Recent Updates
Recently added Catalysts

SRP-1005

Phase 1

Huntington's Disease | Small molecule | Neurology |Sarepta Therapeutics, Inc.|Last Updated: Sep 4, 2026

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials1
Total Enrollment32

FDA Designations

No designations recorded

Clinical trial landscape

SRP-1005 · 1 trial · 1 indication

Phase 1 1
NCT07536061A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's DiseaseHuntington's Disease
RECRUITING32 Analytics
PHASE1RECRUITING
A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease
Huntington's DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

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 Endpoints

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
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1: SRP-1005 Dose 1EXPERIMENTALParticipants will receive SRP-1005 or placebo.
Cohort 2: SRP-1005 Dose 2EXPERIMENTALParticipants will receive SRP-1005 or placebo.
Cohort 3: SRP-1005 Dose 3EXPERIMENTALParticipants will receive SRP-1005 or placebo.
Cohort 4: SRP-1005 Dose 4EXPERIMENTALParticipants will receive SRP-1005 or placebo.

Interventions

NameTypeDescription
SRP-1005DRUGSubcutaneous injection
PlaceboDRUGSubcutaneous injection
Unlock Study Design Details

Eligibility Criteria

Age Range21 Years to 70 Years
SexALL
Healthy VolunteersNo
Study Sites4

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 a...

Countries:AustraliaNew ZealandSwitzerlandUnited Kingdom
Unlock Eligibility Criteria

Competitive Landscape -Huntington's Disease 8 trials

Recent Changes (Last 90 Days)

LOWSep 4, 2026NCT07536061lastUpdatePostDate: changed

Frequently asked questions about SRP-1005

What is SRP-1005 used for?

SRP-1005 is an investigational small molecule being developed for the treatment of Huntington's Disease. It is currently in Phase 1 clinical development. The drug is being studied in a first-in-human trial to evaluate its effects in participants with Huntington's Disease.

What does SRP-1005 target?

The specific molecular target of SRP-1005 has not been disclosed. It is a small molecule being developed for Huntington's Disease, but its mechanism of action has not been publicly detailed.

Who makes SRP-1005?

SRP-1005 is being developed by Sarepta Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol SRPT. The company is conducting a Phase 1 clinical trial of SRP-1005 in participants with Huntington's Disease.

What phase is SRP-1005 in?

SRP-1005 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. A first-in-human study is currently recruiting participants with Huntington's Disease to evaluate the drug's effects.

What clinical trials is SRP-1005 in?

SRP-1005 is being studied in a Phase 1 clinical trial registered as NCT07536061, titled 'A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease.' The trial is recruiting 32 participants in New Zealand and is currently active.

Is SRP-1005 the same as any other drug?

No alternative names for SRP-1005 have been disclosed. It is identified solely by its development code SRP-1005 and is being studied under this name in clinical trials for Huntington's Disease.