Duchenne Muscular DystrophyPhase 3PTCEMFNCT03642145
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
A Phase 3 study testing Deflazacort in people with Duchenne Muscular Dystrophy, run by PTC Therapeutics.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 4 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Deflazacort
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 31 Oct 2018to 31 Jul 2021
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 4 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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:
- Major renal or hepatic impairment
- Immunosuppression or other contraindications for corticosteroid treatment
- History of chronic systemic fungal or viral infections
Show all 13Show fewer
- Diabetes mellitus or significant glucose intolerance
- Idiopathic hypercalciuria
- 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.
About this study
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
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
- Duchenne Muscular Dystrophy
- Being tested
- Deflazacort
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeflazacortDrug
Also called Emflaza®
Deflazacort tablets will be administered as per schedule and dose specified in respective arms.
| Study group | Role | What they receive |
|---|---|---|
| Arm A: Deflazacort 0.9 mg/kg | Experimental | 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. Receives: Drug: Deflazacort |
| Arm B: Deflazacort 0.45 mg/kg | Experimental | 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. Receives: Drug: Deflazacort |
| Natural History Control Group | No intervention | 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 being measured
The primary outcome is the main question the study is designed to answer.
Period 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Measured: 52 weeks
Period 1 and 2: Change From Baseline in Vital Signs and Electrocardiogram (ECG) at Week 52
Measured: Baseline, Week 52
Period 1 and 2: Change From Baseline in the Child Behavior Checklist Score at Week 52
Measured: 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
Measured: Baseline, Week 52
Period 1 and 2: Mean Change From Baseline in Height at Week 52
Measured: Baseline, Week 52
Period 1 and 2: Mean Change From Baseline in Body Weight at Week 52
Measured: Baseline, Week 52
Period 1 and 2: Mean Change From Baseline in Height Percentile for Age at Week 52
Measured: Baseline, Week 52
Period 1 and 2: Number of Participants With Clinically Significant Laboratory Tests
Measured: 52 weeks
Secondary outcomes (4)
Period 1: Peak Plasma Concentration (Cmax) of Deflazacort
Measured: 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
Measured: 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
Measured: Pre-dose, 0.25, 2, 4, and 6 hours post-dose at Baseline (Week 1) and Week 13
Period 1: Clearance (CL) of Deflazacort
Measured: Pre-dose, 0.25, 2, 4, and 6 hours post-dose at Baseline (Week 1) and Week 13
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2018
- Study start31 Oct 2018
- Primary completion31 Jul 2021 (estimated)
- Study completion31 Jul 2021 (estimated)
Why it stopped: 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.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- PTC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Francesco Bibbiani, MD, Study director, PTC Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03642145
- Sponsor's protocol ID
- PTCEMF-GD-003
- Acronym
- PTCEMF
- Status last verified
- May 2019
- First submitted
- 10 Jul 2018
- Last updated
- 21 Jun 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03642145
- Acronym / sponsor ID
- PTCEMF / PTCEMF-GD-003
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: 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.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 31 Oct 2018 (actual)
- Primary completion
- 31 Jul 2021 (estimated)
- Study completion
- 31 Jul 2021 (estimated)
- First posted
- 22 Aug 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Arm A: Deflazacort 0.9 mg/kg | Experimental | Drug: DeflazacortParticipants 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. |
| Arm B: Deflazacort 0.45 mg/kg | Experimental | Drug: DeflazacortParticipants 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. |
| Natural History Control Group | No intervention | –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. |
Who may qualify
- Ages
- 2 to 4 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (13 listed)
- 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:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DeflazacortAlso: Emflaza®Deflazacort tablets will be administered as per schedule and dose specified in respective arms. | Drug | Arm A: Deflazacort 0.9 mg/kg, Arm B: Deflazacort 0.45 mg/kg |
Primary outcome
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 Vital Signs and Electrocardiogram (ECG) 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: 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: Mean Change From Baseline in Height 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
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
4 secondary outcomes
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: 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: 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
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
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jun 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Francesco Bibbiani, MD study director
PTC Therapeutics
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Deflazacort in people with Duchenne Muscular Dystrophy, run by PTC Therapeutics.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 21 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Period 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs) (time frame: 52 weeks). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jun 2019.