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A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy

NCT02036463 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL

Last updated 2015-02-18

No results posted yet for this study

Summary

Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disease for which no curative treatment has yet been identified, making it important to slow progression and improve the quality of life among affected boys and young men. Treatment with corticosteroids is standard of care for patients with DMD five years old and older, due to the robust observation that this intervention lengthens the interval prior to loss of ambulation but is associated with many side effects. This clinical trial will be conducted in the youngest age group able to receive corticosteroids orally and on whom study outcomes are measurable, ages 3 to 7 years. This is a randomized, double blinded, double masked, placebo-controlled clinical trial that will explore whether better synchronization of corticosteroid administration with the circadian rhythm will provide improved tolerability and at least comparable efficacy to current standards in which corticosteroids are always given in the morning. Furthermore, the trial provides a unique opportunity to rigorously evaluate corticosteroid effects in the young DMD patient, both for efficacy as compared to placebo and as a study of the impact of corticosteroid chronotherapy, or delayed release, on increased tolerability over standard therapy. The main hypothesis is that synchronization of the timing of corticosteroid dosing will improve medication tolerability in children, while maintaining (non-inferiority) the efficacy of corticosteroid. The study also offers a unique opportunity to measure several biomarkers as well as novel genetic modifiers that may further impact the response to corticosteroid in DMD.

Conditions

  • Duchenne Muscular Dystrophy (DMD)

Interventions

DRUG

Prednisone

DRUG

Placebo

Sponsors & Collaborators

  • Children's National Research Institute

    collaborator OTHER

  • Ann & Robert H Lurie Children's Hospital of Chicago

    lead OTHER

Principal Investigators

  • Nancy Kuntz, MD · Ann & Robert H Lurie Children's Hospital of Chicago

Study Design

Allocation
RANDOMIZED
Purpose
SUPPORTIVE_CARE
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
3 Years
Max Age
6 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-11-30
Primary Completion
2015-02-28
Completion
2015-02-28

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02036463 on ClinicalTrials.gov