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NCT Number: NCT06585449

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

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

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Key information

Conditions

Huntington's Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Chorea Cognition Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dementia Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Huntington Disease Mental Disorders Movement Disorders Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases

Age range

25 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Clinical Trial Site, Edmonton, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ALN-HTT02

Drug

ALN-HTT02 will be administered intrathecally

Placebo

Drug

Placebo will be administered intrathecally

Primary outcomes

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

    Time frame: Up to 12 months

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

    Time frame: Up to 12 months

  3. Frequency of AEs in the OLE Part of the Study

    Time frame: Up to 36 months

Secondary outcomes

  1. 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

  2. Concentrations of ALN-HTT02 in Plasma

    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

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

  3. 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

  4. Concentrations of ALN-HTT02 in Urine

    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

    Fractional Excretion of ALN-HTT02

Study contacts

Contact information is provided by the study sponsor or research team.

Alnylam Clinical Trial Information Line

CONTACT

[email protected]

1-877-ALNYLAM

Alnylam Clinical Trial Information Line

CONTACT

[email protected]

1-877-256-9526

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study 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

Important dates

Study start
2024
Primary completion
2028
Study completion
2028
First posted
Sep 5, 2024
Registry last updated
Jul 15, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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