Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease
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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
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Huntington Disease | Drug: WVE-120101 | Phase 1 Phase 2 |
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 |

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

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

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

Study Director: | Medical Director, MD | Wave Life Sciences |
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 |
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 |
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 |