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

Phase 1RecruitingNCT06585449

Alnylam Pharmaceuticals66 enrolled

Overview

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses 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 \<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

Locations (19)

Clinical Trial Site

Edmonton, Canada

RECRUITING

Clinical Trial Site

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

Frequency of AEs in the OLE Part of the Study

Time frame: Up to 36 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 ; Baseline up to Month 36 in the OLE 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; Up to Month 36 in the OLE 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 ; Up to Month 36 in the OLE 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; Up to Month 36 in the OLE 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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