Recruiting
Phase 1

ALN-HTT02

Sponsor:

Alnylam Pharmaceuticals

Code:

NCT06585449

Conditions

Huntington's Disease

Eligibility Criteria

Sex: All

Age: 25 - 70

Healthy Volunteers: Not accepted

Interventions

ALN-HTT02

Placebo

Study Details

Brief summary:

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses of ALN-HTT02.

Conditions

Huntington's Disease

Study ID

NCT06585449

Start date

Oct 14, 2024

Status verified date

Oct, 2026

Completion date

Jul 5, 2028

Anticipated

Primary completion date

Jul 5, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 25 - 70

Healthy Volunteers: Not accepted

Inclusion Criteria

  • Has stage 2 or early Stage 3 Huntington's disease (HD), per the Huntington's Disease Integrated Staging System (HD-ISS)

Exclusion Criteria

  • Has significant structural or degenerative neurologic disease other than Huntington's Disease (HD) at screening
  • Has primary or secondary immune compromise at screening due to infections, medical conditions, or chronic therapies
  • Has alanine aminotransferase or aspartate aminotransferase >2× upper limit of normal (ULN)
  • Has an estimated glomerular filtration rate (eGFR) of <60 mL/min/1.73m\^2 at screening
  • Has received an investigational agent within the last 1 year or 5 half-lives (if known)

Note: other protocol defined inclusion / exclusion criteria apply

Study Design

Enrollment

66 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: 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. Participants have the option to receive repeat doses of ALN-HTT02 in the Open-label Extension (OLE) Part of the study.

placebo comparator: 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. Participants have the option to receive repeat doses of ALN-HTT02 in the OLE Part of the study.

Interventions

ALN-HTT02

ALN-HTT02 will be administered intrathecally

Placebo

Placebo will be administered intrathecally

Primary outcome measure

  • 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 ]
  • Frequency of AEs in the OLE Part of the Study [ Time Frame: Up to 36 months ]

Central Contacts and Locations

Central contacts

Alnylam Clinical Trial Information Line

1-877-ALNYLAMclinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

1-877-256-9526clinicaltrials@alnylam.com

Locations

Clinical Trial Site

Recruiting

Edmonton, Canada, T6G 2B7

Clinical Trial Site

Recruiting

Montreal, Canada, H2W 1T8

Clinical Trial Site

Recruiting

Ottawa, Canada, K1Y4E9

Clinical Trial Site

Recruiting

Vancouver, Canada, V6T 2B5

More Information

Sponsor

Alnylam Pharmaceuticals

Last update posted

Oct 15, 2026

Last verified

Oct, 2026

Keywords

  • siRNA
  • ALN-HTT02
  • Intrathecal
  • HD-ISS Stage 2
  • HD-ISS early Stage 3
  • Neurodegenerative disorder

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-25. This information was provided to ClinicalTrials.gov by Alnylam Pharmaceuticals on 2026-10-15. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.