A Study to Evaluate ALN-HTT02 in Adult Patients with Huntington’s Disease

Phase 1

Recruiting

• Conditions: Huntington’s disease
• Interventions: Drug: ALN-HTT02; Drug: Placebo

• Registration Number: 2024-515732-68-00
• Lead Sponsor: Alnylam Pharmaceuticals Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Posted: 2024-12-23
• Last Update Posted: 2025-06-20

Recruitment & Eligibility

• Status: Ongoing, recruiting
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ALN-HTT02	ALN-HTT02	Participants will be administered a single dose of ALN-HTT02 during the Double-blind Part of the study. Participants who received ALN-HTT02 in the Double-blind part of the study will not receive an additional dose during the Open-label part of the study
Placebo + ALN-HTT02	ALN-HTT02	Participants will be administered a single dose of placebo during the Double-blind Part of the study. Participants will have the option to receive a single dose of ALN-HTT02 during the Open-Label Part of the study.
Placebo + ALN-HTT02	Placebo	Participants will be administered a single dose of placebo during the Double-blind Part of the study. Participants will have the option to receive a single dose of ALN-HTT02 during the Open-Label Part of the study.

Primary Outcome Measures

Name	Time	Method
Frequency of Adverse Events (AEs) in the Double-blind Part of the Study	Up to 12 months
Frequency of AEs in the Open-label Part of the Study	Up to 12 months

Secondary Outcome Measures

Name	Time	Method
Change from Baseline in Levels of Mutant Huntingtin (mHTT) in Cerebrospinal Fluid (CSF)	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	Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study	Area Under the Plasma Concentration-time Curve (AUC) Maximum Observed Plasma Concentration (Cmax) Time to Maximum Plasma Concentration (Tmax)
Concentrations of ALN-HTT02 in Cerebrospinal Fluid (CSF)	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	Up to Month 12 in the Double-blind Part of the study; Up to Month 12 in the Open-label Part of the study	Fractional Excretion of ALN-HTT02

Trial Locations

Locations (7)

George-Huntington-Institut GmbH; 🇩🇪 Muenster, Germany

Technische Universitaet Dresden; 🇩🇪 Dresden, Germany

Katholisches Klinikum Bochum gGmbH; 🇩🇪 Bochum, Germany

Kbo Isar-Amper-Klinikum Taufkirchen (Vils); 🇩🇪 Taufkirchen/Vils, Germany

Universitaetsklinikum Ulm AöR; 🇩🇪 Ulm, Germany

Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.; 🇩🇪 Bonn, Germany

Charite Universitaetsmedizin Berlin KöR; 🇩🇪 Berlin, Germany

George-Huntington-Institut GmbH

🇩🇪Muenster, Germany

Ralf Reilmann

Site contact

004925178878811

ralf.reilmann@ghi-muenster.de