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

Phase 1RecruitingNCT06585449

Alnylam Pharmaceuticals54 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Huntington's Disease

Interventions

ALN-HTT02DRUG

ALN-HTT02 will be administered intrathecally

PlaceboDRUG

Placebo will be administered intrathecally

Eligibility

Sex: ALLMin age: 25 YearsMax age: 70 Years

Medical Language ↔ 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 \<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

Locations (19)

Clinical Trial Site

Edmonton, Alberta, Canada

RECRUITING

Outcomes

Primary Outcomes

Frequency of Adverse Events (AEs) in the Double-blind Part of the Study

Time frame: Up to 12 months

Frequency of AEs in the Open-label Part of the Study

Time frame: Up to 12 months

Secondary Outcomes

Change from Baseline in Levels of Mutant Huntingtin (mHTT) in Cerebrospinal Fluid (CSF)

Time frame: 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

Concentrations of ALN-HTT02 in Plasma

Area Under the Plasma Concentration-time Curve (AUC) Maximum Observed Plasma Concentration (Cmax) Time to Maximum Plasma Concentration (Tmax)

Time frame: Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study

Concentrations of ALN-HTT02 in Cerebrospinal Fluid (CSF)

Time frame: Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study

Concentrations of ALN-HTT02 in Urine

Fractional Excretion of ALN-HTT02

Time frame: Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study

Central Contacts

Alnylam Clinical Trial Information Line

CONTACT

1-877-ALNYLAM clinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

CONTACT

1-877-256-9526 clinicaltrials@alnylam.com

Data from ClinicalTrials.gov

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