ALN-HTT02-001
Recruiting
This is a Phase 1b. Randomized, double-blind, placebo-controlled study. Looking at the safety, tolerability PK and PD of a new drug, ALN-HTT02 in adult HD patients.
Placebo-controlled means that one of the groups will be given a substance with no active ingredients (also known as a ‘placebo’). It looks like the drug being tested but does not contain any real medicine. Comparing results from the different groups helps the researchers know whether any changes seen result from the drug or occur by chance.
Double-blinded means that neither the participant nor the clinical trial doctor can choose or know the group the participant is in until the trial is over. This approach helps to prevent bias.
The drug or placebo will be delivered by lumbar puncture(injected into the spine). It will aim to lower both the normal and mutant allele.
Up to 54 participants will be recruited. The duration of the trial will be 12 months.
For contact clinicaltrials@alnylam.com
Ages Eligible
for Study:
25 Years to 70 Years (Adult, Older Adult )
Sexes Eligible
for Study:
All
Accepts Healthy Volunteers:
No
- 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 <45 mL/min/1.73m^2 at screening
- Has received an investigational agent within the last 1 year or 5 half-lives (if known)