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NCT Number: NCT02667483

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

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.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

5 year–10 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Kobe University Hospital, Hyōgo, Kobe-shi, Japan

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

DS-5141b

Drug

DS-5141b, Subcutaneous injection

Primary outcomes

  1. Number of Participants Reporting at Least One Treatment-emergent Adverse Event (TEAE) In Participants With Duchenne Muscular Dystrophy

    Time frame: 48 Weeks of Part 2-Extension-2

    A treatment-emergent adverse event (TEAE) is defined as an adverse event that emerges during treatment having been absent prior to treatment or reemerges during treatment or worsens in severity during treatment.

  2. Pharmacokinetic Parameter Maximum Concentration (Cmax) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy

    Time frame: Week 48 of Part 2-Extension-2

    Pharmacokinetic parameters were assessed using non-compartmental methods.

  3. Pharmacokinetic Parameter Area Under the Curve (AUC) Tau of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy

    Time frame: Week 48 of Part 2-Extension-2

    Pharmacokinetic parameters were assessed using non-compartmental methods.

  4. Pharmacokinetic Parameter Time to Maximum Concentration (Tmax) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Muscular Dystrophy

    Time frame: Week 48 of Part 2-Extension-2

    Pharmacokinetic parameters were assessed using non-compartmental methods.

  5. Pharmacokinetic Parameter Half-life (T1/2) of DS-5141a (Free Form of DS-5141b) in Participants With Duchenne Dystrophy

    Time frame: Week 48 of Part 2-Extension-2

    Pharmacokinetic parameters were assessed using non-compartmental methods.

  6. Mean Dystrophin Protein Expression in Muscle Tissue

    Time frame: Week 48 of Part 2-Extension-2

Secondary outcomes

  1. Number of Participants With Exon 45-skipped Dystrophin mRNA Expression in Muscle Tissue Posttreatment

    Time frame: Week 48 of Part 2-Extension-2

Sponsors and collaborators

Lead sponsor

Daiichi Sankyo Co., Ltd.

Industry

Collaborators

  • Daiichi Sankyo

Registry information

Official study title

Phase I/II Study of DS-5141b: Open-label Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Important dates

Study start
2015
Primary completion
2020
Study completion
2020
First posted
Jan 29, 2016
Registry last updated
Mar 7, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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