Duchenne Muscular DystrophyPhase 3NCT00110669
High-dose Prednisone in Duchenne Muscular Dystrophy
A Phase 3 study testing Prednisone in people with Duchenne Muscular Dystrophy, run by Cooperative International Neuromuscular Research Group at 1 site in United States.
Trial summary
This study will help to determine whether a high-dose weekly course of prednisone therapy is safer than and at least as effective as daily dose therapy for people with Duchenne muscular dystrophy (DMD). Read more about this study
- Who is it for?
- Ages 4 to 10 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Prednisone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2004to Feb 2008
- Phase
- Phase 3
- 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 10 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 4 to 10 years of age
- Ambulant
- Confirmed DMD Diagnosis
- Steroid naive
- Evidence of muscle weakness by MRC score or clinical functional evaluation
- Ability to provide reproducible QMT bicep score
Who cannot join
- History of significant concomitant illness or significant impairment of renal or hepatic function, or other contraindication to steroid therapy
- Symptomatic DMD carrier
- Positive PPD
- Lack of prior exposure to chickenpox or immunization
- Use of carnitine, glutamine, Coenzyme Q10, other amino acids or any herbal medications within the last 3 months
- History of symptomatic cardiomyopathy
- Prior attainment of quota for the age group in which the patient belongs
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
Official title: A Randomized Study of Daily vs. High-dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy
This study will help to determine whether a high-dose weekly course of prednisone therapy is safer than and at least as effective as daily dose therapy for people with Duchenne muscular dystrophy (DMD). Boys who are enrolled in this study should not have taken carnitine, other amino acids, creatine, glutamine, Coenzyme Q10 or any herbal medicines within the last three months. There will be a two-visit screening to take place in one week to ensure a reproducible manual muscle test. The subject will then be randomized and put into either the daily or weekly regimen. The duration of the study is twelve 28-day treatment cycles (approximately 12 months) with follow-up visits at month one, three and then every three months.
Read the detailed description
Duchenne muscular dystrophy (DMD) is the most common lethal inherited disorder worldwide. Despite the exponential increase in our understanding of the disorder since the discovery and characterization of the causative gene and its product dystrophin in 1987, current therapeutic management remains largely supportive. Awaiting a final genetic cure to be available in the future, further investments in developing better drug therapies for DMD remain important. The effect of a high dose prednisone regimen will be evaluated in comparison to a daily dose regimen in a multi-center, randomized, double-blind placebo-controlled 4-arm study. Ambulant children aged 4-10 years with an established DMD diagnosis will be studied. Patients will undergo 2 screening evaluations within 1 week. Patients will be randomized into treatment groups on the second screening visit, followed by a 12-month treatment period. During the treatment period, patients will be evaluated at monthly intervals. The primary endpoints are percentage change in average muscle strength score and QMT performance for specific muscle groups. Secondary endpoints include timed function tests, functional grades for arms and legs, and pulmonary function tests.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Prednisone
- Keywords
- Muscular dystrophy, Duchenne and Beckers, Beckers Muscular dystrophy
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PrednisoneDrug
Prednisone and dummy preparations for this study will be obtained from Frank's Pharmacy in Ocala, FL and will be supplied as a tablet containing 2.5mg, 5mg, 10mg, 20mg or 50mg Prednisone. Inactive "dummy" pills of similar look/taste will be supplied to maintain blinding.
| Study group | Role | What they receive |
|---|---|---|
| High Dose Prednisone | Active comparator | Subjects who are randomized to the high-dose prednisone arm of the study will receive the following starting dose:
•Prednisone at 10.0 mg/kg/wk (divided into two doses given on Saturday and Sunday) Receives: Drug: Prednisone |
| Daily Prednisone | Active comparator | Subjects who are randomized to the daily prednisone arm of the study will receive the following starting dose:
•Prednisone at 0.75 mg/kg/d Receives: Drug: Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quantitative muscle strength will be measured using the CINRG Quantitative Measurement System (CQMS)
Measured: February 2008
Primary strength endpoints will be quantitative myometry (QMT) scores of the upper and lower extremities, consisting of paired flexor/extensor groups.
Measured: February 2008
Secondary outcomes (2)
Secondary strength endpoints will include individual QMT scores of elbow and knee flexors and extensors and hand grip, manual muscle testing scores, which will be measured using the Medical Research Council's (MRC) muscle strength scoring method.
Measured: February 2008
Side-effect profiles will assessed by monitoring side-effects, including differences in growth (height and weight), calculated weight/height ratio, bone density, cataract formation, blood glucose, blood pressure and behavioral changes.
Measured: February 2008
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2005 (estimated)
- Study startJan 2004
- Primary completionDec 2007
- Study completionFeb 2008
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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Sponsor type
- Research network
- Investigators
- Diana Escolar, MD, Study chair, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00110669
- Sponsor's protocol ID
- CNMC0601
- Status last verified
- Oct 2011
- First submitted
- 12 May 2005
- Last updated
- 27 Oct 2011
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Study of Daily vs. High-dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT00110669
- Acronym / sponsor ID
- CNMC0601
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Muscular dystrophy, Duchenne and Beckers, Beckers Muscular dystrophy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Cooperative International Neuromuscular Research GroupResearch network
- Study start
- Jan 2004
- Primary completion
- Dec 2007 (actual)
- Study completion
- Feb 2008 (actual)
- First posted
- 13 May 2005 (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 whether a high-dose weekly course of prednisone therapy is safer than and at least as effective as daily dose therapy for people with Duchenne muscular dystrophy (DMD). Boys who are enrolled in this study should not have taken carnitine, other amino acids, creatine, glutamine, Coenzyme Q10 or any herbal medicines within the last three months. There will be a two-visit screening to take place in one week to ensure a reproducible manual muscle test. The subject will then be randomized and put into either the daily or weekly regimen. The duration of the study is twelve 28-day treatment cycles (approximately 12 months) with follow-up visits at month one, three and then every three months.
Detailed description
Duchenne muscular dystrophy (DMD) is the most common lethal inherited disorder worldwide. Despite the exponential increase in our understanding of the disorder since the discovery and characterization of the causative gene and its product dystrophin in 1987, current therapeutic management remains largely supportive. Awaiting a final genetic cure to be available in the future, further investments in developing better drug therapies for DMD remain important. The effect of a high dose prednisone regimen will be evaluated in comparison to a daily dose regimen in a multi-center, randomized, double-blind placebo-controlled 4-arm study. Ambulant children aged 4-10 years with an established DMD diagnosis will be studied. Patients will undergo 2 screening evaluations within 1 week. Patients will be randomized into treatment groups on the second screening visit, followed by a 12-month treatment period. During the treatment period, patients will be evaluated at monthly intervals. The primary endpoints are percentage change in average muscle strength score and QMT performance for specific muscle groups. Secondary endpoints include timed function tests, functional grades for arms and legs, and pulmonary function tests.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| High Dose Prednisone | Active comparator | Drug: PrednisoneSubjects who are randomized to the high-dose prednisone arm of the study will receive the following starting dose: •Prednisone at 10.0 mg/kg/wk (divided into two doses given on Saturday and Sunday) |
| Daily Prednisone | Active comparator | Drug: PrednisoneSubjects who are randomized to the daily prednisone arm of the study will receive the following starting dose: •Prednisone at 0.75 mg/kg/d |
Who may qualify
- Ages
- 4 to 10 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 4 to 10 years of age
- Ambulant
- Confirmed DMD Diagnosis
- Steroid naive
- Evidence of muscle weakness by MRC score or clinical functional evaluation
Key exclusion criteria (7 listed)
- History of significant concomitant illness or significant impairment of renal or hepatic function, or other contraindication to steroid therapy
- Symptomatic DMD carrier
- Positive PPD
- Lack of prior exposure to chickenpox or immunization
- Use of carnitine, glutamine, Coenzyme Q10, other amino acids or any herbal medications within the last 3 months
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 |
|---|---|---|
| PrednisonePrednisone and dummy preparations for this study will be obtained from Frank's Pharmacy in Ocala, FL and will be supplied as a tablet containing 2.5mg, 5mg, 10mg, 20mg or 50mg Prednisone. Inactive "dummy" pills of similar look/taste will be supplied to maintain blinding. | Drug | Daily Prednisone, High Dose Prednisone |
Primary outcome
Quantitative muscle strength will be measured using the CINRG Quantitative Measurement System (CQMS)
Time frame: February 2008
Primary strength endpoints will be quantitative myometry (QMT) scores of the upper and lower extremities, consisting of paired flexor/extensor groups.
Time frame: February 2008
2 secondary outcomes
Secondary strength endpoints will include individual QMT scores of elbow and knee flexors and extensors and hand grip, manual muscle testing scores, which will be measured using the Medical Research Council's (MRC) muscle strength scoring method.
Time frame: February 2008
Side-effect profiles will assessed by monitoring side-effects, including differences in growth (height and weight), calculated weight/height ratio, bone density, cataract formation, blood glucose, blood pressure and behavioral changes.
Time frame: February 2008
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 27 Oct 2011.
- CINRG public websiteLink 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.
Diana Escolar, MD study chair
Children's National Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Prednisone in people with Duchenne Muscular Dystrophy, 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?
The sponsor lists 6 inclusion and 7 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 27 Oct 2011. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Quantitative muscle strength will be measured using the CINRG Quantitative Measurement System (CQMS) (time frame: February 2008). 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 27 Oct 2011.