Duchenne Muscular DystrophyNCT02592941
Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
A study testing Deflazacort in people with Duchenne Muscular Dystrophy, run by PTC Therapeutics at 42 sites in United States.
Trial summary
The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new drug application is under preparation and… Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Deflazacort
- How many people
- Not given
- Where
- 42 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 5 years and older
- All sexes
- Child, Adult, Older adult
Main Inclusion Criteria:
- Confirmed diagnosis of Duchenne muscular dystrophy
- The patient is ≥ 5 years old
- Current on all childhood vaccinations including the chicken pox vaccine
Main Exclusion Criteria:
- History or current medication condition that could affect safety or poses an additional risk
- Hypersensitivity or allergic reaction to steroids or their formulations
Trial locations and contacts
42 sites in 1 country.
42 sites in 1 country
United States42 sites
University of Michigan Health System
Ann Arbor, Michigan, 48109
Children's Healthcare of Atlanta- Scottish Rite
Atlanta, Georgia, 30324
Children's Hospital Colorado
Aurora, Colorado, 80045
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Boston Children's Hospital
Boston, Massachusetts, 02115
Neurosciences Institute, Neurology-Charlotte
Charlotte, North Carolina, 28207
University of Virginia Children's Hospital
Charlottesville, Virginia, 22903
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Rush University Medical Center
Chicago, Illinois, 60612
Shriner's Hospital for Children - Chicago
Chicago, Illinois, 60707
Wesley Neurology Clinic
Cordova, Tennessee, 38018
Children's Medical Center
Dallas, Texas, 75235
Wayne State University- Children's Hospital of Michigan
Detroit, Michigan, 48201
Cook Children's Medical Center
Fort Worth, Texas, 76104
University of Florida Clinical Research Center
Gainesville, Florida, 32610
Helen DeVos Children's Hospital Neurology Clinic
Grand Rapids, Michigan, 49503
Greenville Health System
Greenville, South Carolina, 29615
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Texas Children's Hospital
Houston, Texas, 77030
Riley Hospital for Children at IU Health
Indianapolis, Indiana, 46202
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
Las Vegas Clinic
Las Vegas, Nevada, 89145
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
UCLA Health
Los Angeles, California, 90095
Nicklaus Children's Hospital
Miami, Florida, 33155
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Children's Specialty Group
Norfolk, Virginia, 23510
Childrens Hospital of Orange County
Orange, California, 92868
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Oregon Health & Science University
Portland, Oregon, 97239
Shriner's Hospital for Children
Portland, Oregon, 97239
Virginia Commonwealth University
Richmond, Virginia, 23298
UC Davis Medical Center
Sacramento, California, 95817
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101
University of Texas Health Science Center - San Antonio
San Antonio, Texas, 78229
Seattle Children's Hospital
Seattle, Washington, 98105
Shriner's Hospital for Children - Tampa
Tampa, Florida, 33612
Carle Foundation Hospital
Urbana, Illinois, 61801
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Wake Forest School of Medicine
Winston-Salem, North Carolina, 27157
About this study
Official title: An Open Label, Expanded Access Protocol Intended to Provide Treatment With MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or Adults With Duchenne Muscular Dystrophy
The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new drug application is under preparation and review. Enrollment is open to all eligible patients.
Read the detailed description
Deflazacort will be supplied free of charge and shipped directly to patients or caregivers, as appropriate. Treating physicians will continue to follow their standard of care activities and procedures for management of DMD. Treating physicians participating in the expanded access program are required to collect/document any patient or caregiver reported safety events and report to the sponsor..
- 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 immediately to the active metabolite, 21 desacetyl-DFZ. The elimination of this metabolite is primarily via the urine in humans. Its potency is approximately 70 to 90% of prednisone and 6 mg of deflazacort has approximately the same anti-inflammatory potency as 5 mg of prednisolone or prednisone.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2015 (estimated)
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
- Expanded access
Sponsor and investigators
- Lead sponsor
- PTC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Parexel, Dohmen Life Science Services
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02592941
- Sponsor's protocol ID
- MP-104-CL-037
- Status last verified
- Feb 2018
- First submitted
- 29 Oct 2015
- Last updated
- 28 Feb 2018
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Expanded Access Protocol Intended to Provide Treatment With MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or Adults With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02592941
- Acronym / sponsor ID
- MP-104-CL-037
- Conditions
- Duchenne Muscular Dystrophy
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Parexel, Dohmen Life Science Services
- First posted
- 30 Oct 2015 (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.
The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new drug application is under preparation and review. Enrollment is open to all eligible patients.
Detailed description
Deflazacort will be supplied free of charge and shipped directly to patients or caregivers, as appropriate. Treating physicians will continue to follow their standard of care activities and procedures for management of DMD. Treating physicians participating in the expanded access program are required to collect/document any patient or caregiver reported safety events and report to the sponsor..
Study design
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
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 immediately to the active metabolite, 21 desacetyl-DFZ. The elimination of this metabolite is primarily via the urine in humans. Its potency is approximately 70 to 90% of prednisone and 6 mg of deflazacort has approximately the same anti-inflammatory potency as 5 mg of prednisolone or prednisone. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Feb 2018.
- www.accessdmd.comLink provided by the sponsor.
Related trials for Duchenne Muscular Dystrophy
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
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.
The record lists no contact. Use the official record to reach the sponsor.
42 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Deflazacort in people with Duchenne Muscular Dystrophy, run by PTC Therapeutics at 42 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?
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: status not confirmed, as of the sponsor's last update on 28 Feb 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 42 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.
Are results available for this study?
Results are not available in the source record.
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.
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 28 Feb 2018.