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

NCT06585449 · tracked via the Priya Life Science UK tracker

Sponsor

Alnylam Pharmaceuticals

Phase

Phase 1

Started

2024-10-14

Last updated

2026-09-16

Condition(s) studied

Huntington's Disease

Investigational drug(s) / intervention(s)

ALN-HTT02Placebo

ALN-HTT02: ALN-HTT02 will be administered intrathecally

Placebo: Placebo will be administered intrathecally

Study 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.

Eligibility

Sex

ALL

Min age

25 Years

Max age

70 Years

Healthy volunteers

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

Primary outcome measure(s)

Frequency of Adverse Events (AEs) in the Double-blind Part of the Study — Up to 12 months
Frequency of AEs in the Open-label Part of the Study — Up to 12 months
Frequency of AEs in the OLE Part of the Study — Up to 36 months

Trial sites (19)

Facility	City	Region	Status
Clinical Trial Site	Edmonton	Canada	Recruiting
Clinical Trial Site	Montreal	Canada	Recruiting
Clinical Trial Site	Ottawa	Canada	Recruiting
Clinical Trial Site	Vancouver	Canada	Recruiting
Clinical Trial Site	Berlin	Germany	Recruiting
Clinical Trial Site	Bochum	Germany	Recruiting
Clinical Trial Site	Bonn	Germany	Recruiting
Clinical Trial Site	Dresden	Germany	Recruiting
Clinical Trial Site	Münster	Germany	Recruiting
Clinical Trial Site	Taufkirchen	Germany	Recruiting
Clinical Trial Site	Ulm	Germany	Recruiting
Clinical Trial Site	Birmingham	United Kingdom	Recruiting
Clinical Trial Site	Cambridge	United Kingdom	Recruiting
Clinical Trial Site	Cardiff	United Kingdom	Recruiting
Clinical Trial Site	Glasgow	United Kingdom	Recruiting
Clinical Trial Site	Greater Manchester	United Kingdom	Recruiting
Clinical Trial Site	London	United Kingdom	Recruiting
Clinical Trial Site	Oxford	United Kingdom	Recruiting
Clinical Trial Site	Plymouth	United Kingdom	Recruiting

More Alnylam Pharmaceuticals trials in the UK

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TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy Recruiting

A Study to Evaluate ALN-5288 in Patients With Alzheimer's Disease Recruiting

A Study to Evaluate ALN-4915 in Adult Healthy Volunteers Recruiting

A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy Enrolling by invitation

Other trials for the same condition

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease Active, not recruiting

A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06585449 on ClinicalTrials.gov ↗ ← All trials in the UK