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SRP-1003

Phase 1

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

Target and mechanism

Molecular targetDMPK
ModalitySmall molecule

Also known as SRP-1003 IV Infusion, SRP-1003 Infusion, SRP-1003 SC Injection

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 · 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