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

June 3, 2026 updated by: Sarepta Therapeutics, Inc.

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

32

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
  • Phone Number: 1-888-SAREPTA (1-888-727-3782)
  • Email: SareptAlly@sarepta.com

Study Locations

  • New Zealand
      • Auckland, New Zealand, 622
        • Not yet recruiting
        • Pacific Clinical Research Network - Auckland (PCRN Auckland)
        • Contact:
        • Principal Investigator:
          • Richard Roxburgh, FRACP, PHD
      • Christchurch, New Zealand, 8014
        • Recruiting
        • New Zealand Brain Research Institute
        • Contact:
        • Principal Investigator:
          • Timothy Anderson, MD, FRACP

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1: SRP-1005 Dose 1
Participants will receive SRP-1005 or placebo.
Drug: Placebo
Subcutaneous injection
Drug: SRP-1005
Subcutaneous injection
Experimental: Cohort 2: SRP-1005 Dose 2
Participants will receive SRP-1005 or placebo.
Drug: Placebo
Subcutaneous injection
Drug: SRP-1005
Subcutaneous injection
Experimental: Cohort 3: SRP-1005 Dose 3
Participants will receive SRP-1005 or placebo.
Drug: Placebo
Subcutaneous injection
Drug: SRP-1005
Subcutaneous injection
Experimental: Cohort 4: SRP-1005 Dose 4
Participants will receive SRP-1005 or placebo.
Drug: Placebo
Subcutaneous injection
Drug: SRP-1005
Subcutaneous injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Baseline through Week 24

Secondary Outcome Measures

Outcome Measure
Time Frame
Plasma Concentration of SRP-1005
Time Frame: Baseline through Day 17
Baseline through Day 17
Urine Concentration of SRP-1005
Time Frame: Baseline through Day 17
Baseline through Day 17
Cerebrospinal Fluid Concentration of SRP-1005
Time Frame: Baseline through Week 24
Baseline through Week 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sarepta Therapeutics, Inc.

Investigators

  • Study Director: Medical Director, Sarepta Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 12, 2026

Primary Completion (Estimated)

November 30, 2027

Study Completion (Estimated)

November 30, 2027

Study Registration Dates

First Submitted

April 10, 2026

First Submitted That Met QC Criteria

April 10, 2026

First Posted (Actual)

April 17, 2026

Study Record Updates

Last Update Posted (Actual)

June 4, 2026

Last Update Submitted That Met QC Criteria

June 3, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SRP-1005-101

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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