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