RECRUITINGPhase 1INTERVENTIONAL
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
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.
Who May Be Eligible (Plain English)
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
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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
Treatments Being Tested
DRUG
ALN-HTT02
ALN-HTT02 will be administered intrathecally
DRUG
Placebo
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