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