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Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04617847
Recruitment Status : Enrolling by invitation
First Posted : November 5, 2020
Last Update Posted : November 9, 2020
Sponsor:
Information provided by (Responsible Party):
Wave Life Sciences Ltd.

Study Description
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Brief Summary:
WVE-HDSNP1-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120101 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism, rs362307 (SNP1). To participate in the study, patients must have completed the Phase 1b/2a clinical study WVE-HDSNP1-001.

Condition or disease Intervention/treatment Phase
Huntington Disease Drug: WVE-120101 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Open-label Extension Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-120101 in Patients With Huntington's Disease
Actual Study Start Date : April 13, 2020
Estimated Primary Completion Date : April 2023
Estimated Study Completion Date : April 2023

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Arms and Interventions
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Arm Intervention/treatment
Experimental: WVE-120101 (Dose A) Drug: WVE-120101
WVE-120101 is a stereopure antisense oligonucleotide (ASO). It is administered monthly via intrathecal injection.


Outcome Measures
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Primary Outcome Measures :
  1. Safety: Number of patients with adverse events (AEs) [ Time Frame: Day 1 to Week 101 (end of study) ]
  2. Safety: Number of patients with a severe AE [ Time Frame: Day 1 to Week 101 (end of study) ]
  3. Safety: Number of patients with serious AEs (SAEs) [ Time Frame: Day 1 to Week 101 (end of study) ]
  4. Safety and Tolerability: Number of patients who withdraw due to AEs [ Time Frame: Day 1 to Week 101 (end of study) ]

Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • 1. Patient successfully completed the Phase 1b/2a study with WVE-120101, WVE-HDSNP1-001.

Key Exclusion Criteria:

  • 1. Received an investigational drug other than WVE-120101, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer.
  • 2. Inability to undergo brain MRI (with or without sedation).
  • 3. Clinically significant medical finding on the physical examination other than HD that, in the judgment of the Investigator, will make the patient unsuitable for participation in and/or completion of the study procedures.
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): NCT04617847


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: NCT04617847    
Other Study ID Numbers: WVE-HDSNP1-002
First Posted: November 5, 2020    Key Record Dates
Last Update Posted: November 9, 2020
Last Verified: November 2020
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