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

NCT ID: NCT06585449

Last Updated: 2025-11-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

54 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-14

Study Completion Date

2028-07-05

Brief Summary

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

Detailed Description

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Conditions

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Huntington's Disease

Keywords

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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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

Group Type EXPERIMENTAL

ALN-HTT02

Intervention Type DRUG

ALN-HTT02 will be administered intrathecally

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.

Group Type PLACEBO_COMPARATOR

ALN-HTT02

Intervention Type DRUG

ALN-HTT02 will be administered intrathecally

Placebo

Intervention Type DRUG

Placebo will be administered intrathecally

Interventions

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ALN-HTT02

ALN-HTT02 will be administered intrathecally

Intervention Type DRUG

Placebo

Placebo will be administered intrathecally

Intervention Type DRUG

Eligibility Criteria

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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)
Minimum Eligible Age

25 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alnylam Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Alnylam Pharmaceuticals

Locations

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Clinical Trial Site

Edmonton, , Canada

Site Status RECRUITING

Clinical Trial Site

Montreal, , Canada

Site Status RECRUITING

Clinical Trial Site

Ottowa, , Canada

Site Status RECRUITING

Clinical Trial Site

Vancouver, , Canada

Site Status RECRUITING

Clinical Trial Site

Bayern, , Germany

Site Status RECRUITING

Clinical Trial Site

Berlin, , Germany

Site Status RECRUITING

Clinical Trial Site

Bochum, , Germany

Site Status RECRUITING

Clinical Trial Site

Bonn, , Germany

Site Status RECRUITING

Clinical Trial Site

Dresden, , Germany

Site Status RECRUITING

Clinical Trial Site

Ulm, , Germany

Site Status RECRUITING

Clinical Trial Site

Venusberg, , Germany

Site Status RECRUITING

Clinical Trial Site

Birmingham, , United Kingdom

Site Status RECRUITING

Clinical Trial Site

Cambridge, , United Kingdom

Site Status RECRUITING

Clinical Trial Site

Cardiff, , United Kingdom

Site Status RECRUITING

Clinical Trial Site

Glasgow, , United Kingdom

Site Status RECRUITING

Clinical Trial Site

London, , United Kingdom

Site Status RECRUITING

Clinical Trial Site

Manchester, , United Kingdom

Site Status RECRUITING

Clinical Trial Site

Oxford, , United Kingdom

Site Status RECRUITING

Clinical Trial Site

Plymouth, , United Kingdom

Site Status RECRUITING

Countries

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Canada Germany United Kingdom

Central Contacts

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Alnylam Clinical Trial Information Line

Role: CONTACT

Phone: 1-877-ALNYLAM

Email: [email protected]

Alnylam Clinical Trial Information Line

Role: CONTACT

Phone: 1-877-256-9526

Email: [email protected]

Other Identifiers

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ALN-HTT02-001

Identifier Type: -

Identifier Source: org_study_id