Duchenne Muscular DystrophyPhase 1NCT02295748
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
A Phase 1 study testing Deflazacort in people with Duchenne Muscular Dystrophy, run by PTC Therapeutics at 4 sites in United States.
Trial summary
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study. Read more about this study
- Who is it for?
- Ages 4 years and older, 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 24 Aug 2017
- 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 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject is capable of understanding and complying with protocol requirements.
- 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. Subjects under age 7 at the time of study entry who turn age 7 will sign and date a written informed assent form (IAF) at the visit following their 7th birthday, if required by the site's IRB.
- Subject participated in and received at least one dose of study medication in the MP-104-CL-005 protocol.
- The subject must have confirmed diagnosis of Duchenne Muscular Dystrophy defined as:
- onset of weakness before 5 years of age;
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- proximal muscle weakness;
- increased serum creatine kinase more than 10 times the upper limit of normal (ULN);
- muscle biopsy and dystrophin analyses consistent with DMD or DNA mutation and analysis by PCR or Southern blot techniques to detect gene deletions.
- The subject weighs at least 13 kg and has a body mass index (BMI) of ≤ 40 kg/m2.
- Willingness and ability to comply with scheduled visits, oral drug administration, and study procedures including blood sample draws for safety labs.
- Up to date on all childhood vaccinations, including 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 MP-104-CL-005, observational cohort studies or non-interventional studies.
- 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, clinically significant abnormal clinical chemistry laboratory parameters that may affect safety at Day 0.
- The subject 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
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- Immunosuppression or other contraindications for corticosteroid treatment
- History of chronic systemic fungal or viral infections
- Diabetes mellitus
- Idiopathic hypocalciuria
- Symptomatic cardiomyopathy at Day 0
- 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.
- 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 Day 0.
- Hemoglobin level below the lower limit of normal at Day 0.
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
Rochester, New York, 14642
University of Utah
Salt Lake City, Utah, 84112
About this study
Official title: An Open-Label, Multi-Center, Long-Term Extension Study to Evaluate the Safety and Tolerability of Orally Administrated Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
Read the detailed description
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
Subjects may have received up to 8 (+2 days) doses of deflazacort during the MP-104-CL-005 PK study, at which point treatment will be continued at the same dose and frequency until such time that deflazacort becomes commercially available or the study is terminated. Concomitant corticosteroid therapy will be prohibited during the study while the subject is taking deflazacort.
Subjects will be given medication to take at home for once-daily, morning dosing following the 8 hour PK sample on Day 8 (+2 days) of the MP-104-CL-005 PK study. Subjects will begin dosing at home the day following Day 8 (+2 days) of the MP-104-CL-005 PK study.
Safety will be monitored throughout the study by repeated clinical and laboratory evaluations every 3 months for the first year, and then every 3 or 6 months, depending on subjects' disease status.
Subjects will be contacted via telephone between study visits to determine if any adverse events (AE) have occurred since the last study visit. Subjects who terminate the study early will be contacted if the Principal Investigator (PI) deems necessary.
Treatment will be continued for a maximum of three years or until such time that deflazacort becomes commercially available or the study is terminated.
- 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 DFZ
Deflazacort, a glucocorticoid with anti-inflammatory and immunosuppressive effects, is used in treating a variety of diseases. Pharmacologically it is an inactive pro-drug which is metabolized rapidly to the active drug 21-desacetyldeflazacort.
| Study group | Role | What they receive |
|---|---|---|
| Deflazacort | Experimental | 0.9 mg/kg oral deflazacort (between 2 to 12 x 6mg tablets based on body weight) will be administered daily. Receives: Drug: Deflazacort |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number, frequency, and severity of adverse events
Long-term safety and tolerability will be characterized by the number, frequency, and severity of adverse events from Day 1 through End of Study or Early Termination.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2014 (estimated)
- Study startDec 2014
- Primary completion24 Aug 2017
- Study completion24 Aug 2017
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
- Treatment
- 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
- Marathon Bioscience Center, Study director, Marathon Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02295748
- Sponsor's protocol ID
- MP-104-CL-022OLE
- Status last verified
- Jan 2017
- First submitted
- 18 Nov 2014
- Last updated
- 8 Dec 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Multi-Center, Long-Term Extension Study to Evaluate the Safety and Tolerability of Orally Administrated Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02295748
- Acronym / sponsor ID
- MP-104-CL-022OLE
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Dec 2014 (actual)
- Primary completion
- 24 Aug 2017 (actual)
- Study completion
- 24 Aug 2017 (actual)
- First posted
- 20 Nov 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.
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
Detailed description
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
Subjects may have received up to 8 (+2 days) doses of deflazacort during the MP-104-CL-005 PK study, at which point treatment will be continued at the same dose and frequency until such time that deflazacort becomes commercially available or the study is terminated. Concomitant corticosteroid therapy will be prohibited during the study while the subject is taking deflazacort.
Subjects will be given medication to take at home for once-daily, morning dosing following the 8 hour PK sample on Day 8 (+2 days) of the MP-104-CL-005 PK study. Subjects will begin dosing at home the day following Day 8 (+2 days) of the MP-104-CL-005 PK study.
Safety will be monitored throughout the study by repeated clinical and laboratory evaluations every 3 months for the first year, and then every 3 or 6 months, depending on subjects' disease status.
Subjects will be contacted via telephone between study visits to determine if any adverse events (AE) have occurred since the last study visit. Subjects who terminate the study early will be contacted if the Principal Investigator (PI) deems necessary.
Treatment will be continued for a maximum of three years or until such time that deflazacort becomes commercially available or the study is terminated.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Deflazacort | Experimental | Drug: Deflazacort0.9 mg/kg oral deflazacort (between 2 to 12 x 6mg tablets based on body weight) will be administered daily. |
Who may qualify
- Ages
- 4 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (15 listed)
- Subject is capable of understanding and complying with protocol requirements.
- 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. Subjects under age 7 at the time of study entry who turn age 7 will sign and date a written informed assent form (IAF) at the visit following their 7th birthday, if required by the site's IRB.
- Subject participated in and received at least one dose of study medication in the MP-104-CL-005 protocol.
- The subject must have confirmed diagnosis of Duchenne Muscular Dystrophy defined as:
Key exclusion criteria (17 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 MP-104-CL-005, observational cohort studies or non-interventional studies.
- 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, clinically significant abnormal clinical chemistry laboratory parameters that may affect safety at Day 0.
- The subject has, in the judgment of the Investigator, a history or current medical condition that could affect safety including, but not limited to:
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: DFZDeflazacort, a glucocorticoid with anti-inflammatory and immunosuppressive effects, is used in treating a variety of diseases. Pharmacologically it is an inactive pro-drug which is metabolized rapidly to the active drug 21-desacetyldeflazacort. | Drug | Deflazacort |
Primary outcome
Number, frequency, and severity of adverse events
Time frame: 3 years
Long-term safety and tolerability will be characterized by the number, frequency, and severity of adverse events from Day 1 through End of Study or Early Termination.
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 8 Dec 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.
Marathon Bioscience Center study director
Marathon Pharmaceuticals
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 17 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 8 Dec 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?
Number, frequency, and severity of adverse events (time frame: 3 years). 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 8 Dec 2017.