Duchenne Muscular DystrophyPhase 1NCT02251600
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
A Phase 1 study testing Deflazacort in people with Duchenne Muscular Dystrophy, run by PTC Therapeutics at 4 sites in United States.
Trial summary
Study to characterize the single-state and steady-state dosing of oral deflazacort in pediatric and adolescents subjects. Read more about this study
- Who is it for?
- Ages 4 to 16 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
- 24 participants
- Where
- 4 sitesUnited States
- Study dates
- Dec 2014to Oct 2015
- Phase
- Phase 1
- 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 4 to 16 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- In the opinion of the investigator, the subject is capable of understanding and complying with protocol requirements.
- The subject or, when applicable, the subject'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.
- If above the age of 7, the subject signs and dates a written, informed assent form (IAF) and any required privacy authorization prior to the initiation of any study procedures.
- The subject must have confirmed diagnosis of Duchenne Muscular Dystrophy defined as muscle biopsy and dystrophin analyses consistent with DMD or DNA mutation and analysis by PCR or Southern blot techniques to detect gene deletions as well as:
- onset of weakness before 5 years of age;
- proximal muscle weakness;
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- increased serum creatine kinase more than 10 times the upper limit of normal (ULN);
- The subject is male and aged 4 to 16 years, inclusive.
- The subject weighs at least 13 kg and has a Body Mass Index (BMI) of ≤ 40.
- Willingness and ability to comply with scheduled visits, oral drug administration, and study procedures including blood sample draws (total blood volume collected not to exceed 50 mL for the study duration or 25 mL on any single study day [≤ 3 mL/kg])
- The subject has a life expectancy of >1 year.
- Up to date on all childhood vaccinations, specifically varicella vaccine (chicken pox).
- Baseline health is judged to be stable based on medical history, physical examination, laboratory profiles, vital signs, or ECGs at screening, as deemed by the Investigator.
- Continuous non smoker who has not used nicotine containing products for at least 3 months prior to the first dose.
- The subject is able to take tablets.
Who cannot join
- The subject has received any investigational compound and/or has participated in another clinical study within 90 days prior to study treatment with the exception of observational cohort studies or non-interventional studies.
- The subject has received deflazacort within 30 days or previous discontinued deflazacort due to an intolerable reaction.
- The subject is an immediate family member, study site employee, or is in a dependent relationship with a study site employee who is involved in the conduct of this study (e.g. spouse, parent, child, sibling) or may consent under duress.
- Any significant finding on the Columbia suicide severity rating scale (C SSRS) for subjects (ages 12-16, inclusive), in the opinion of the PI, warrants exclusion from this study.
- The subject has, in the judgment of the investigator, clinically significant abnormal clinical chemistry laboratory parameters that may affect safety at Screening.
- The subject has, in the judgment of the investigator, a history or current medical condition that could affect safety including, but not limited to:
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- Major renal or hepatic impairment
- Immunosuppression or other contraindications for corticosteroid treatment
- History of chronic systemic fungal or viral infections
- Diabetes mellitus
- Idiopathic hypocalcuria
- Symptomatic cardiomyopathy at screening
- The subject has a history of hypersensitivity or allergic reaction to steroids or their formulations including, but not limited to lactose, sucrose, etc.
- Inability to take tablets as assessed by site investigator.
- Unable to refrain from or anticipates the use of:
- Any medications at least 4 hours before and after dosing on PK Days 1 and 8.
- Any vitamins, vitamins with minerals, and/or meal supplementation (e.g., Ensure, Boost, etc.) at least 4 hours before and after dosing on PK Days 1 and 8.
- Any drug, including prescription and non prescription medications, and herbal remedies known to be significant inhibitors of CYP 3A4 enzymes and/or P gp for 14 days prior to the first dose of study drug and throughout the study. Appropriate sources will be consulted by the PI or designee to confirm lack of pharmacokinetic/pharmacodynamic interaction with study drug. Acetaminophen may be permitted during the study (doses to be based upon appropriate age/weight ranges.
- Any drugs known to be significant inducers of CYP 3A4 enzymes and/or P gp for 28 days prior to the first dose of study drug and throughout the study. Appropriate sources will be consulted by the PI or designee to confirm lack of PK/pharmacodynamics interaction with study drug.
- Subject is mentally or legally incapacitated or has significant emotional problems at the time of screening visit or expected during the conduct of the study.
- History of any illness that, in the opinion of the PI, might confound the results of the study or poses an additional risk to the subject by their participation in the study.
- Positive urine drug or alcohol results at screening or check in.
- Positive urine cotinine at screening.
- Positive results at screening for HIV, HBsAg, or HCV.
- Hemoglobin level below the lower limit of normal at screening.
- Donation of blood or significant blood loss within 56 days prior to the first dose of study drug.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Lurie Children's Hospital
Chicago, Illinois, 60611
UCLA
Los Angeles, California, 90095
University of Rochester Medical Center
Rochester, New York, 14642
University of Utah
Salt Lake City, Utah, 84112
About this study
Official title: A Multi-center Study to Evaluate the Pharmacokinetics of 21-Desacetyldeflazacort and the Safety of Deflazacort After Oral Administration of Deflazacort Tablets to Children and Adolescent Subjects With Duchenne Muscular Dystrophy
Study to characterize the single-state and steady-state dosing of oral deflazacort in pediatric and adolescents subjects.
Read the detailed description
This is an open label, single period study in 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) with at least 12 subjects between the ages of 4-12 (children). Subjects taking maintenance corticosteroid therapy will be required to take their dose of corticosteroid 24 hours (± 2 hours) prior to the first dose of deflazacort on Day 1. Subjects receiving deflazacort as maintenance corticosteroid therapy must take their last dose at least 30 days prior to the first dose of deflazacort on Day 1. Concomitant corticosteroid therapy will be prohibited during the study while the subject is taking deflazacort. On Day 1, a single oral dose of deflazacort under fasting conditions will be administered in the CRU followed by blood sampling for plasma analysis of DFZ and 21-desacetyl-DFZ for 8 hours. Following the 8 hour PK sample on Day 1, subjects will be given medication to take at home for once-daily, morning dosing on Days 2 through 7 (+2 days). On Day 8 (+2 days), subjects will return for a PK sample predose and an 8th oral dose of deflazacort under fasting conditions which will be administered in the CRU followed by PK sampling for 8 hours.
Safety will be monitored throughout the study by repeated clinical and laboratory evaluations on Days 1 and 8 (+2 days). Subjects will be contacted via telephone approximately 7 days (± 1 day) following study drug administration on Day 8 (+2 days) for a follow-up assessment to determine if any adverse event (AE) has occurred since the last study visit. Subjects who terminate the study early will be contacted if the Principal Investigator (PI) deems necessary.
Subjects that complete this study or receive at least one dose of study medication will be eligible for an open-label extension study with deflazacort treatment conducted under a separate protocol.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Deflazacort
- Keywords
- Pediatric, Adolescent, 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 DFZ
Oral deflazacort administered once daily at 0.9 mg/kg for eight days
| Study group | Role | What they receive |
|---|---|---|
| Deflazacort | Experimental | This is an open label and single period study , dosed with 0.9mg/kg Deflazacort. Receives: Drug: Deflazacort |
What is being measured
The primary outcome is the main question the study is designed to answer.
The area under the plasma concentration time curve, from time 0 to the last measurable concentration non-zero, for single-state pharmacokinetics on Day 1 of deflazacort and 21-desacetyl-DFZ, the active metabolite
The area under the plasma concentration time curve, from time 0 to the last measurable concentration non-zero, for single-state pharmacokinetics on Day 1 of deflazacort and 21-desacetyl-DFZ, the active metabolite
Measured: Day 1, Day 8
The area under the plasma concentration versus time curve over the final dosing interval for steady state pharmacokinetics on Day 8 of deflazacort and 21-desacetyl-DFZ, the active metabolite
The area under the plasma concentration versus time curve over the final dosing interval for steady state pharmacokinetics on Day 8 of deflazacort and 21-desacetyl-DFZ, the active
Measured: Day 8
Secondary outcomes (1)
Number of participants with adverse events as a measure of safety and tolerability
To assess the safety and tolerability of single-dose and steady-state deflazacort in DMD subjects.
Measured: Day 1-8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2014 (estimated)
- Study startDec 2014
- Primary completionOct 2015
- Study completionOct 2015
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 1
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- PTC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Katherine Smith, MD, Study chair, Drug Safety Solutions/Celerion
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02251600
- Sponsor's protocol ID
- MP-104-CL-005
- Status last verified
- Aug 2017
- First submitted
- 22 Sep 2014
- Last updated
- 18 Aug 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center Study to Evaluate the Pharmacokinetics of 21-Desacetyldeflazacort and the Safety of Deflazacort After Oral Administration of Deflazacort Tablets to Children and Adolescent Subjects With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02251600
- Acronym / sponsor ID
- MP-104-CL-005
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Pediatric, Adolescent, Deflazacort
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Dec 2014
- Primary completion
- Oct 2015 (actual)
- Study completion
- Oct 2015 (actual)
- First posted
- 29 Sep 2014 (estimated)
- 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.
Study to characterize the single-state and steady-state dosing of oral deflazacort in pediatric and adolescents subjects.
Detailed description
This is an open label, single period study in 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) with at least 12 subjects between the ages of 4-12 (children). Subjects taking maintenance corticosteroid therapy will be required to take their dose of corticosteroid 24 hours (± 2 hours) prior to the first dose of deflazacort on Day 1. Subjects receiving deflazacort as maintenance corticosteroid therapy must take their last dose at least 30 days prior to the first dose of deflazacort on Day 1. Concomitant corticosteroid therapy will be prohibited during the study while the subject is taking deflazacort. On Day 1, a single oral dose of deflazacort under fasting conditions will be administered in the CRU followed by blood sampling for plasma analysis of DFZ and 21-desacetyl-DFZ for 8 hours. Following the 8 hour PK sample on Day 1, subjects will be given medication to take at home for once-daily, morning dosing on Days 2 through 7 (+2 days). On Day 8 (+2 days), subjects will return for a PK sample predose and an 8th oral dose of deflazacort under fasting conditions which will be administered in the CRU followed by PK sampling for 8 hours.
Safety will be monitored throughout the study by repeated clinical and laboratory evaluations on Days 1 and 8 (+2 days). Subjects will be contacted via telephone approximately 7 days (± 1 day) following study drug administration on Day 8 (+2 days) for a follow-up assessment to determine if any adverse event (AE) has occurred since the last study visit. Subjects who terminate the study early will be contacted if the Principal Investigator (PI) deems necessary.
Subjects that complete this study or receive at least one dose of study medication will be eligible for an open-label extension study with deflazacort treatment conducted under a separate protocol.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Deflazacort | Experimental | Drug: DeflazacortThis is an open label and single period study , dosed with 0.9mg/kg Deflazacort. |
Who may qualify
- Ages
- 4 to 16 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (15 listed)
- In the opinion of the investigator, the subject is capable of understanding and complying with protocol requirements.
- The subject or, when applicable, the subject'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.
- If above the age of 7, the subject signs and dates a written, informed assent form (IAF) and any required privacy authorization prior to the initiation of any study procedures.
- The subject must have confirmed diagnosis of Duchenne Muscular Dystrophy defined as muscle biopsy and dystrophin analyses consistent with DMD or DNA mutation and analysis by PCR or Southern blot techniques to detect gene deletions as well as:
- The subject is male and aged 4 to 16 years, inclusive.
Key exclusion criteria (26 listed)
- The subject has received any investigational compound and/or has participated in another clinical study within 90 days prior to study treatment with the exception of observational cohort studies or non-interventional studies.
- The subject has received deflazacort within 30 days or previous discontinued deflazacort due to an intolerable reaction.
- The subject is an immediate family member, study site employee, or is in a dependent relationship with a study site employee who is involved in the conduct of this study (e.g. spouse, parent, child, sibling) or may consent under duress.
- Any significant finding on the Columbia suicide severity rating scale (C SSRS) for subjects (ages 12-16, inclusive), in the opinion of the PI, warrants exclusion from this study.
- The subject has, in the judgment of the investigator, clinically significant abnormal clinical chemistry laboratory parameters that may affect safety at Screening.
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: DFZOral deflazacort administered once daily at 0.9 mg/kg for eight days | Drug | Deflazacort |
Primary outcome
The area under the plasma concentration time curve, from time 0 to the last measurable concentration non-zero, for single-state pharmacokinetics on Day 1 of deflazacort and 21-desacetyl-DFZ, the active metabolite
Time frame: Day 1, Day 8
The area under the plasma concentration time curve, from time 0 to the last measurable concentration non-zero, for single-state pharmacokinetics on Day 1 of deflazacort and 21-desacetyl-DFZ, the active metabolite
The area under the plasma concentration versus time curve over the final dosing interval for steady state pharmacokinetics on Day 8 of deflazacort and 21-desacetyl-DFZ, the active metabolite
Time frame: Day 8
The area under the plasma concentration versus time curve over the final dosing interval for steady state pharmacokinetics on Day 8 of deflazacort and 21-desacetyl-DFZ, the active
1 secondary outcome
Number of participants with adverse events as a measure of safety and tolerability
Time frame: Day 1-8
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Aug 2017.
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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.
Katherine Smith, MD study chair
Drug Safety Solutions/Celerion
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Deflazacort in people with Duchenne Muscular Dystrophy, run by PTC Therapeutics at 4 sites in United States.
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 15 inclusion and 26 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: finished, as of the sponsor's last update on 18 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
The area under the plasma concentration time curve, from time 0 to the last measurable concentration non-zero, for single-state pharmacokinetics on Day 1 of deflazacort and 21-desacetyl-DFZ, the active metabolite (time frame: Day 1, Day 8). The study lists 2 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 18 Aug 2017.