Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
SRP-4045 · 2 trials · 1 indication
Adverse event (AE) was any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with the investigational drug. AEs also included abnormal physical examination findings (Physical examination were conducted per protocol and any clinically significant abnormal findings were recorded in medical history if pre-existing or addressed as an AE if new or worsening). TEAEs was defined as AEs that started, worsened, or became serious on or after the start of first infusion through 148 weeks. Number of participants with TEAEs were reported.
Laboratory parameters included serum chemistry (hepatic chemistry and renal chemistry), hematology, coagulation, and urinalysis. Number of participants with potentially clinically significant abnormal finding were reported as TEAEs. The Investigator determined whether abnormal assessment results were potentially clinically significant or not. Potentially clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.
Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Number of participants with at least one potentially clinically significant abnormal vital signs findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were potentially clinically significant or not. Potential clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.
Twelve-lead ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the participant was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel. Number of participants with potentially clinically significant abnormalities in ECG reported as TEAEs were presented here. The Investigator determined whether abnormal assessment results were potentially clinically significant or not.
Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study.The ECHO was reviewed and interpreted by medically qualified personnel. Number of participants with potentially clinically significant abnormalities in ECHO were reported.
| Arm | Type | Description |
|---|---|---|
| SRP-4045 | EXPERIMENTAL | Participants amenable to exon 45 skipping will receive SRP-4045 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study). |
| SRP-4053 | EXPERIMENTAL | Participants amenable to exon 53 skipping will receive SRP-4053 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study). |
| Placebo followed by SRP-4045 or SRP-4053 | PLACEBO_COMPARATOR | Participants amenable to exon 45 or 53 skipping will receive SRP-4045 or SRP-4053 placebo-matching IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 or SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study). |
| Placebo (double-blind dose titration) | PLACEBO_COMPARATOR | Participants with genotypically confirmed Duchenne muscular dystrophy (DMD) characterized by deletions amenable to exon 45 skipping will receive placebo-matching to casimersen intravenous (IV) infusions, once weekly over approximately 12 weeks in the double-blind period. |
| SRP-4045 (double-blind dose titration) | EXPERIMENTAL | Participants with genotypically confirmed DMD characterized by deletions amenable to exon 45 skipping will receive weekly IV infusions of casimersen at four escalating dose levels, each for at least 2 weeks: 4 milligrams per kilograms (mg/kg) during Week 1 to Week 2, followed by 10 mg/kg during Week 3 to Week 4, followed by 20 mg/kg during Week 5 to Week 6, followed by 30 mg/kg beginning at Week 7 and continue over approximately Week 12 in the double-blind period. |
| SRP-4045 (open label extension period) | EXPERIMENTAL | All participants who completed double blind period will be enrolled to receive casimersen 30 mg/kg once weekly, for up to Week 144 in the open label extension period. |
| Name | Type | Description |
|---|---|---|
| SRP-4045 | DRUG | SRP-4045 solution for IV infusion |
| SRP-4053 | DRUG | SRP-4053 solution for IV infusion |
| Placebo | DRUG | SRP-4045 or SRP-4053 placebo-matching solution for IV infusion |
Inclusion Criteria: * Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53 skipping * Stable dose of oral corticosteroids for at least 24 weeks prior to Week 1, and the dose is expected to remain constant throughout the study (except for modifications to accommodate cha...
SRP-4045 is an investigational small molecule being developed for the treatment of Duchenne Muscular Dystrophy (DMD). It is designed for male patients, with clinical trials enrolling participants as young as 6 years old. The drug is currently in Phase 3 clinical development, though the specific mechanism of action has not been detailed.
SRP-4045 is developed by Sarepta Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol SRPT. Sarepta is conducting clinical trials for this drug in Duchenne Muscular Dystrophy, with studies completed in multiple countries including the United States, Europe, and Asia.
SRP-4045 is in Phase 3 clinical development for Duchenne Muscular Dystrophy. It is an investigational drug and has not been approved by regulatory authorities. The Phase 3 trial has been completed, along with a Phase 1 study, but the drug remains in clinical development.
SRP-4045 has been studied in two completed clinical trials. The Phase 3 trial (NCT02500381) enrolled 228 participants and evaluated SRP-4045 in combination with another drug in Duchenne Muscular Dystrophy. A Phase 1 dose-titration and open-label extension study (NCT02530905) enrolled 12 patients with advanced DMD.
SRP-4045 is also known as casimersen. In clinical trials, it has been studied alongside another investigational drug, SRP-4053 (golodirsen). The Phase 3 trial NCT02500381 specifically evaluated both SRP-4045 and SRP-4053 in participants with Duchenne Muscular Dystrophy.
The Phase 3 trial of SRP-4045 (NCT02500381) was a randomized, double-blind, placebo-controlled study in 228 male participants with Duchenne Muscular Dystrophy. It was conducted across multiple countries, including the United States, and enrolled patients aged 6 years and older. The trial has been completed.