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Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

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Daiichi Sankyo

Status and phase

Completed
Phase 2
Phase 1

Conditions

Duchenne Muscular Dystrophy

Treatments

Drug: DS-5141b

Study type

Interventional

Funder types

Industry
Other

Identifiers

NCT02667483
DS5141-A-J101
153072 (Registry Identifier)

Details and patient eligibility

About

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.

Enrollment

8 patients

Sex

Male

Ages

5 to 10 years old

Volunteers

No Healthy Volunteers

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 <11 years.
  • Patients able to walk at least 325 meters in the 6-minutes walk test.
  • Glucocorticoid-naive patients, or patients who have used glucocorticoids for at least 6 months prior to enrollment in this study with no dose changes for at least 3 months prior to enrollment.

Exclusion criteria

  • A genetic mutation that can not be expected the expression of dystrophin protein by dystrophin gene exon 45 skipping.
  • A concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function.
  • Current or history of severe disorder.
  • Left ventricular ejection fraction (LEVF) <55%.
  • Corrected QT interval (QTc) >0.45 sec.

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

8 participants in 1 patient group

DS-5141b
Experimental group
Description:
DS-5141b, Subcutaneous injection Part 1: DS-5141b will be injected subcutaneously once a week for 2 weeks at the following dose levels. Dose escalation will be performed. DS-5141b will be administered at dose levels 1 and 3 in Cohort 1 and at dose levels 2 and 4 in Cohort 2. * Level 1: 0.1 mg/kg * Level 2: 0.5 mg/kg * Level 3: 2.0 mg/kg * Level 4: 6.0 mg/kg Part 2: Two doses of DS-5141b will be selected based on the results obtained in Part 1. Each selected dose will be administered subcutaneously once a week for 12 weeks. Part 2-Extension-2: Two doses, 2.0 mg/kg or 6.0 mg/kg, of DS-5141b will be administered subcutaneously once a week for 48 weeks.
Treatment:
Drug: DS-5141b

Trial documents
1

Trial contacts and locations

2

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Data sourced from clinicaltrials.gov

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