A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy
Status:
Withdrawn
Trial end date:
2015-02-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
Ann & Robert H Lurie Children's Hospital of Chicago
Collaborators:
Children's National Research Institute Children's Research Institute