Phase 1
N=12
Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT02530905 ↗Enrolled (actual)
12
Serious AEs
8.3%
Results posted
May 2021
Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) — 4; 5; 3; 3 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- SRP-4045 (Drug); Placebo (Drug)
- Age
- Pediatric, Adult · 7+ yrs
- Sex
- Male
- Sponsor
- Sarepta Therapeutics, Inc.
- Primary completion
- Oct 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) |
4; 5; 3; 3; 7; 12 | — |
| PRIMARY Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs |
0; 0; 0; 0; 1; 1 | — |
| PRIMARY Number of Participants With Potentially Clinically Significant Abnormalities in Electrocardiogram (ECG) Reported as TEAEs |
0; 0; 0; 0; 0; 1 | — |
| PRIMARY Number of Participants With Potentially Clinically Significant Abnormalities in Echocardiograms (ECHO) |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Maximum Plasma Concentration (Cmax) of Casimersen |
13700; 39400; 64400; 119000; 115000 | — |
| SECONDARY Time to Reach Maximum Plasma Concentration (Tmax) of Casimersen |
1.11; 1.03; 1.03; 0.94; 0.95 | — |
| SECONDARY Area Under Concentration-time Curve From Time of Dosing to the Last Measurable Concentration (AUClast) of Casimersen in Plasma |
23100; 59500; 101000; 188000; 182000 | — |
| SECONDARY Area Under Concentration-Time Curve From Time Zero Pre-dose to Twenty-Four Hours Post-dose (AUC0-24) of Casimersen in Plasma |
23200; 59500; 101000; 188000; 182000 | — |
| SECONDARY Area Under the Concentration-Time Curve From Time Zero Extrapolated to the Infinity (AUCinf) of Casimersen in Plasma |
23300; 58300; 101000; 189000; 182000 | — |
| SECONDARY Apparent Volume of Distribution at Steady State (Vss) of Casimersen |
0.369; 0.343; 0.407; 0.319; 0.367 | — |
| SECONDARY Elimination Half-life (T1/2) of Casimersen |
2.92; 3.29; 3.71; 3.82; 3.45 | — |
| SECONDARY Total Clearance (CL) of Casimersen |
0.177; 0.181; 0.205; 0.163; 0.180 | — |
| SECONDARY Mean Residence Time Extrapolated to Infinity (MRTinf) of Casimersen |
2.08; 1.89; 1.98; 1.96; 2.04 | — |
| SECONDARY Double-Blind Period: Renal Clearance (CLR) of Casimersen |
0.137; 0.162; 0.209; 0.177 | — |
Summary
This is a first-in-human dose-titration and open-label extension study to assess safety, tolerability, and pharmacokinetics of SRP-4045 in advanced-stage Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 45 skipping.
Eligibility Criteria
Inclusion Criteria
- Genotypically confirmed DMD (amenable to exon 45 skipping).
- Stable cardiac and pulmonary function.
- Limited or no ambulation.
- On a stable dose of oral corticosteroids for at least 24 weeks OR has not received corticosteroids for at least 24 weeks.
Exclusion Criteria
- Current or previous treatment with the experimental agents SMT C1100 (BMN-195) or PRO045.
- Other experimental treatment in the past 12 weeks.
- If on cardiac medication, must be on a stable dose for the past 12 weeks.
- Major surgery within the past 3 months.
Other inclusion/exclusion criteria apply.
Data sourced from ClinicalTrials.gov (NCT02530905). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.