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A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT03642145 · Status: WITHDRAWN · Phase: PHASE3 · Type: INTERVENTIONAL

Last updated 2019-06-21

No results posted yet for this study

Summary

The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males with DMD aged greater than or equal to (\>=) 2 to lesser than (\<) 5 years.

The study will comprise of 2 periods (Period 1: 52-week safety and pharmacokinetics \[PK\], and Period 2: 52-week extension). Participants will be randomized in a 1:1 ratio to one of 2 treatment arms: 0.9 mg/kg deflazacort, and 0.45 mg/kg of deflazacort. A historic control group (which should match the study population as closely as possible) will be used as a comparator to characterize the safety and tolerability of deflazacort.

Conditions

Interventions

DRUG

Deflazacort

Deflazacort tablets will be administered as per schedule and dose specified in respective arms.

Sponsors & Collaborators

Principal Investigators

  • Francesco Bibbiani, MD · PTC Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
2 Years
Max Age
4 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-10-31
Primary Completion
2021-07-31
Completion
2021-07-31
FDA Drug
Yes

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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 NCT03642145 on ClinicalTrials.gov