NCT06585449RecruitingPhase 1
A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease
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
Source: ClinicalTrials.gov · Last updated 15 Oct 2026
Brief summary (registry, in English)
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses of ALN-HTT02.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 66 (estimated)
- First submitted
- 3 Sept 2024
- Start date
- 14 Oct 2024
- Primary completion date
- 5 Jul 2028
- Lead sponsor
- Alnylam Pharmaceuticals
- Diseases
- Huntington's disease
- NCT number
- NCT06585449
What you commit to
- Study type: Interventional · Phase 1
- Planned participants: 66
- From 14 Oct 2024 to the primary completion date, 5 Jul 2028
- 19 sites in 3 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Has received an investigational agent within the last 1 year or 5 half-lives (if known)
- Note: other protocol defined inclusion / exclusion criteria apply
Other illness
- 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
Lab values
- 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
Who the study is looking for
- Has stage 2 or early Stage 3 Huntington's disease (HD), per the Huntington's Disease Integrated Staging System (HD-ISS)
Age in the registry: 25 years to 70 years · Sex: All · Does not accept healthy volunteers
Sites (19)
Sites in registry order. Pick your city on the disease page to sort them by distance.
This page is information, not medical advice. Discuss any trial with your doctor.