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

RecruitingPhase 1Ages 21 Years - 70 YearsAllSarepta Therapeutics, Inc.
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In plain words

What the trial is, and whether it could suit you

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

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Key Inclusion Criteria: * Genetically confirmed diagnosis of huntingtin (HTT) cytosine-adenine-guanine repeat length โ‰ฅ40. * 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.
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What it tests

  • SRP-1005
  • Placebo
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Where

5 sites
Westmead Hospital
Westmead, Australia
Royal Melbourne Hospital
Parkville, Australia
New Zealand Brain Research Institute
Christchurch, New Zealand
Siloah AG
Gรผmligen, Switzerland
University Hospital of Wales
Cardiff, United Kingdom
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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