Duchenne Muscular DystrophyPhase 3NCT00004646
Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy
A Phase 3 study testing prednisone in people with Duchenne Muscular Dystrophy, run by National Center for Research Resources (NCRR).
Trial summary
OBJECTIVES: I. Characterize the effect of prednisone on muscle protein metabolism in patients with Duchenne muscular dystrophy. II. Determine whether prednisone changes levels of insulin-like growth factor 1, growth hormone, and insulin. III. Read more about this study
- Who is it for?
- Ages 5 to 15 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- prednisone
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- Apr 1995to not given
- 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 5 to 15 years
- Men only
- No healthy volunteers
- Child
PROTOCOL ENTRY CRITERIA:
- Ambulatory males with Duchenne muscular dystrophy
- No medical/psychiatric contraindication to protocol therapy
- No requirement for regular use of prescription medication
About this study
OBJECTIVES: I. Characterize the effect of prednisone on muscle protein metabolism in patients with Duchenne muscular dystrophy.
II. Determine whether prednisone changes levels of insulin-like growth factor 1, growth hormone, and insulin.
III. Characterize the effect of prednisone on muscle morphometry and muscle localization of utrophin.
IV. Compare the prednisone response in patients with Duchenne muscular dystrophy to that seen in normal individuals and in patients with facioscapulohumeral dystrophy.
Read the detailed description
PROTOCOL OUTLINE: This is a randomized, double-blind study. Patients are randomly assigned to prednisone or placebo. Therapy is administered daily for 12 weeks; prednisone is then tapered.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- prednisone
- Keywords
- Duchenne muscular dystrophy, genetic diseases and dysmorphic syndromes, muscular dystrophy, neurologic and psychiatric disorders, rare disease
- Medical subject headings
- Muscular Dystrophy, Duchenne, Genetic Diseases, Inborn, Muscular Dystrophies, Neurologic Manifestations, Mental Disorders, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
prednisoneDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2000 (estimated)
- Study startApr 1995
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
- Masking
- Double blind
- Enrolment
- 20 participants
Sponsor and investigators
- Lead sponsor
- National Center for Research Resources (NCRR)
- Sponsor type
- US National Institutes of Health
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), University of Rochester
- Investigators
- Robert Griggs, Study chair, University of Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004646
- Sponsor's protocol ID
- 199/11695
- Other IDs
- URMC-2251
- Status last verified
- Dec 2001
- First submitted
- 24 Feb 2000
- Last updated
- 24 Jun 2005
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2005. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT00004646
- Acronym / sponsor ID
- 199/11695
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne muscular dystrophy, genetic diseases and dysmorphic syndromes, muscular dystrophy, neurologic and psychiatric disorders, rare disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants
- Lead sponsor
- National Center for Research Resources (NCRR)US National Institutes of Health
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), University of Rochester
- Study start
- Apr 1995
- First posted
- 25 Feb 2000 (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.
OBJECTIVES: I. Characterize the effect of prednisone on muscle protein metabolism in patients with Duchenne muscular dystrophy.
II. Determine whether prednisone changes levels of insulin-like growth factor 1, growth hormone, and insulin.
III. Characterize the effect of prednisone on muscle morphometry and muscle localization of utrophin.
IV. Compare the prednisone response in patients with Duchenne muscular dystrophy to that seen in normal individuals and in patients with facioscapulohumeral dystrophy.
Detailed description
PROTOCOL OUTLINE: This is a randomized, double-blind study. Patients are randomly assigned to prednisone or placebo. Therapy is administered daily for 12 weeks; prednisone is then tapered.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Masking
- Double blind
Who may qualify
- Ages
- 5 to 15 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 |
|---|---|---|
| prednisone | 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 24 Jun 2005.
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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.
Robert Griggs study chair
University of Rochester
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing prednisone in people with Duchenne Muscular Dystrophy, run by National Center for Research Resources (NCRR).
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 24 Jun 2005. Status can change between updates, so confirm with the study contact before making plans.
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.
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 24 Jun 2005.