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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03642145
• Date of registration: 10/07/2018
• Prospective Registration: Yes
• Primary sponsor: PTC Therapeutics
• Public title: A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) PTCEMF
• Scientific title: A 52-Week Phase 3B Randomized Open-Label Study Evaluating the Safety and Pharmacokinetics of Emflaza® (Deflazacort) Compared to a Comparable Natural History Control Group in Males Aged =2 to <5 Years With Duchenne Muscular Dystrophy (DMD) Followed by a 52-Week Extension Period
• Date of first enrolment: October 31, 2018
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT03642145
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

United States

Contacts

• Name: Francesco Bibbiani, MD
• Affiliation: PTC Therapeutics

Key inclusion & exclusion criteria

Inclusion Criteria:

- In the opinion of the Investigator, the participant and parent(s)/caregiver are
capable of complying with protocol requirements.

- The participant's legally acceptable representative signs and dates a written informed
consent form and any required privacy authorization prior to the initiation of any
study procedures.

- The participant must have a diagnosis of DMD defined by genetic or biopsy confirmation
of DMD or have documented, increased serum creatine kinase more than 40 times the
upper limit of normal (ULN) and shown phenotypic signs of DMD.

- The participant weighs between 11 kilograms (kg) and 50 kg at screening visit.

- Ability to comply with scheduled visits, oral drug administration, and study
procedures.

- The participant is current on childhood vaccinations according to the Center for
Disease Control (CDC) recommended immunizations for children from birth through 6
years old. Note: The investigator should discuss timing of receipt of the varicella
vaccine with the caregiver prior to initiation of chronic steroid treatment.
Administration of live or live attenuated vaccines is not recommended in participants
receiving immunosuppressive doses of corticosteroids. Participants whose caregivers
decline vaccinations as a matter of personal belief may be included.

- Baseline health is judged to be stable based on medical history, physical examination,
laboratory profiles, and vital signs at screening, as deemed by the Investigator.

- The participant is able to ingest the oral tablets either whole or crushed.

Exclusion Criteria:

- The participant has received 4 weeks or more of continuous corticosteroid therapy
within 3 months of study screening visit.

- The participant has, in the judgment of the Investigator, clinically significant
abnormal clinical laboratory parameters at screening or baseline that may affect
safety.

- The participant has, in the judgment of the Investigator, a history or current medical
condition that could affect safety including, but not limited to:

1. Major renal or hepatic impairment

2. Immunosuppression or other contraindications for corticosteroid treatment

3. History of chronic systemic fungal or viral infections

4. Diabetes mellitus or significant glucose intolerance

5. Idiopathic hypercalciuria

6. Symptomatic cardiomyopathy Note: Elective surgeries can be discussed with medical
monitor.

- The participant has a history of hypersensitivity or allergic reaction to steroids or
their formulations including, but not limited to lactose, sucrose, etc.

- The participant has received any drug, including prescription and non-prescription
medications, and herbal remedies known to be significant inhibitors and/or inducers of
cytochrome P3A4 (CYP3A4) enzymes and/or P glycoprotein (P-gp) 14 days prior to the
first dose of study drug.

- The participant has an indication that requires long-term use of strong CYP3A4
inhibitors and/or inducers that would interfere with the pharmacokinetics of
deflazacort.

- The participant has received any investigational compound and/or has participated in
another clinical study within 30 days prior to study treatment with the exception of
observational cohort studies or non-interventional studies.



Age minimum: 2 Years
Age maximum: 4 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Deflazacort

Primary Outcome(s)

Period 1 and 2: Mean Change From Baseline in Height Percentile for Age at Week 52 [Time Frame: Baseline, Week 52]

Period 1 and 2: Number of Participants With Clinically Significant Laboratory Tests [Time Frame: 52 weeks]

Period 1 and 2: Mean Change From Baseline in Height at Week 52 [Time Frame: Baseline, Week 52]

Period 1 and 2: Change From Baseline in Vital Signs and Electrocardiogram (ECG) at Week 52 [Time Frame: Baseline, Week 52]

Period 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs) [Time Frame: 52 weeks]

Period 1 and 2: Change From Baseline in the Normalized Measure of Bone Density Change (Z-score) for the Dual Energy X-ray Absorptiometry (DEXA) at Week 52 [Time Frame: Baseline, Week 52]

Period 1 and 2: Change From Baseline in the Child Behavior Checklist Score at Week 52 [Time Frame: Baseline, Week 52]

Period 1 and 2: Mean Change From Baseline in Body Weight at Week 52 [Time Frame: Baseline, Week 52]

Secondary Outcome(s)

Period 1: Area Under the Curve (AUC) of Deflazacort [Time Frame: Pre-dose, 0.25, 2, 4, and 6 hours post-dose at Baseline (Week 1) and Week 13]

Period 1: Clearance (CL) of Deflazacort [Time Frame: Pre-dose, 0.25, 2, 4, and 6 hours post-dose at Baseline (Week 1) and Week 13]

Period 1: Peak Plasma Concentration (Cmax) of Deflazacort [Time Frame: Pre-dose, 0.25, 2, 4, and 6 hours post-dose at Baseline (Week 1) and Week 13]

Period 1: Volume of Distribution (Vd) of Deflazacort [Time Frame: Pre-dose, 0.25, 2, 4, and 6 hours post-dose at Baseline (Week 1) and Week 13]

Secondary ID(s)

PTCEMF-GD-003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors