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

• Register: ClinicalTrials.gov
• Last refreshed on: 21 February 2022
• Main ID: NCT03225833
• Date of registration: 17/07/2017
• Prospective Registration: Yes
• Primary sponsor: Wave Life Sciences Ltd.
• Public title: Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease PRECISION-HD1
• Scientific title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-120101 Administered Intrathecally in Patients With Huntington's Disease
• Date of first enrolment: July 17, 2017
• Target sample size: 61
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03225833
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 1/Phase 2

Countries of recruitment

Australia, Canada, Denmark, France, Germany, Poland, United Kingdom

Contacts

• Name: Medical Director, MD
• Affiliation: Wave Life Sciences

Key inclusion & exclusion 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



Age minimum: 25 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Huntington's Disease

Intervention(s)

Drug: Placebo

Drug: WVE-120101

Primary Outcome(s)

Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs [Time Frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])]

Safety: Severity of Adverse Events [Time Frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])]

Safety: Number of Patients With Serious TEAEs [Time Frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])]

Safety: Number of Patients With Treatment-emergent Adverse Events (TEAEs) [Time Frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])]

Secondary Outcome(s)

Clinical Effects: Total Functional Capacity (TFC) [Time Frame: Day 1 to last observation - up to Day 140 (32 mg cohort) or Day 196 (all other cohorts)]

PK: Area Under the Plasma Concentration-time Curve (AUClast) [Time Frame: Patients participating in Period 1 (SAD) had PK samples collected on Day 1 predose through 24-48 hours postdose. Patients participating in Period 2 (MAD) had PK samples collected predose on Day 112 and through 4 hours postdose.]

PK: Time of Occurrence of Cmax (Tmax) [Time Frame: Patients participating in Period 1 (SAD) had PK samples collected on Day 1 predose through 24-48 hours postdose. Patients participating in Period 2 (MAD) had PK samples collected predose on Day 112 and through 4 hours postdose.]

Pharmacokinetics (PK): Maximum Observed Concentration (Cmax) [Time Frame: Patients participating in Period 1 (SAD) had PK samples collected on Day 1 predose through 24-48 hours postdose. Patients participating in Period 2 (MAD) had PK samples collected predose on Day 112 and through 4 hours postdose.]

PK: Terminal Elimination Half Life [Time Frame: Patients participating in Period 1 (SAD) had PK samples collected on Day 1 predose through 24-48 hours postdose. Patients participating in Period 2 (MAD) had PK samples collected predose on Day 112 and through 4 hours postdose.]

Pharmacodynamics [Time Frame: Day 1 to last observation - up to Day 140 (32 mg cohort) or Day 196 (all other cohorts)]

Secondary ID(s)

WVE-HDSNP1-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 10/02/2022
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03225833