RecruitingPhase 1NCT07536061

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

A Randomized, Double-Blind, Placebo-Controlled, Dose Escalation Study to Evaluate the Effect of SRP-1005 in Subjects With Huntington's Disease (INSIGHTT)

Sponsor

Sarepta Therapeutics, Inc.

Enrollment

32 participants

Start Date

May 12, 2026

Study Type

INTERVENTIONAL

Conditions

Huntington’s Disease

Summary

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

Eligibility

Min Age: 21 YearsMax Age: 70 Years

Inclusion Criteria3

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.

Exclusion Criteria6

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.