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Looking for participantsPhase1

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

Sponsor: Alnylam Pharmaceuticals

NCT ID: NCT06585449

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
ALN-HTT02 (drug), Placebo (drug)
How long the study runs
Study runs about 45 months (dates as stated)
About the drug or intervention
ALN-HTT02 — drug: ALN-HTT02 will be administered intrathecally · Placebo — drug: Placebo will be administered intrathecally
Patient visit burden
Not specified by the sponsor
Type of study
Testing a treatment
Ages
25 Years to 70 Years
Who
All
Number of participants
66
Started
2024-10-14
Last checked
2026-09

Plain English Summary

What is this study?

  • • Testing a new treatment for huntington's disease
  • • Phase1 - 66 participants
  • • The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses of ALN-HTT02

Who can take part?

  • • Ages 25 Years to 70 Years
  • • Diagnosed with huntington's disease

Where?

  • • Birmingham - Clinical Trial Site
  • • Cambridge - Clinical Trial Site
  • • Cardiff - Clinical Trial Site
  • • Glasgow - Clinical Trial Site
  • • +4 more UK sites

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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

Huntington's Disease

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 25 Years - 70 Years
  • Who can join: All genders

Biomarkers mentioned

eGFR

What the study is looking for

  • ✓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
See the full 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 Is This Study? (8 UK sites)

Clinical Trial Site

Birmingham B15 2TT, United Kingdom

Recruiting

Clinical Trial Site

Cambridge CB2 0PY, United Kingdom

Recruiting

Clinical Trial Site

Cardiff CF103AX, United Kingdom

Recruiting

Clinical Trial Site

Glasgow G51 4TF, United Kingdom

Recruiting

Clinical Trial Site

Greater Manchester M13 9WL, United Kingdom

Recruiting

Clinical Trial Site

London WC1N 3BG, United Kingdom

Recruiting

Clinical Trial Site

Oxford OX3 9DU, United Kingdom

Recruiting

Clinical Trial Site

Plymouth PL6 8DH, United Kingdom

Recruiting

How to Get in Touch

Alnylam Clinical Trial Information Line

Sponsor contact

CONTACT

1-877-ALNYLAM clinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

Sponsor contact

CONTACT

1-877-256-9526 clinicaltrials@alnylam.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-09