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

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ClinicalTrials.gov Identifier: NCT03642145
Recruitment Status : Withdrawn (Study is no longer necessary given the safety and efficacy of emflaza in this age range has already been established after review of already available data.)
First Posted : August 22, 2018
Last Update Posted : June 21, 2019
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

Brief 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.


Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Deflazacort Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : October 31, 2018
Estimated Primary Completion Date : July 31, 2021
Estimated Study Completion Date : July 31, 2021


Arm Intervention/treatment
Experimental: Arm A: Deflazacort 0.9 mg/kg
Participants will receive approximately 0.9 mg/kg deflazacort once daily orally for 52 weeks in Period 1 and for 52 weeks in Period 2. The target dose could be varied +/- 20 percent (%) depending upon the available tablet strengths and change in participant's weight.
Drug: Deflazacort
Deflazacort tablets will be administered as per schedule and dose specified in respective arms.
Other Name: Emflaza®

Experimental: Arm B: Deflazacort 0.45 mg/kg
Participants will receive approximately 0.45 mg/kg deflazacort once daily orally for 52 weeks in Period 1. Participants will either continue to receive 0.45 mg/kg deflazacort or escalated dose of deflazacort (0.9 mg/kg) once daily orally in Period 2 at the investigator's discretion and in consultation with the caregiver. The target dose could be varied +/- 20% depending upon the available tablet strengths and change in participant's weight.
Drug: Deflazacort
Deflazacort tablets will be administered as per schedule and dose specified in respective arms.
Other Name: Emflaza®

No Intervention: Natural History Control Group
Control participants matching to the study population as closely as possible, will be used as a comparator to characterize the safety and tolerability of deflazacort.



Primary Outcome Measures :
  1. Period 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs) [ Time Frame: 52 weeks ]
  2. Period 1 and 2: Change From Baseline in Vital Signs and Electrocardiogram (ECG) at Week 52 [ Time Frame: Baseline, Week 52 ]
  3. Period 1 and 2: Change From Baseline in the Child Behavior Checklist Score at Week 52 [ Time Frame: Baseline, Week 52 ]
  4. 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 ]
  5. Period 1 and 2: Mean Change From Baseline in Height at Week 52 [ Time Frame: Baseline, Week 52 ]
  6. Period 1 and 2: Mean Change From Baseline in Body Weight at Week 52 [ Time Frame: Baseline, Week 52 ]
  7. Period 1 and 2: Mean Change From Baseline in Height Percentile for Age at Week 52 [ Time Frame: Baseline, Week 52 ]
  8. Period 1 and 2: Number of Participants With Clinically Significant Laboratory Tests [ Time Frame: 52 weeks ]

Secondary Outcome Measures :
  1. 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 ]
  2. 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 ]
  3. 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 ]
  4. 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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 4 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03642145


Sponsors and Collaborators
PTC Therapeutics
Investigators
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Study Director: Francesco Bibbiani, MD PTC Therapeutics

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Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT03642145     History of Changes
Other Study ID Numbers: PTCEMF-GD-003
First Posted: August 22, 2018    Key Record Dates
Last Update Posted: June 21, 2019
Last Verified: May 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Deflazacort
Anti-Inflammatory Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs