A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Conditions

Duchenne Muscular Dystrophy

Brief summary

Part A: Incidence of Adverse Events (AEs), Baseline up to 75 weeks, Part B: Change From Baseline in Dystrophin Protein Level at Week 28

Detailed description

Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen, Pre-dose and at multiple time points (up to 32 hours) after end of infusion, Part A: PK: Urine Concentration of Vesleteplirsen, Pre-dose and at multiple time periods (up to 48 hours) after end of infusion, Part B: Change From Baseline in Exon-Skipping Levels at Week 28, Part B: Incidence of Adverse Events (AEs), Baseline up to Week 304, Part B: PK: Plasma Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusion, Part B: PK: Urine Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusion, Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay at Week 28

Related papers

No related papers found yet.

Interventions

DRUGVESLETEPLIRSEN(SRP-5051)

Eligibility

Sex/Gender

Male

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Part A: Incidence of Adverse Events (AEs), Baseline up to 75 weeks, Part B: Change From Baseline in Dystrophin Protein Level at Week 28	—

Secondary

Measure	Time frame
Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen, Pre-dose and at multiple time points (up to 32 hours) after end of infusion, Part A: PK: Urine Concentration of Vesleteplirsen, Pre-dose and at multiple time periods (up to 48 hours) after end of infusion, Part B: Change From Baseline in Exon-Skipping Levels at Week 28, Part B: Incidence of Adverse Events (AEs), Baseline up to Week 304, Part B: PK: Plasma Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusion, Part B: PK: Urine Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusion, Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay at Week 28	—

Measure

Time frame

Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen, Pre-dose and at multiple time points (up to 32 hours) after end of infusion, Part A: PK: Urine Concentration of Vesleteplirsen, Pre-dose and at multiple time periods (up to 48 hours) after end of infusion, Part B: Change From Baseline in Exon-Skipping Levels at Week 28, Part B: Incidence of Adverse Events (AEs), Baseline up to Week 304, Part B: PK: Plasma Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusion, Part B: PK: Urine Concentration of Vesleteplirsen, Part B predose and at multiple timepoints (up to 48 hours) after end of infusion, Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay at Week 28

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Countries

Belgium, Germany, Italy, Netherlands, Spain

Outcome results

None listed