A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease
Phase 1 – testing safety in a small group of people
Conditions:
Huntington's Disease
Sponsor: Sarepta Therapeutics, Inc.
trial.available_in:
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Overview
This is a first-in-human, multi-center trial studying the effects of SRP-1005 in participants with Huntington's disease (HD).
Who can participate
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.
Locations
1
New Zealand (1)
Pacific Clinical Research Network - Auckland (PCRN Auckland)