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)

Status

Recruiting

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07536061

Enrollment

Registered

2026-04-17

Start date

2026-05-12

Completion date

2027-11-30

Last updated

2026-09-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Huntington's Disease

Keywords

Huntington's disease, SRP-1005, Dose Escalation, First in Human

Brief summary

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

Interventions

DRUGSRP-1005

Subcutaneous injection

DRUGPlacebo

Subcutaneous injection

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

TRIPLE (Subject, Caregiver, Investigator)

Eligibility

Sex/Gender

ALL

Age

21 Years to 70 Years

Healthy volunteers

Inclusion criteria

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/

Design outcomes

Primary

Measure	Time frame
Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)	Baseline through Week 24

Secondary

Measure	Time frame
Plasma Concentration of SRP-1005	Baseline through Day 17
Urine Concentration of SRP-1005	Baseline through Day 17
Cerebrospinal Fluid Concentration of SRP-1005	Baseline through Week 24

Countries

Australia, New Zealand, Switzerland, United Kingdom

Contacts

CONTACTSarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4

SareptAlly@sarepta.com1-888-SAREPTA (1-888-727-3782)

STUDY_DIRECTORMedical Director

Sarepta Therapeutics, Inc.

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Sep 5, 2026