Duchenne Muscular DystrophyNCT03433807
Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)
A study testing Idebenone in people with Duchenne Muscular Dystrophy, run by Santhera Pharmaceuticals at 12 sites in United States.
Trial summary
The primary objective of this Expanded Access Program is to provide idebenone as a treatment for eligible participants with Duchenne Muscular Dystrophy before it is commercially available in the United States (U.S.) for the indication of DMD. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Idebenone
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 12 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 8 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Documented diagnosis of DMD (severe dystrophinopathy) and clinical features consistent of typical DMD at diagnosis (i.e., documented delayed motor skills and muscle weakness by age 5 years) and who in the opinion of the Treating physician would benefit from treatment with idebenone. DMD should be confirmed by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e., absent or <5% of normal) on Western blot or immunostaining.
- Minimum 8 years old at Prescreening.
- PEF or FVC ≤80% and >25% of predicted value based on most recent assessment noted in the patient's medical record and subsequently confirmed at the Enrollment Visit.
- Able to understand program requirements and swallow program medication.
- Signed and dated Informed Consent Form (to be obtained at the Enrollment Visit from patient or parent/legal guardian (if applicable) prior to performing any program-specific procedures and dispensing idebenone to the patient).
Who cannot join
- Eligible for and able to participate in an ongoing clinical trial of idebenone.
- Is at high-risk of a fatal outcome from lung infection and/or advanced cardiomyopathy in the opinion of the Treating physician.
- Known moderate or severe impairment of hepatic function or severe impairment of renal function.
- Prior or ongoing medical condition or laboratory abnormality which in the Treating physician's opinion may put the patient at significant risk or may interfere significantly with the patient's participation in the program.
- Abuse of drugs or alcohol, which in Treating physician's opinion would interfere with the compliance to treatment.
- Known individual hypersensitivity to idebenone or to any of the ingredients/excipients of the program medication.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Kennedy Krieger
Baltimore, Maryland, 21287
Carolina's Healthcare System
Charlotte, North Carolina, 28207
University of Virginia Children's Hospital
Charlottesville, Virginia, 22904
Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Iowa
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
Yale New Haven Hospital
New Haven, Connecticut, 06510
Columbia University Pediatric Neuromuscular Center
New York, New York, 10032
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Phoenix Children's Hospital
Phoenix, Arizona, 85016
St. Luke's Rehabilitation Institute
Spokane, Washington, 99202
About this study
Official title: Expanded Access Protocol (EAP) of Idebenone in Patients With Duchenne Muscular Dystrophy
The primary objective of this Expanded Access Program is to provide idebenone as a treatment for eligible participants with Duchenne Muscular Dystrophy before it is commercially available in the United States (U.S.) for the indication of DMD.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Idebenone
- Keywords
- idebenone, DMD, Duchenne
- 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.
IdebenoneDrug
900 mg idebenone/day (2 tablets to be taken 3 times a day with meals)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Feb 2018
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
- Santhera Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03433807
- Sponsor's protocol ID
- SNT-EAP-002
- Status last verified
- Apr 2023
- First submitted
- 8 Feb 2018
- Last updated
- 21 Apr 2023
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Protocol (EAP) of Idebenone in Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT03433807
- Acronym / sponsor ID
- SNT-EAP-002
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- idebenone, DMD, Duchenne
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Santhera PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 15 Feb 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 Expanded Access Program is to provide idebenone as a treatment for eligible participants with Duchenne Muscular Dystrophy before it is commercially available in the United States (U.S.) for the indication of DMD.
Study design
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Documented diagnosis of DMD (severe dystrophinopathy) and clinical features consistent of typical DMD at diagnosis (i.e., documented delayed motor skills and muscle weakness by age 5 years) and who in the opinion of the Treating physician would benefit from treatment with idebenone. DMD should be confirmed by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e., absent or <5% of normal) on Western blot or immunostaining.
- Minimum 8 years old at Prescreening.
- PEF or FVC ≤80% and >25% of predicted value based on most recent assessment noted in the patient's medical record and subsequently confirmed at the Enrollment Visit.
- Able to understand program requirements and swallow program medication.
- Signed and dated Informed Consent Form (to be obtained at the Enrollment Visit from patient or parent/legal guardian (if applicable) prior to performing any program-specific procedures and dispensing idebenone to the patient).
Key exclusion criteria (6 listed)
- Eligible for and able to participate in an ongoing clinical trial of idebenone.
- Is at high-risk of a fatal outcome from lung infection and/or advanced cardiomyopathy in the opinion of the Treating physician.
- Known moderate or severe impairment of hepatic function or severe impairment of renal function.
- Prior or ongoing medical condition or laboratory abnormality which in the Treating physician's opinion may put the patient at significant risk or may interfere significantly with the patient's participation in the program.
- Abuse of drugs or alcohol, which in Treating physician's opinion would interfere with the compliance to treatment.
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 |
|---|---|---|
| Idebenone900 mg idebenone/day (2 tablets to be taken 3 times a day with meals) | 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 21 Apr 2023.
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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.
The record lists no contact. Use the official record to reach the sponsor.
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Idebenone in people with Duchenne Muscular Dystrophy, run by Santhera Pharmaceuticals at 12 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 5 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 21 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 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 21 Apr 2023.