 48149
Principal Investigator: Ralf Reilman, MD         
kbo-Isar-Amper-Klinikum Taufkirchen (Vils) Recruiting
Taufkirchen, Germany, 84416
Principal Investigator: Alzbeta Mühlbäck, MD         
Poland
Szpital Sw. Wojciecha Recruiting
Gdańsk, Poland, 80-462
Principal Investigator: Jaroslaw Slawek, MD         
Instytut Psychiatrii I Neurologii Recruiting
Warsaw, Poland, 02-957
Principal Investigator: Grzegorz Witkowski, MD, PhD         
United Kingdom
Royal Devon and Exeter Hospital NHS Trust Recruiting
Exeter, Devon, United Kingdom, EX2 5DW
Principal Investigator: Timothy P Harrower, PhD         
Cardiff University, Schools of Medicine and Biosciences Recruiting
Cardiff, Wales, United Kingdom, CF14 4XW
Principal Investigator: Anne Rosser, PhD, MBBChir, MRCP         
Royal Liverpool University Hospital Recruiting
Liverpool, United Kingdom, L7 8XP
Principal Investigator: Richard Rhys Davies, MA BMBCh PhD FRCP         
Sponsors and Collaborators
Wave Life Sciences Ltd.
Investigators
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Study Director: Medical Director, MD Wave Life Sciences
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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: November 1, 2021
Last Verified: October 2021
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