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NCT Number: NCT07536061

A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease

This is a first-in-human, multi-center trial studying the effects of SRP-1005 in participants with Huntington's disease (HD).

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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

21 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Pacific Clinical Research Network - Auckland (PCRN Auckland), Auckland, New Zealand

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Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • Genetically confirmed diagnosis of huntingtin (HTT) cytosine-adenine-guanine repeat length ≥40.
  • Participant has HD Integrated Staging System (HD-ISS) Stage 2 or Mild Stage 3. At screening, the participant must be classified with the Enroll-HD HD-ISS Modified Calculator as either Stage 2 or Mild Stage 3.
  • Participants who are of childbearing potential, or with partners of childbearing potential, who are sexually active must agree to use a highly effective method of contraception throughout study participation, and for at least 90 days following the end of study.

Key Exclusion Criteria:

  • Any condition that would compromise the safety or feasibility of lumbar puncture or magnetic resonance imaging (MRI).
  • Presence of other significant neurological or systemic illnesses.
  • Current, chronic or active human immunodeficiency virus, hepatitis B/C.
  • Recent use of investigational agents or HTT-lowering therapies.
  • Uncontrolled psychiatric illness or substance use disorders.
  • Pregnancy or lactation.

Note: Other inclusion/exclusion criteria apply.

Treatment and study plan

SRP-1005

Drug

Subcutaneous injection

Placebo

Drug

Subcutaneous injection

Primary outcomes

  1. Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)

    Time frame: Baseline through Week 24

Secondary outcomes

  1. Plasma Concentration of SRP-1005

    Time frame: Baseline through Day 17

  2. Urine Concentration of SRP-1005

    Time frame: Baseline through Day 17

  3. Cerebrospinal Fluid Concentration of SRP-1005

    Time frame: Baseline through Week 24

Study contacts

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

Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4

CONTACT

[email protected]

1-888-SAREPTA (1-888-727-3782)

Sponsors and collaborators

Lead sponsor

Sarepta Therapeutics, Inc.

Industry

Registry information

Official study title

A Randomized, Double-Blind, Placebo-Controlled, Dose Escalation Study to Evaluate the Effect of SRP-1005 in Subjects With Huntington's Disease (INSIGHTT)

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Apr 17, 2026
Registry last updated
Jun 4, 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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