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RecruitingNCT07536061Updated Sep 4, 2026

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

A Phase 1 interventional study of SRP-1005 and Placebo in Huntington's Disease, sponsored by Sarepta Therapeutics, Inc.. Recruiting at 4 sites in 4 countries. Open to participants aged 21 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-09-04.

Sponsored by Sarepta Therapeutics, Inc. · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
32
Allocation
Randomized
Ages
21 Years to 70 Years
Sex
All
01

Study summary

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

02

Conditions studied

  • Huntington's Disease

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Keywords

  • Huntington's disease
  • SRP-1005
  • Dose Escalation
  • First in Human
03

Who can participate

Ages eligible
21 Years to 70 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

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.

04

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Care provider, Investigator)
Enrollment
32 participants (estimated)

Study arms

  • Experimental
    Cohort 1: SRP-1005 Dose 1

    Participants will receive SRP-1005 or placebo.

    Drug: SRP-1005 · Drug: Placebo

  • Experimental
    Cohort 2: SRP-1005 Dose 2

    Participants will receive SRP-1005 or placebo.

    Drug: SRP-1005 · Drug: Placebo

  • Experimental
    Cohort 3: SRP-1005 Dose 3

    Participants will receive SRP-1005 or placebo.

    Drug: SRP-1005 · Drug: Placebo

  • Experimental
    Cohort 4: SRP-1005 Dose 4

    Participants will receive SRP-1005 or placebo.

    Drug: SRP-1005 · Drug: Placebo

Interventions

  • DrugSRP-1005

    Subcutaneous injection

  • DrugPlacebo

    Subcutaneous injection

05

What researchers measure

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

06

Study locations

4 of 4 sites recruiting
  • Westmead Hospital
    Westmead, New South Wales 2145, Australia
    Recruiting
  • New Zealand Brain Research Institute
    Christchurch, 8014, New Zealand
    Recruiting
  • Siloah AG
    Gümligen, Canton of Bern 3073, Switzerland
    Recruiting
  • University Hospital of Wales
    Cardiff, 3AT, United Kingdom
    Recruiting
07

Registry details

Key details

Study ID
NCT07536061
Lead sponsor
Sarepta Therapeutics, Inc.
Responsible party
Sponsor
First posted
Apr 17, 2026
Start date
May 12, 2026
Primary completion
Nov 30, 2027 (estimated)
Completion
Nov 30, 2027 (estimated)
Last update
Sep 4, 2026

Study contacts

Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
Contact
SareptAlly@sarepta.com
1-888-SAREPTA (1-888-727-3782)
Medical Director
study director · Sarepta Therapeutics, Inc.

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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