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
226655
Contact name
Stuart Ritchie
Contact email
Sponsor organisation
WAVE Life Sciences Ltd
Eudract number
2016-005059-10
Duration of Study in the UK
1 years, 7 months, 7 days
Research summary
Huntington’s Disease (HD) is a rare inherited neurological disease that results in degeneration of the brain cells, causing cognitive and psychiatric disabilities in addition to a progressive loss of coordination with involuntary\nmovements.\n\nThere 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.\nHD 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.\nStudy drug WVE-120101 acts by selectively reducing mHtt protein. The Sponsor of the study thinks that if you can lower the amount of mHtt protein, you may be able to treat HD.\nThis 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.\nForty-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 up for 8 weeks.\nFollowing a safety review, 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 over 8 weeks, and patients will be followed for 14 weeks after the last dose.\nStudy procedures will include analysis of blood, urine, and spinal fluid, vital signs, ECGs, physical exams, MRI, and HD disease scale assessments.\n\nThe study is sponsored by WAVE Life Sciences Ltd.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
17/NE/0186
Date of REC Opinion
27 Jun 2017
REC opinion
Unfavourable Opinion