A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease
ConditionHuntington's Disease
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age25-70
SponsorAlnylam Pharmaceuticals
The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses of ALN-HTT02.
Has stage 2 or early Stage 3 Huntington's disease (HD), per the Huntington's Disease Integrated Staging System (HD-ISS)
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