Overview

Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease

Status:
Terminated
Trial end date:
2021-05-11
Target enrollment:
0
Participant gender:
All
Summary
PRECISION-HD1 is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of WVE-120101 in adult patients with early manifest Huntington's disease (HD) who carry a targeted single nucleotide polymorphism (SNP) rs362307 (SNP1).
Phase:
Phase 1/Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Wave Life Sciences Ltd.
Criteria
Key Inclusion Criteria:

- Prescreened with targeted SNP on the same allele as the pathogenic CAG expansion

- Ambulatory, male or female patients aged ≥25 - ≤65 years

- Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease
Rating Scale (UHDRS) Diagnostic Confidence Score = 4

- Early manifest HD, Stage I or Stage II based on UHDRS Total Functional Capacity Scores
≥7 and ≤13

Key Exclusion Criteria:

- Malignancy or received treatment for malignancy, other than treated basal cell or
squamous cell carcinoma of the skin, within the previous 5 years.

- Received investigational drug or implantable device in prior 3 months or
investigational oligonucleotide in prior 6 months or 5 half-lives of the
oligonucleotide, whichever is longer

- Clinically significant medical condition, unstable psychiatric symptoms, substance
abuse, or pregnancy

- Inability to undergo brain MRI

- Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or
unsuccessful lumbar puncture