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Also known as SRP-1003 IV Infusion, SRP-1003 Infusion, SRP-1003 SC Injection
SRP-1003 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| SRP-1003 IV Infusion | EXPERIMENTAL | Single or multiple doses of SRP-1003 by intravenous (IV) infusion |
| Placebo by IV Infusion | PLACEBO_COMPARATOR | Single or multiple doses of placebo by IV infusion |
| SRP-1003 SC Injection | EXPERIMENTAL | Single or multiple doses of SRP-1003 by subcutaneous (SC) injection |
| Placebo by SC Injection | PLACEBO_COMPARATOR | Single or multiple doses of placebo by SC injection |
| Name | Type | Description |
|---|---|---|
| SRP-1003 IV Infusion | DRUG | SRP-1003 by IV infusion |
| Placebo IV Infusion | DRUG | 0.9% sodium chloride (NaCl) calculated volume to match active treatment by IV infusion |
| SRP-1003 SC Injection | DRUG | SRP-1003 by SC injection(s) |
| Placebo SC Injection | DRUG | 0.9% NaCl calculated volume to match active treatment by SC injection(s) |
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...
SRP-1003 is an investigational small molecule drug being developed by Arrowhead Pharmaceuticals, Inc. for the treatment of myotonic dystrophy type 1, a rare inherited muscle disorder. It is currently in Phase 1 clinical development and has not been approved by any regulatory agency.
SRP-1003 is being studied as a potential treatment for myotonic dystrophy type 1, a rare genetic condition that causes progressive muscle weakness, myotonia, and other systemic symptoms. It is investigational and has not been approved for any indication. Its safety and efficacy in this patient population are still being evaluated in clinical trials.
SRP-1003 targets DMPK, the gene and transcript associated with myotonic dystrophy type 1. The disease is caused by expanded CTG repeats in the DMPK gene, and SRP-1003 is designed as a small molecule directed at this target. It is being evaluated in a Phase 1 study in adults with the condition.
SRP-1003 is being developed by Arrowhead Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker symbol ARWR. The company is the sponsor of the Phase 1 clinical trial evaluating SRP-1003 in participants with type 1 myotonic dystrophy.
SRP-1003 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory authority. The ongoing Phase 1 trial is enrolling participants with type 1 myotonic dystrophy to evaluate the drug's safety and tolerability.
SRP-1003 is being evaluated in one active clinical trial, NCT06138743, titled "Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy." The Phase 1 study is randomized, double-blind, and placebo-controlled, with an planned enrollment of 78 adults aged 18 and older across sites in Australia, Belgium, Canada, France, Germany, Italy, New Zealand, Spain, Taiwan, Thailand, and the United Kingdom.
Yes. SRP-1003 IV Infusion, SRP-1003 Infusion, and SRP-1003 SC Injection are alternative names referring to the same investigational drug, SRP-1003, developed by Arrowhead Pharmaceuticals for myotonic dystrophy type 1. These names reflect different routes of administration being studied in the Phase 1 program.