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

Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age25-70
SponsorAlnylam Pharmaceuticals

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.

Eligibility criteria

Qualifiers

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

Disqualifiers

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/1.73m² at screening

Trial design

Treatments tested in this trial

  • ALN-HTT02
  • Placebo

Treatment groups

66 Participants
are divided into 2 treatment groups

Sponsors and collaborators