Phase 2
N=8
Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT02667483 ↗Enrolled (actual)
8
Serious AEs
12.5%
Results posted
Mar 2024
Primary outcome: Primary: Number of Participants Reporting at Least One Treatment-emergent Adverse Event (TEAE) In Participants With Duchenne Muscular Dystrophy — 3; 5 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- DS-5141b (Drug)
- Age
- Pediatric · 5+ yrs
- Sex
- Male
- Sponsor
- Daiichi Sankyo Co., Ltd.
- Primary completion
- Oct 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Reporting at Least One Treatment-emergent Adverse Event (TEAE) In Participants With Duchenne Muscular Dystrophy |
3; 5 | — |
| PRIMARY Pharmacokinetic Parameter Maximum Concentration (Cmax) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy |
1320; 5190 | — |
| PRIMARY Pharmacokinetic Parameter Area Under the Curve (AUC) Tau of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy |
23100; 102000 | — |
| PRIMARY Pharmacokinetic Parameter Time to Maximum Concentration (Tmax) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy |
1.98; 4.07 | — |
| PRIMARY Pharmacokinetic Parameter Half-life (T1/2) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Dystrophy |
32.4 | — |
| PRIMARY Mean Dystrophin Protein Expression in Muscle Tissue |
2.51; 1.86; 3.65; 2.43 | — |
| SECONDARY Number of Participants With Exon 45-skipped Dystrophin mRNA Expression in Muscle Tissue Posttreatment |
3; 5 | — |
Summary
This is a phase I/II study to evaluate the safety, tolerability, efficacy, and pharmacokinetic (PK) profile of DS-5141b in patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping and to determine the dosage for subsequent studies.
Eligibility Criteria
Inclusion Criteria
- Confirmation of out-of-frame deletion(s) that could be corrected by dystrophin gene exon 45 skipping.
- Intact muscles of adequate quality for biopsy to allow evaluation of the efficacy of the study drug.
- Boys aged from 5 years to 0.45 sec.
Data sourced from ClinicalTrials.gov (NCT02667483). 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.