Muscular Dystrophy, DuchennePhase 2NCT00033813
KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy
A Phase 2 study testing Oxatomide (tinset) in people with Muscular Dystrophy, Duchenne, run by Cooperative International Neuromuscular Research Group at 1 site in United States.
Trial summary
This study will help to determine the safety and efficacy of the mast cell stabilizer Oxatomide as a treatment for Duchenne muscular dystrophy (DMD). Boys with DMD who are enrolled in this study will should not have taken steroids to treat DMD for at least twelve months, and should not have taken any nutritional… Read more about this study
- Who is it for?
- Ages 5 to 10 years, men only
- Conditions the study is for
- Muscular Dystrophy, Duchenne
- What is being tested?
- Oxatomide (tinset)
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2002to Dec 2006
- Phase
- Phase 2
- 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 to 10 years
- Men only
- No healthy volunteers
- Child
Subject Inclusion Criteria
- 5 to 10 years of age
- ambulatory
- diagnosis of DMD confirmed by at least one of the following:
- Positive x-linked family history of DMD in older male relatives (onset by 5 years, wheelchair bound by 12 years), or;
- Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, or;
- Gene deletion test positive (missing one or more exons) in the central rod domain (exons 25-60) of dystrophin, where reading frame can be predicted as 'out of frame', and clinical picture consistent with typical DMD.
- glucocorticosteroid-naive (i.e. has not been treated with prednisone or deflazacort within the past year)
- Evidence of muscle weakness by MRC score or clinical functional evaluation
- QMT biceps score variability no greater than 10% between screening visits
Subject Exclusion Criteria
- Failure to achieve one or more of the inclusion criteria listed above
- Inability to suitably cooperate with strength assessments
- Symptomatic DMD carrier
- Use of oxatomide (or other anti-histamine drugs) within the last 6 months for DMD or any other disease
- Use of creatine monohydrate or glutamine within the last 6 months
- Use of carnitine, Coenzyme Q10, other amino acids or any herbal medications within the last 3 months
- History of symptomatic cardiomyopathy
- History of impairment of hepatic function
- History of significant concomitant illness or significant impairment of renal function.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
This study will help to determine the safety and efficacy of the mast cell stabilizer Oxatomide as a treatment for Duchenne muscular dystrophy (DMD). Boys with DMD who are enrolled in this study will should not have taken steroids to treat DMD for at least twelve months, and should not have taken any nutritional supplements for at least three months. Subjects will complete a two screening visits within a one-week period, and if enrolled will then have their strength tested monthly for three months before beginning therapy with Oxatomide. Once Oxatomide therapy is started, participants will have their strength tested monthly for six months. Following the six month treatment period, participants will be given the option to remain on Oxatomide until the study is completed.
- Conditions
- Muscular Dystrophy, Duchenne
- Being tested
- Oxatomide (tinset)
- Keywords
- Duchenne, muscular, dystrophy, controlled clinical trial, CINRG
- 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.
Oxatomide (tinset)Drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2002 (estimated)
- Study startJan 2002
- Primary completionDec 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Dystrophy, DuchenneStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants
Sponsor and investigators
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Sponsor type
- Research network
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00033813
- Sponsor's protocol ID
- KUL0401
- Status last verified
- Mar 2014
- First submitted
- 10 Apr 2002
- Last updated
- 10 Mar 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT00033813
- Acronym / sponsor ID
- KUL0401
- Conditions
- Muscular Dystrophy, Duchenne
- Keywords
- Duchenne, muscular, dystrophy, controlled clinical trial, CINRG
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants
- Lead sponsor
- Cooperative International Neuromuscular Research GroupResearch network
- Study start
- Jan 2002
- Primary completion
- Dec 2006 (actual)
- First posted
- 11 Apr 2002 (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 study will help to determine the safety and efficacy of the mast cell stabilizer Oxatomide as a treatment for Duchenne muscular dystrophy (DMD). Boys with DMD who are enrolled in this study will should not have taken steroids to treat DMD for at least twelve months, and should not have taken any nutritional supplements for at least three months. Subjects will complete a two screening visits within a one-week period, and if enrolled will then have their strength tested monthly for three months before beginning therapy with Oxatomide. Once Oxatomide therapy is started, participants will have their strength tested monthly for six months. Following the six month treatment period, participants will be given the option to remain on Oxatomide until the study is completed.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 5 to 10 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
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 |
|---|---|---|
| Oxatomide (tinset) | Drug | – |
Primary outcome
Not reported in the source record.
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 10 Mar 2014.
- The Cooperative International Neuromuscular Research GroupLink provided by the sponsor.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Oxatomide (tinset) in people with Muscular Dystrophy, Duchenne, run by Cooperative International Neuromuscular Research Group at 1 site 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: finished, as of the sponsor's last update on 10 Mar 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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.
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 10 Mar 2014.