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SRP-1003 Infusion

Phase 1

Myotonic Dystrophy 1 | Small molecule | Rare Disease |Arrowhead Pharmaceuticals, Inc.|Last Updated: Aug 11, 2026

Target and mechanism

ModalitySmall molecule

Also known as SRP-1003 IV Infusion

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment78

FDA Designations

No designations recorded

Clinical trial landscape

SRP-1003 Infusion · 1 trial · 1 indication

Phase 1 1
NCT06138743Study of SRP-1003 in Participants With Type 1 Myotonic DystrophyMyotonic Dystrophy 1
RECRUITING78 Analytics
PHASE1RECRUITING
Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy
Myotonic Dystrophy 1Unlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants with Treatment-emergent Adverse Events Over Time Through End of Study (EOS)
Single-dose phase (Part 1): Up to Day 90 (EOS); multiple-dose phase (Part 2): Up to Day 180 (EOS)

Secondary Endpoints

PK of SRP-1003: Maximum Observed Plasma Concentration (Cmax)
Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
PK of SRP-1003: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)
Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
PK of SRP-1003: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)
Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
SRP-1003 IV InfusionEXPERIMENTALSingle or multiple doses of SRP-1003 by intravenous (IV) infusion
Placebo by IV InfusionPLACEBO_COMPARATORSingle or multiple doses of placebo by IV infusion
SRP-1003 SC InjectionEXPERIMENTALSingle or multiple doses of SRP-1003 by subcutaneous (SC) injection
Placebo by SC InjectionPLACEBO_COMPARATORSingle or multiple doses of placebo by SC injection

Interventions

NameTypeDescription
SRP-1003 IV InfusionDRUGSRP-1003 by IV infusion
Placebo IV InfusionDRUG0.9% sodium chloride (NaCl) calculated volume to match active treatment by IV infusion
SRP-1003 SC InjectionDRUGSRP-1003 by SC injection(s)
Placebo SC InjectionDRUG0.9% NaCl calculated volume to match active treatment by SC injection(s)
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites35

Key Inclusion Criteria: * Genetically confirmed diagnosis of DM1 * Clinician-assessed signs of DM1 including clinically apparent myotonia * Onset of DM1 symptoms occurred after the age of 12 years * Walk for at least 10 meters independently at screening * Participants of childbearing potential must...

Countries:AustraliaBelgiumCanadaFranceGermanyItalyNew ZealandSpainTaiwanThailandUnited Kingdom
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Recent Changes (Last 90 Days)

MEDIUMAug 11, 2026NCT06138743primaryCompletionDate: changed
MEDIUMAug 11, 2026NCT06138743primaryCompletionDate: changed

Frequently asked questions about SRP-1003 Infusion

What is SRP-1003 Infusion used for?

SRP-1003 Infusion is an investigational small molecule being developed for the treatment of Myotonic Dystrophy 1, a rare genetic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is administered via intravenous infusion.

What does SRP-1003 target?

SRP-1003 is a small molecule designed to address the underlying cause of Myotonic Dystrophy 1. The specific molecular target has not been disclosed in available information. The drug is being evaluated in a Phase 1 clinical trial to assess its safety and tolerability in affected individuals.

Who makes SRP-1003 Infusion?

SRP-1003 Infusion is being developed by Arrowhead Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker ARWR. The company is conducting a Phase 1 clinical trial to evaluate the drug in participants with Myotonic Dystrophy 1.

What phase is SRP-1003 Infusion in?

SRP-1003 Infusion is in Phase 1 clinical development. It is an investigational drug and has not received FDA approval. The ongoing Phase 1 trial is recruiting participants with Type 1 Myotonic Dystrophy to evaluate the drug's safety, tolerability, and pharmacokinetics.

What clinical trials is SRP-1003 Infusion in?

SRP-1003 Infusion is being studied in one Phase 1 clinical trial, identified as NCT06138743, titled 'Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy.' This randomized, double-blind, placebo-controlled trial is recruiting 78 participants aged 18 years and older across multiple countries, including Australia, Belgium, Canada, France, Germany, Italy, New Zealand, Spain, Taiwan, Thailand, and the United Kingdom.

Is SRP-1003 Infusion the same as SRP-1003 IV Infusion?

Yes, SRP-1003 Infusion is also known as SRP-1003 IV Infusion. Both names refer to the same investigational drug being developed by Arrowhead Pharmaceuticals for Myotonic Dystrophy 1. The drug is administered intravenously, which is reflected in the alternative name.