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Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy

StatusRecruiting
PhasePhase 1/2
Started2024-03-04
View on ClinicalTrials.gov ↗
Trial flagged as At Risk
Primary completion pushed: 2026-12 → 2026-12-31
Primary endpoint reworded 1 of 1 changed
See other at-risk trials from Sarepta Therapeutics, Inc.

Amendment history

2026-08-07
Study Identification, Study Status, Sponsor/Collaborators, Study Description, Conditions, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v11
Primary endpoint(s) modified1 entry, revised
WasNumber of Participants with Treatment -Emergent Adverse Events (TEAEs) Over Time Through End of Study (EOS)
NowNumber of Participants with Treatment-emergent Adverse Events Over Time Through End of Study (EOS)
Sponsor changed: Arrowhead Pharmaceuticals → Sarepta Therapeutics, Inc.+1 characters
WasArrowhead Pharmaceuticals
NowSarepta Therapeutics, Inc.
Trial sites expanded: 11 → 35 locations
Primary completion pushed: 2026-12 → 2026-12-31
Completion pushed: 2026-12 → 2026-12-31
InterventionsARO-DM1 Intravenous (IV) Infusion (Drug), ARO-DM1 subcutaneous (SC) injection (Drug), Placebo Intravenous (IV) Infusion (Drug), Placebo…→Placebo IV Infusion (Drug), Placebo SC Injection (Drug), SRP-1003 IV Infusion (Drug), SRP-1003 SC Injection (Drug)
Eligibility criteria+16 characters
Key Inclusion Criteria: Genetically confirmed diagnosis of DM1 C [...] ge of 12 years Walk for at least 10 meters independently at Screeningscreening SubjectsParticipants of childbearing potential must agree to use highly effectiv [...] nd of study or last dose of study drug, whichever is later. SubjectsParticipants must not donate sperm or eggs during the study and for at l [...] end of study or last dose of study drug whichever is later. Key Exclusion Criteria: Inadequately controlled diabetes Confirm [...] riod Clinically significant cardiac, liver or renal disease HIVHuman immunodeficiency virus infection (seropositi [...] [...]
Secondary endpoints11 entries, revised
Pharmacokinetics (PK) of AROSRP-DM11003: Maximum Observed Plasma Concentration (Cmax) PK of AROSRP-DM11003: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24) PK of AROSRP-DM11003: Area Under the Plasma Concentration Versus Time Curve from [...] the Last Quantifiable Plasma Concentration (AUClast) PK of AROSRP-DM11003: Area Under the Plasma Concentration Versus Time Curve from Zero to Infinity (AUCinf)
Study title+5 characters
Study of AROSRP-DM11003 in SubjectsParticipants With Type 1 Myotonic Dystrophy
2025-11-06
Study Status, Study Description, Study Design, Arms and Interventions, Outcome Measures, Eligibility v10
Primary completion date2026-09→2026-12
Completion date2026-09→2026-12
Enrollment target90→78
Study arms2→4
InterventionsARO-DM1 for Injection (Drug), Placebo (Drug)→ARO-DM1 Intravenous (IV) Infusion (Drug), ARO-DM1 subcutaneous (SC) injection (Drug), Placebo Intravenous (IV) Infusion (Drug), Placebo…
Eligibility criteriarevised
[...] osis of congenital DM1 Uncontrolled hypertension History of Tibialistibialis Anterioranterior (TA) biopsy within 3 months of Day 1 or planning to undergo [...] agulation parameters at Screening including platelet count, Internationalinternational Normalizednormalized Ratioratio (INR), prothrombin time, and activated partial thromboplast [...]
Secondary endpointsdetails revised at 5 of 11 entries
Primary endpoints2 to 1 entries
Change from Baseline in Skeletal Muscle Dystrophia Myotonica-Protein Kinase (DMPK) Mediated Changes in the Composite Myotonic Dystrophy Type 1 Splicing Indices
2025-08-07
Study Status, Study Design, Outcome Measures, Eligibility, Contacts/Locations v9
Primary completion date2025-06→2026-09
Completion date2025-06→2026-09
Enrollment target36→90
Study sites9→11
Eligibility criteria-336 characters
[...] irmed diagnosis of congenital DM1 Uncontrolled hypertension History of thromboembolic events History of Tibialis Anterior (TA) biopsy within 3 months of [...] mbin time, and activated partial thromboplastin time (APTT) History or presence of any of the following: hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome or myeloproliferative diseases, inability to ambulate, use of hormone-based contraceptives and peri/post- menopausal hormone replacement therapy ≤ 16 weeks prior to Day [...] Note: Additional inclusion/exclusion criteria may apply per protocol
Secondary endpoints8 to 11 entries
PKChange offrom ARO-DM1:Baseline at Day 120 for Video Hand Opening Time to(vHOT) Change Maximumfrom ObservedBaseline PlasmaOver ConcentrationTime for the Timed Up and Go Test (TmaxTUG) Assessment PKChange offrom AROBaseline Over Time for the 10-DM1:Meter EliminationWalk/Run half-lifeTest (t1/210MWT) Assessment PKChange offrom AROBaseline Over Time for the Hand-DM1:held ApparentQuantitative SystemicDynamometry ClearanceAssessment Change from Baseline Over Time for the Video Hand Opening Time (CL/FvHOT) Assessment PKChange offrom ARO-DM1:mBaseline ApparentOver Terminal-phaseTime Volumefor ofthe [...]
Primary endpoints1 to 2 entries
Change from Baseline in Skeletal Muscle Dystrophia Myotonica-Protein Kinase (DMPK) Mediated Changes in the Composite Myotonic Dystrophy Type 1 Splicing Indices
2025-03-23
Study Status, Study Design v8
PhasePhase 1→Phase 1/2
2025-02-26
Study Status, Contacts/Locations v7
Study sites4→9
Show 6 earlier versions
2024-09-30
Study Identification, Study Status, Study Design, Outcome Measures, Eligibility, Contacts/Locations v6
Primary completion date2025-10→2025-06
Completion date2026-10→2025-06
Enrollment target48→36
Study sites3→4
Eligibility criteria+511 characters
Inclusion Criteria: Genetically confirmed diagnosis of DM1 Clinician-assessed signedsigns of DM1 including clinically apparent myotonia Onset of DM1 [...] irmed diagnosis of congenital DM1 Uncontrolled hypertension History of thromboembolic events History of Tibialis Anterior (TA) biopsy within 3 months of [...] eriod of 5 half-lives of the medication prior to Screening. Abnormal coagulation parameters at Screening including platelet count, International Normalized Ratio (INR), prothrombin time, and activated partial thromboplastin time (APTT) History or presence of any of the following: hypercoagulable s [...] [...]
2024-06-26
Study Status, Contacts/Locations v5
Study sites2→3
2024-04-17
Study Status, Contacts/Locations v4
Study sites1→2
2024-03-12
Study Status, Contacts/Locations v3
Re-verified, no change to tracked fields
2024-03-05
Not Yet Recruiting→Recruiting Study Status, Contacts/Locations v2
Trial statusNot Yet Recruiting→Recruiting
Start dateestimated 2024-03→confirmed 2024-03-04
Study sites0→1
2024-02-23
Study Status v1
Completion date2025-10→2026-10
Start date2024-01→2024-03
2023-11-14
Original filing
This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.
Trial Details
NCT Number NCT06138743
Lead Sponsor Sarepta Therapeutics, Inc.
Conditions Myotonic Dystrophy 1
Enrollment 78 participants
Start Date 2024-03-04
Primary Completion 2026-12-31 (estimated)
Study Completion 2026-12-31 (estimated)
Updated on ClinicalTrials.gov 2026-08-11