Home › Trials › NCT06585449

Evaluating ALN-HTT02 for treating Huntington's disease in adults

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

PHASE1 · Alnylam Pharmaceuticals · NCT06585449

This study is testing a new drug called ALN-HTT02 to see if it is safe and effective for adults with early-stage Huntington's disease.

Quick facts

PhasePHASE1
Study typeInterventional
Enrollment66 (estimated)
Ages25 Years to 70 Years
SexAll
SponsorAlnylam Pharmaceuticals (industry)
Locations19 sites (Edmonton and 18 other locations)
Trial IDNCT06585449 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety, tolerability, pharmacodynamics, and pharmacokinetics of a single dose of ALN-HTT02 in adult patients diagnosed with stage 2 or early stage 3 Huntington's disease. Participants will receive either the investigational drug or a placebo to determine its effects. The study will closely monitor participants for any adverse reactions and measure how the drug is processed in the body. The findings could provide insights into the potential of ALN-HTT02 as a treatment option for Huntington's disease.

Who should consider this trial

Good fit: Ideal candidates for this study are adults with stage 2 or early stage 3 Huntington's disease.

Not a fit: Patients with significant neurological diseases other than Huntington's or those with severe immune compromise may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could offer a new therapeutic option for patients with Huntington's disease, potentially slowing disease progression.

How similar studies have performed: While this approach using siRNA is innovative, similar studies targeting genetic mechanisms in neurodegenerative diseases have shown promise.

Eligibility criteria

Show full inclusion / 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 \<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

Where this trial is running

Edmonton and 18 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Huntington's Disease, siRNA, ALN-HTT02, Intrathecal, HD-ISS Stage 2, HD-ISS early Stage 3, Neurodegenerative disorder

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.