A Randomized, Double-blind, Placebo-controlled Study, to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered Single Ascending Doses of ALN-HTT02 in Adult Patients With Huntington's Disease
Overview
- Phase
- Phase 1
- Intervention
- ALN-HTT02
- Conditions
- Not specified
- Sponsor
- Alnylam Pharmaceuticals Inc.
- Enrollment
- 15
- Locations
- 1
- Primary Endpoint
- Frequency of Adverse Events (AEs) in the Double-blind Part of the Study
- Status
- Recruiting
- Last Updated
- 4 months ago
Overview
Brief Summary
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of a single dose of ALN-HTT02.
Investigators
Clinical Trial Information
Scientific
Alnylam Pharmaceuticals Inc.
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
ALN-HTT02
Participants will be administered a single dose of ALN-HTT02 during the Double-blind Part of the study. Participants who received ALN-HTT02 in the Double-blind part of the study will not receive an additional dose during the Open-label part of the study
Intervention: ALN-HTT02
Placebo + ALN-HTT02
Participants will be administered a single dose of placebo during the Double-blind Part of the study. Participants will have the option to receive a single dose of ALN-HTT02 during the Open-Label Part of the study.
Intervention: ALN-HTT02
Placebo + ALN-HTT02
Participants will be administered a single dose of placebo during the Double-blind Part of the study. Participants will have the option to receive a single dose of ALN-HTT02 during the Open-Label Part of the study.
Intervention: Placebo
Outcomes
Primary Outcomes
Frequency of Adverse Events (AEs) in the Double-blind Part of the Study
Time Frame: Up to 12 months
Frequency of AEs in the Open-label Part of the Study
Time Frame: Up to 12 months
Secondary Outcomes
- Change from Baseline in Levels of Mutant Huntingtin (mHTT) in Cerebrospinal Fluid (CSF)(Baseline up to Month 12 in the Double-blind Part of the study; Baseline up to Month 12 in the Open-label Part of the study)
- Concentrations of ALN-HTT02 in Plasma(Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study)
- Concentrations of ALN-HTT02 in Cerebrospinal Fluid (CSF)(Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study)
- Concentrations of ALN-HTT02 in Urine(Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study)