NCT07536061

Brief Summary

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

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
32

participants targeted

Target at P50-P75 for phase_1

Timeline
19mo left

Started May 2026

Geographic Reach
1 country

2 active sites

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 10, 2026

Completed
7 days until next milestone

First Posted

Study publicly available on registry

April 17, 2026

Completed
18 days until next milestone

Study Start

First participant enrolled

May 5, 2026

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 30, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 30, 2027

Last Updated

April 17, 2026

Status Verified

April 1, 2026

Enrollment Period

1.6 years

First QC Date

April 10, 2026

Last Update Submit

April 10, 2026

Conditions

Huntington's Disease

Keywords

Huntington's diseaseSRP-1005Dose EscalationFirst in Human

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (3)

  • 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

Study Arms (4)

Cohort 1: SRP-1005 Dose 1

EXPERIMENTAL

Participants will receive SRP-1005 or placebo.

Drug: SRP-1005Drug: Placebo

Cohort 2: SRP-1005 Dose 2

EXPERIMENTAL

Participants will receive SRP-1005 or placebo.

Drug: SRP-1005Drug: Placebo

Cohort 3: SRP-1005 Dose 3

EXPERIMENTAL

Participants will receive SRP-1005 or placebo.

Drug: SRP-1005Drug: Placebo

Cohort 4: SRP-1005 Dose 4

EXPERIMENTAL

Participants will receive SRP-1005 or placebo.

Drug: SRP-1005Drug: Placebo

Interventions

SRP-1005DRUG

Subcutaneous injection

Cohort 1: SRP-1005 Dose 1Cohort 2: SRP-1005 Dose 2Cohort 3: SRP-1005 Dose 3Cohort 4: SRP-1005 Dose 4
PlaceboDRUG

Subcutaneous injection

Cohort 1: SRP-1005 Dose 1Cohort 2: SRP-1005 Dose 2Cohort 3: SRP-1005 Dose 3Cohort 4: SRP-1005 Dose 4

Eligibility Criteria

Age21 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Pacific Clinical Research Network - Auckland (PCRN Auckland)

Auckland, 622, New Zealand

Location

New Zealand Brain Research Institute

Christchurch, 8014, New Zealand

Location

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesDementiaChoreaDyskinesiasMovement DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCognition DisordersNeurocognitive DisordersMental Disorders

Study Officials

  • Medical Director

    Sarepta Therapeutics, Inc.

    STUDY DIRECTOR

Central Study Contacts

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

CONTACT

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

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 10, 2026

First Posted

April 17, 2026

Study Start

May 5, 2026

Primary Completion (Estimated)

November 30, 2027

Study Completion (Estimated)

November 30, 2027

Last Updated

April 17, 2026

Record last verified: 2026-04

Locations

NZ(2)