Clinical trial NCT06585449: A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Huntington's Disease
Keywords: siRNA
ALN-HTT02
Intrathecal
HD-ISS Stage 2
HD-ISS early Stage 3
Neurodegenerative disorder
Lead sponsor: Alnylam Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2024-09-05
Enrollment: 54
Sex: ALL
Minimum age (years): 25
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of a single dose of ALN-HTT02.


Eligibility criteria: 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 \<45 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

Primary outcomes:
measure: Frequency of Adverse Events (AEs) in the Double-blind Part of the Study
timeFrame: Up to 12 months
measure: Frequency of AEs in the Open-label Part of the Study
timeFrame: Up to 12 months
Secondary outcomes:
measure: Change from Baseline in Levels of Mutant Huntingtin (mHTT) in Cerebrospinal Fluid (CSF)
timeFrame: Baseline up to Month 12 in the Double-blind Part of the study; Baseline up to Month 12 in the Open-label Part of the study
measure: Concentrations of ALN-HTT02 in Plasma
timeFrame: Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study
measure: Concentrations of ALN-HTT02 in Cerebrospinal Fluid (CSF)
timeFrame: Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study
measure: Concentrations of ALN-HTT02 in Urine
timeFrame: Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study
Other outcomes:
Locations:
city: Edmonton
country: Canada
facility: Clinical Trial Site
city: Montreal
country: Canada
facility: Clinical Trial Site
city: Ottowa
country: Canada
facility: Clinical Trial Site
city: Vancouver
country: Canada
facility: Clinical Trial Site
city: Bayern
country: Germany
facility: Clinical Trial Site
city: Bochum
country: Germany
facility: Clinical Trial Site
city: Venusberg
country: Germany
facility: Clinical Trial Site
city: Birmingham
country: United Kingdom
facility: Clinical Trial Site
city: Cambridge
country: United Kingdom
facility: Clinical Trial Site
city: Cardiff
country: United Kingdom
facility: Clinical Trial Site
city: London
country: United Kingdom
facility: Clinical Trial Site
city: Oxford
country: United Kingdom
facility: Clinical Trial Site
Study officials:
affiliation: Alnylam Pharmaceuticals
name: Medical Director
role: STUDY_DIRECTOR
References: