Recent Updates
Recently added Catalysts

SRP-1001

Phase 1

Muscular Dystrophy, Facioscapulohumeral | Small molecule | Neurology |Arrowhead Pharmaceuticals, Inc.|Last Updated: Aug 11, 2026

Target and mechanism

ModalitySmall molecule

Also known as SRP-1001 for Injection

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment60

FDA Designations

No designations recorded

Clinical trial landscape

SRP-1001 · 1 trial · 1 indication

Phase 1 1
NCT06131983Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1Muscular Dystrophy, Facioscapulohumeral
RECRUITING60 Analytics
PHASE1RECRUITING
Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1
Muscular Dystrophy, Facioscapulohumeral
NCT0613198360
Unlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Treatment-Emergent Adverse Events Over Time Through End of Study
Part 1: Up to Day 90; Part 2: Up to Day 360

Secondary Endpoints

PK of SRP-1001: Maximum Observed Plasma Concentration (Cmax)
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
PK of SRP-1001: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
PK of SRP-1001: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)
Part 1: through 48 hours post-dose (all cohorts) and through 48 hours post second dose (Cohorts 3 & 4 only); Part 2: through 8 hours post first and second dose
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
SRP-1001EXPERIMENTALSRP-1001 for injection
PlaceboPLACEBO_COMPARATORSodium chloride (0.9%)

Interventions

NameTypeDescription
SRP-1001 for InjectionDRUGSingle or multiple doses of SRP-1001 by intravenous (IV) infusion
PlaceboDRUGCalculated volume to match active treatment by IV infusion
Unlock Study Design Details

Eligibility Criteria

Age Range16 Years to 70 Years
SexALL
Healthy VolunteersNo
Study Sites16

Key Inclusion Criteria: * Genetically confirmed FSHD1 based on screening evaluation or source verifiable medical record * Clinical severity score between 3 and 8 (scale, 0 to 10) * Must have an eligible lower extremity muscle for biopsy as determined from MRI by a central reader, with muscle fat fr...

Countries:AustraliaCanadaGermanyItalyNetherlandsNew ZealandSpain
Unlock Eligibility Criteria

Recent Changes (Last 90 Days)

MEDIUMAug 11, 2026NCT06131983primaryCompletionDate: changed
MEDIUMAug 11, 2026NCT06131983primaryCompletionDate: changed

Frequently asked questions about SRP-1001

What is SRP-1001 used for?

SRP-1001 is an investigational small molecule being developed for the treatment of facioscapulohumeral muscular dystrophy type 1, a form of muscular dystrophy. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.

Who makes SRP-1001?

SRP-1001 is being developed by Arrowhead Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol ARWR. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational drug.

What phase is SRP-1001 in?

SRP-1001 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory agencies and is still undergoing clinical trials to assess its safety and effectiveness in patients with facioscapulohumeral muscular dystrophy type 1.

What clinical trials is SRP-1001 in?

SRP-1001 is being studied in a Phase 1 clinical trial identified as NCT06131983, titled 'Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1'. The trial is currently recruiting participants and is expected to enroll approximately 60 individuals.

Is SRP-1001 the same as SRP-1001 for Injection?

Yes, SRP-1001 is also known as SRP-1001 for Injection. This alternative name reflects the route of administration used in clinical trials, where the drug is given as an injection to participants.

What is the design of the SRP-1001 clinical trial?

The SRP-1001 Phase 1 trial is a randomized, double-blind, placebo-controlled study. It is enrolling adult and adolescent participants aged 16 years and older with facioscapulohumeral muscular dystrophy type 1. The trial is being conducted in multiple countries, including Australia, Canada, Germany, Italy, Netherlands, New Zealand, and Spain.