Tests ALN-HTT02 in adults with Huntington's disease
Tests a drug given into the spinal fluid in adults with Huntington's disease.
Key facts
- Who runs it
- Alnylam Pharmaceuticals
- Where
- Birmingham, Cambridge, Cardiff, Glasgow, Greater Manchester, London, Oxford, and Plymouth, United KingdomSee the 8 sitesAlso in 2 other countries, 11 sites
- Trial code
- Registry title
- A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease
- Status
- Recruiting
- Phase
- Phase 1
- People taking part
- 66 people, planned
- Started
- 14 Oct 2024
- Expected to end
- 5 Jul 2028
- Type of study
- Interventional, 2 groupsOne group gets a placebo (a dummy treatment), for comparison.
- Treatments tested
- ALN-HTT02
- Placebo
- Record updated
- 15 October 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with Huntington's disease at stage 2 or early stage 3. It tests the safety and effects of ALN-HTT02.
- Age
- 25 Years to 70 Years
- Sex
- All sexes can join.
- Disease stage
- Stage 2 or early Stage 3 Huntington's disease, by the Huntington's Disease Integrated Staging System (HD-ISS).
- Earlier treatment
- Cannot have received an investigational agent within the last 1 year or 5 half-lives (if known).
Main reasons for not taking part
- Significant structural or degenerative brain or nerve disease other than Huntington's disease
- Weakened immune system from infections, medical conditions, or long-term therapies
- Alanine aminotransferase or aspartate aminotransferase above 2× the upper limit of normal
- Estimated glomerular filtration rate (eGFR) below 60 mL/min/1.73m^2
- Investigational agent received within the last 1 year or 5 half-lives (if known)
The registry criteria, word for word
Show all 6 criteriaShow less
Who can take part
- Has stage 2 or early Stage 3 Huntington's disease (HD), per the Huntington's Disease Integrated Staging System (HD-ISS)
Who cannot take part
- Has received an investigational agent within the last 1 year or 5 half-lives (if known); Note: other protocol defined inclusion / exclusion criteria apply
- 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
Check if you might fit
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 25 and 70 years old?
Where in United Kingdom
Also in 2 other countries, 11 sites
- BirminghamHospital not named in the registryTaking people
- CambridgeHospital not named in the registryTaking people
- Cardiff, WalesHospital not named in the registryTaking people
- GlasgowHospital not named in the registryTaking people
- Greater ManchesterHospital not named in the registryTaking people
- LondonHospital not named in the registryTaking people
- Oxford, OxfordshireHospital not named in the registryTaking people
- PlymouthHospital not named in the registryTaking people
About the trial
What the trial wants to find out
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses of ALN-HTT02.
The 2 groups in the trial
- ALN-HTT02Treatment being tested
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. Participants have the option to receive repeat doses of ALN-HTT02 in the Open-label Extension (OLE) Part of the study.
- Placebo + ALN-HTT02Placebo, for comparison
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. Participants have the option to receive repeat doses of ALN-HTT02 in the OLE Part of the study.
Treatment: ALN-HTT02 and Placebo
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- 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
- Plain-language title
- Tests ALN-HTT02 in adults with Huntington's diseaseGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Fit questions
- Built from NCT06585449, who can take part
- Record updated
- 15 October 2026