NCT06585449

Brief Summary

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

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
66

participants targeted

Target at P75+ for phase_1

Timeline
26mo left

Started Oct 2024

Typical duration for phase_1

Geographic Reach
3 countries

19 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress42%
Oct 2024Jul 2028

First Submitted

Initial submission to the registry

September 3, 2024

Completed
2 days until next milestone

First Posted

Study publicly available on registry

September 5, 2024

Completed
1 month until next milestone

Study Start

First participant enrolled

October 14, 2024

Completed
3.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 5, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 5, 2028

Last Updated

May 1, 2026

Status Verified

April 1, 2026

Enrollment Period

3.7 years

First QC Date

September 3, 2024

Last Update Submit

April 29, 2026

Conditions

Huntington's Disease

Keywords

siRNAALN-HTT02IntrathecalHD-ISS Stage 2HD-ISS early Stage 3Neurodegenerative disorder

Outcome Measures

Primary Outcomes (3)

  • 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

  • Frequency of AEs in the OLE Part of the Study

    Up to 36 months

Secondary Outcomes (4)

  • 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 ; Baseline up to Month 36 in the OLE 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; Up to Month 36 in the OLE Part of the study

  • 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 ; Up to Month 36 in the OLE 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; Up to Month 36 in the OLE Part of the study

Study Arms (2)

ALN-HTT02

EXPERIMENTAL

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. Participants have the option to receive repeat doses of ALN-HTT02 in the Open-label Extension (OLE) Part of the study.

Drug: ALN-HTT02

Placebo + ALN-HTT02

PLACEBO COMPARATOR

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. Participants have the option to receive repeat doses of ALN-HTT02 in the OLE Part of the study.

Drug: ALN-HTT02Drug: Placebo

Interventions

ALN-HTT02DRUG

ALN-HTT02 will be administered intrathecally

ALN-HTT02Placebo + ALN-HTT02
PlaceboDRUG

Placebo will be administered intrathecally

Placebo + ALN-HTT02

Eligibility Criteria

Age25 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Has stage 2 or early Stage 3 Huntington's disease (HD), per the Huntington's Disease Integrated Staging System (HD-ISS)

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (19)

Clinical Trial Site

Edmonton, T6G 2B7, Canada

RECRUITING

Clinical Trial Site

Montreal, H2W 1T8, Canada

RECRUITING

Clinical Trial Site

Ottawa, K1Y4E9, Canada

RECRUITING

Clinical Trial Site

Vancouver, V6T 2B5, Canada

RECRUITING

Clinical Trial Site

Berlin, 10117, Germany

NOT YET RECRUITING

Clinical Trial Site

Bochum, 44791, Germany

RECRUITING

Clinical Trial Site

Bonn, 53127, Germany

RECRUITING

Clinical Trial Site

Dresden, 01307, Germany

RECRUITING

Clinical Trial Site

Münster, 48149, Germany

RECRUITING

Clinical Trial Site

Taufkirchen, 84416, Germany

RECRUITING

Clinical Trial Site

Ulm, 89081, Germany

RECRUITING

Clinical Trial Site

Birmingham, B15 2TT, United Kingdom

RECRUITING

Clinical Trial Site

Cambridge, CB2 0PY, United Kingdom

RECRUITING

Clinical Trial Site

Cardiff, CF103AX, United Kingdom

RECRUITING

Clinical Trial Site

Glasgow, G51 4TF, United Kingdom

RECRUITING

Clinical Trial Site

Greater Manchester, M13 9WL, United Kingdom

RECRUITING

Clinical Trial Site

London, WC1N 3BG, United Kingdom

RECRUITING

Clinical Trial Site

Oxford, OX3 9DU, United Kingdom

RECRUITING

Clinical Trial Site

Plymouth, PL6 8DH, United Kingdom

RECRUITING

MeSH Terms

Conditions

Huntington DiseaseNeurodegenerative Diseases

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesDementiaChoreaDyskinesiasMovement DisordersHeredodegenerative Disorders, Nervous SystemGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCognition DisordersNeurocognitive DisordersMental Disorders

Study Officials

  • Medical Director

    Alnylam Pharmaceuticals

    STUDY DIRECTOR

Central Study Contacts

Alnylam Clinical Trial Information Line

CONTACT

1-877-ALNYLAMclinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

CONTACT

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

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 3, 2024

First Posted

September 5, 2024

Study Start

October 14, 2024

Primary Completion (Estimated)

July 5, 2028

Study Completion (Estimated)

July 5, 2028

Last Updated

May 1, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

Locations

GB(8)CA(4)DE(7)