Study of WVE-003 in Patients With Huntington's Disease

Wave Life Sciences USA, Inc.47 enrolled

Overview

This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of WVE-003 in adult patients with early-manifest HD who carry the targeted single nucleotide polymorphism (SNP) - SNP3.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Huntington Disease

Interventions

SAD: 30mg WVE-003DRUG

Single ascending dose of 30mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)

SAD: 60mg WVE-003DRUG

Single ascending dose of 60mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)

SAD: 90mg WVE-003DRUG

Single ascending dose of 90mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)

Eligibility

Sex: ALLMin age: 25 YearsMax age: 60 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion 2. Ambulatory, male or female patients aged ≥25 to ≤60 years 3. Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4 4. UHDRS Total Functional Capacity Scores ≥9 and ≤13 Exclusion Criteria: 1. Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years 2. Received any other study drug, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer, with the exception of the following: a. Received WVE-120101 or WVE-120102 within the last 3 months 3. Implantable CNS device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan 4. Inability to undergo brain MRI (with or without sedation) 5. Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture 6. Previously received tominersen

Locations (23)

Westmead Hospital

Westmead, New South Wales, Australia

Outcomes

Primary Outcomes

Safety: Proportion of Patients With Treatment Emergent Adverse Events (TEAEs) Related to Study Drug

The primary outcome for this study was safety and is reported as the proportion of patients with TEAEs related to study drug.

Time frame: Day 1 through Week 24 (single ascending dose Period 1); Day 1 through Week 28 (multi dose Period 2)

Secondary Outcomes

Pharmacokinetics of WVE-003 in Plasma

Parameter analyzed: AUC0-6 = area under the concentration-time curve from time 0 to 6 hrs

Time frame: Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2)

Pharmacokinetics of WVE-003 in Plasma

Parameter analyzed: Cmax = maximum observed concentration.

Time frame: Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2)

Concentration of WVE-003 in Cerebrospinal Fluid (CSF)

WVE-003 concentration in cerebrospinal fluid (CSF) is reported in ng/mL.

Time frame: 28 days post-dose during Period 1 (P1:Day29); 28 days post last dose during Period 2 (P2: Day141)

Data from ClinicalTrials.gov

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