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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT07536061
- Acronym / sponsor ID
- SRP-1005-101
- Conditions
- Huntington's Disease
- Keywords
- Huntington's disease, SRP-1005, Dose Escalation, First in Human
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 12 May 2026 (actual)
- Primary completion
- 30 Nov 2027 (estimated)
- Study completion
- 30 Nov 2027 (estimated)
- First posted
- 17 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a first-in-human, multi-center trial studying the effects of SRP-1005 in participants with Huntington's disease (HD).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: SRP-1005 Dose 1 | Experimental | Drug: SRP-1005, Drug: PlaceboParticipants will receive SRP-1005 or placebo. |
| Cohort 2: SRP-1005 Dose 2 | Experimental | Drug: SRP-1005, Drug: PlaceboParticipants will receive SRP-1005 or placebo. |
| Cohort 3: SRP-1005 Dose 3 | Experimental | Drug: SRP-1005, Drug: PlaceboParticipants will receive SRP-1005 or placebo. |
| Cohort 4: SRP-1005 Dose 4 | Experimental | Drug: SRP-1005, Drug: PlaceboParticipants will receive SRP-1005 or placebo. |
Who may qualify
- Ages
- 21 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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 (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SRP-1005Subcutaneous injection | Drug | Cohort 1: SRP-1005 Dose 1, Cohort 2: SRP-1005 Dose 2, Cohort 3: SRP-1005 Dose 3, Cohort 4: SRP-1005 Dose 4 |
| PlaceboSubcutaneous injection | Drug | Cohort 1: SRP-1005 Dose 1, Cohort 2: SRP-1005 Dose 2, Cohort 3: SRP-1005 Dose 3, Cohort 4: SRP-1005 Dose 4 |
Primary outcome
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
3 secondary outcomes
Plasma Concentration of SRP-1005
Time frame: Baseline through Day 17
Urine Concentration of SRP-1005
Time frame: Baseline through Day 17
Cerebrospinal Fluid Concentration of SRP-1005
Time frame: Baseline through Week 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4 contact
Medical Director study director
Sarepta Therapeutics, Inc.
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SRP-1005 in people with Huntington's Disease, run by Sarepta Therapeutics, Inc. at 5 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Australia, New Zealand, Switzerland, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4 (1-888-SAREPTA (1-888-727-3782), SareptAlly@sarepta.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2026.