OLE study of WVE-120101 in Huntington's Disease
Research type
Research Study
Full title
A Multicenter, Open-label Extension Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-120101 in Patients with Huntington's Disease
IRAS ID
277466
Contact name
Stuart Ritchie
Contact email
Sponsor organisation
WAVE Life Sciences Ltd
Eudract number
2019-003637-42
Duration of Study in the UK
2 years, 8 months, 0 days
Research summary
There is no cure for HD and it is invariably fatal. Some symptoms can be managed with existing medications, but none of these medicines can slow or reverse the disease. HD is caused by known mutations on a single gene, called the Huntingtin gene. All people carry the Huntingtin gene, which makes Huntingtin protein that is important for normal brain development.
The mutation on the HTT gene in people with HD leads to production of another type of protein known as the mutant Huntingtin (mHTT) protein. Study drug WVE-120101 is designed to selectively reduce mHTT protein while leaving normal Huntington protein intact.
Research suggests that selectively lowering mHTT protein has the potential to slow the progression of HD. This is an open label extension of the first clinical study conducted with WVE-120101. The purpose of this OLE is to test the safety, tolerability and clinical effects of WVE-120101 in patients with early HD.50 eligible patients who completed the WVE-HDSNP1-001 study will be enrolled into 1 of 3 dose groups (4, 8 and 16mg) dependent on dose received in WVE-HDSNP1-001 study. WVE-120101 will be administered every 4 weeks for up to 97 weeks. The patients will be monitored for safety every 4 weeks and at week 101. As results from WVE-HDSNP1-001 become available the dose may change if higher doses are found to be acceptable.
Study procedures will include analysis of blood, urine, and spinal fluid, vital signs, ECGs, physical exams, MRI, and HD clinical assessments.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
20/LO/0271
Date of REC Opinion
27 Mar 2020
REC opinion
Unfavourable Opinion