A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease

Alnylam Pharmaceuticals

Status and phase: Enrolling; Phase 1

Conditions: Huntington's Disease

Treatments: Drug: ALN-HTT02; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT06585449

ALN-HTT02-001

Details and patient eligibility

About

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

Enrollment: 54 estimated patients

Sex: All

Ages: 25 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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 <45 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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54 participants in 2 patient groups, including a placebo group

ALN-HTT02

Experimental group

Description:

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

Treatment:

Drug: ALN-HTT02

Placebo + ALN-HTT02

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo

Drug: ALN-HTT02