Phase 1b/2a study of WVE-120101 in patients with Huntington's Disease
Research type
Research Study
Full title
A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-120101 Administered Intrathecally in Patients with Huntington’s Disease
IRAS ID
231809
Contact name
Stuart Ritchie
Contact email
Sponsor organisation
WAVE Life Sciences Ltd
Eudract number
2016-005095-10
Duration of Study in the UK
1 years, 7 months, 7 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 the first clinical study conducted with WVE-120101.The purpose of this double-blind study is to test the safety and tolerability of single and multiple doses of WVE-120101 in patients with early HD.
Forty-eight eligible patients will be enrolled into 1 of 4 dose cohorts (2, 4, 8 and 16 mg). In each cohort, 12 patients (9 active and 3 placebo) will receive a single dose of WVE-120101 or placebo and be followed for at least 8 weeks before any additional dosing may occur.
Following safety review of each single dose cohort, patients may receive 3 additional doses of study drug or placebo at the same dose they received before. These 3 doses will be administered once every 4 weeks, and patients will be followed for 14 weeks after the last dose. 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
17/LO/1269
Date of REC Opinion
1 Sep 2017
REC opinion
Unfavourable Opinion