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

June 20, 2019 updated by: PTC Therapeutics

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Withdrawn

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 year to 6 months (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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 Names:
  • 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 Names:
  • 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.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Period 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time Frame: 52 weeks
52 weeks
Period 1 and 2: Change From Baseline in Vital Signs and Electrocardiogram (ECG) at Week 52
Time Frame: Baseline, Week 52
Baseline, Week 52
Period 1 and 2: Change From Baseline in the Child Behavior Checklist Score at Week 52
Time Frame: Baseline, Week 52
Baseline, Week 52
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
Baseline, Week 52
Period 1 and 2: Mean Change From Baseline in Height at Week 52
Time Frame: Baseline, Week 52
Baseline, Week 52
Period 1 and 2: Mean Change From Baseline in Body Weight at Week 52
Time Frame: Baseline, Week 52
Baseline, Week 52
Period 1 and 2: Mean Change From Baseline in Height Percentile for Age at Week 52
Time Frame: Baseline, Week 52
Baseline, Week 52
Period 1 and 2: Number of Participants With Clinically Significant Laboratory Tests
Time Frame: 52 weeks
52 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
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
Pre-dose, 0.25, 2, 4, and 6 hours post-dose at Baseline (Week 1) and Week 13
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
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
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
Pre-dose, 0.25, 2, 4, and 6 hours post-dose at Baseline (Week 1) and Week 13

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
PTC Therapeutics

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Francesco Bibbiani, MD, PTC Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 31, 2018

Primary Completion (Anticipated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 31, 2021

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 31, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 10, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 20, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 22, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 21, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 20, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • PTCEMF-GD-003

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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