Huntington's DiseasePhase 1NCT07536061
A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease
A Phase 1 study testing SRP-1005 in people with Huntington's Disease, run by Sarepta Therapeutics, Inc. at 5 sites in 4 countries.
Trial summary
This is a first-in-human, multi-center trial studying the effects of SRP-1005 in participants with Huntington's disease (HD). Read more about this study
- Who is it for?
- Ages 21 to 70 years
- Conditions the study is for
- Huntington's Disease
- What is being tested?
- SRP-1005
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 32 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 12 May 2026to 30 Nov 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
5 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4 contact
5 sites in 4 countries
Australia2 sites, 2 recruiting
- Recruiting
Royal Melbourne Hospital
Parkville, Victoria, 3010
SareptAlly@sarepta.com
Dennis Velakoulis MBBS, MPM DipCrim DMedSci FRANZCP
- Recruiting
Westmead Hospital
Westmead, New South Wales, 2145
SareptAlly@sarepta.com
Clement Loy
New Zealand1 site, 1 recruiting
- Recruiting
New Zealand Brain Research Institute
Christchurch, 8014
SareptAlly@sarepta.com
Timothy Anderson, MD, FRACP
Switzerland1 site, 1 recruiting
- Recruiting
Siloah AG
Gümligen, Canton of Bern, 3073
SareptAlly@sarepta.com
Michael Orth, Prof. Dr. med.
United Kingdom1 site, 1 recruiting
- Recruiting
University Hospital of Wales
Cardiff, 3AT
SareptAlly@sarepta.com
Anne Rosser, Prof.
About this study
Official title: 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).
- Conditions
- Huntington's Disease
- Being tested
- SRP-1005, Placebo
- Keywords
- Huntington's disease, SRP-1005, Dose Escalation, First in Human
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SRP-1005Drug
Subcutaneous injection
PlaceboDrug
Subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: SRP-1005 Dose 1 | Experimental | Participants will receive SRP-1005 or placebo. Receives: Drug: SRP-1005; Drug: Placebo |
| Cohort 2: SRP-1005 Dose 2 | Experimental | Participants will receive SRP-1005 or placebo. Receives: Drug: SRP-1005; Drug: Placebo |
| Cohort 3: SRP-1005 Dose 3 | Experimental | Participants will receive SRP-1005 or placebo. Receives: Drug: SRP-1005; Drug: Placebo |
| Cohort 4: SRP-1005 Dose 4 | Experimental | Participants will receive SRP-1005 or placebo. Receives: Drug: SRP-1005; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Measured: Baseline through Week 24
Secondary outcomes (3)
Plasma Concentration of SRP-1005
Measured: Baseline through Day 17
Urine Concentration of SRP-1005
Measured: Baseline through Day 17
Cerebrospinal Fluid Concentration of SRP-1005
Measured: Baseline through Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2026
- Study start12 May 2026
- Primary completion30 Nov 2027 (estimated)
- Study completion30 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington's DiseaseStudies of the same condition, open and under way.
- More trials run by Sarepta Therapeutics, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Sarepta Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07536061
- Sponsor's protocol ID
- SRP-1005-101
- Status last verified
- Oct 2026
- First submitted
- 10 Apr 2026
- Last updated
- 19 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No