First-in-human test of SRP-1005 in Huntington's disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 1, multicenter, randomized trial testing escalating doses of SRP-1005 compared with placebo in individuals with Huntington's disease. Participants have a confirmed HTT gene expansion and are in HD-ISS Stage 2 or Mild Stage 3. The study includes lumbar puncture and MRI to monitor safety, with careful screening for prior therapies and infections. The trial aims to characterize safety, tolerability, and pharmacokinetics, providing initial signals on biological activity in HD.

Why it matters

Who can join: the full rules

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.

Where it's running

• Westmead Hospital
  Westmead, New South Wales, Australia Recruiting
• Royal Melbourne Hospital
  Parkville, Victoria, Australia Recruiting
• New Zealand Brain Research Institute
  Christchurch, New Zealand Recruiting
• Siloah AG
  Gümligen, Canton of Bern, Switzerland Recruiting
• University Hospital of Wales
  Cardiff, United Kingdom Recruiting

Study contacts

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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• New PET tracer ([11C]CHDI-00491009) to image mutant huntingtin in Huntington disease

  This project will test a new PET tracer ([11C]CHDI-00491009) to see if it binds mutant huntingtin aggregates in adults with Huntington disease and in healthy volunteers.

  Recruiting Early Phase 1 CHDI Foundation, Inc. · Leuven · NCT06634628

• Evaluating ALN-HTT02 for treating Huntington's disease in adults

  This study is testing a new drug called ALN-HTT02 to see if it is safe and effective for adults with early-stage Huntington's disease.

  Recruiting at 19 sites Phase 1 Alnylam Pharmaceuticals · Edmonton and 18 other locations · NCT06585449

• Collecting cerebrospinal fluid to aid in developing treatments for Huntington's disease

  This study is collecting spinal fluid and blood samples from people with Huntington's disease to help find better treatments for the condition.

  Recruiting at 32 sites University College, London · Englewood, Colorado and 39 other locations · NCT02855476

• Global registry for Huntington's Disease

  This study is gathering health information from people with Huntington's disease and their families to help researchers learn more about the disease and find better treatments.

  Recruiting at 160 sites CHDI Foundation, Inc. · Birmingham, Alabama and 182 other locations · NCT01574053

• Pridopidine for Huntington's Disease: a 1-year, randomized, placebo-controlled study

  This trial tests whether pridopidine can slow the progression of Huntington's disease in adults compared with placebo over one year.

  Recruiting at 17 sites Phase 3 Prilenia · Birmingham, Alabama and 65 other locations · NCT07609108

• Low-intensity focused ultrasound targeting the globus pallidus to improve movement in Huntington's disease

  This trial tests whether different pulse frequencies of low-intensity focused ultrasound (LIFU) aimed at a deep brain target can safely reduce chorea and other motor symptoms in adults with Huntington's disease.

  Recruiting First Affiliated Hospital of Fujian Medical University · Fuzhou, Fujian · NCT07513844

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.