RecruitingPhase 1NCT06585449

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


Sponsor

Alnylam Pharmaceuticals

Enrollment

66 participants

Start Date

Oct 14, 2024

Study Type

INTERVENTIONAL

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

Min Age: 25 YearsMax Age: 70 Years

Inclusion Criteria1

  • Has stage 2 or early Stage 3 Huntington's disease (HD), per the Huntington's Disease Integrated Staging System (HD-ISS)

Exclusion Criteria5

  • 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)

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Interventions

DRUGALN-HTT02

ALN-HTT02 will be administered intrathecally

DRUGPlacebo

Placebo will be administered intrathecally


Locations(19)

Clinical Trial Site

Edmonton, Canada

Clinical Trial Site

Montreal, Canada

Clinical Trial Site

Ottawa, Canada

Clinical Trial Site

Vancouver, Canada

Clinical Trial Site

Berlin, Germany

Clinical Trial Site

Bochum, Germany

Clinical Trial Site

Bonn, Germany

Clinical Trial Site

Dresden, Germany

Clinical Trial Site

Münster, Germany

Clinical Trial Site

Taufkirchen, Germany

Clinical Trial Site

Ulm, Germany

Clinical Trial Site

Birmingham, United Kingdom

Clinical Trial Site

Cambridge, United Kingdom

Clinical Trial Site

Cardiff, United Kingdom

Clinical Trial Site

Glasgow, United Kingdom

Clinical Trial Site

Greater Manchester, United Kingdom

Clinical Trial Site

London, United Kingdom

Clinical Trial Site

Oxford, United Kingdom

Clinical Trial Site

Plymouth, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT06585449