Duchenne Muscular Dystrophy (DMD)Phase 2NCT02036463
A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy
A Phase 2 study testing Prednisone in people with Duchenne Muscular Dystrophy (DMD), run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial summary
Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disease for which no curative treatment has yet been identified, making it important to slow progression and improve the quality of life among affected boys and young men. Read more about this study
- Who is it for?
- Ages 3 to 6 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- Prednisone
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2014to Feb 2015
- 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 3 to 6 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Genetically confirmed dystrophin mutation compatible with DMD phenotype. Specifically, 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' OR showing complete absence of dystrophin by muscle biopsy.
- Ages between 3 years and < 7 years
- Steroid-naïve
- Signed informed consent
Who cannot join
- Treatment with CoenzymeQ10, creatine, amino acid supplements within 3 months of study entry
- Treatment with cardiac medications: beta-blockers, digoxin, and carvedilol
- Existing medical condition or physical disability that would alter subject's motor development
- Existing medical condition that precludes the use of corticosteroids
- Inability to swallow sample tablet in bite of soft food*
- Investigator assessment that participant or family will not be compliant with treatment or study procedures
- Been on investigational DMD medication for the past 6 months
Trial locations and contacts
1 site in 1 country.
United States1 site
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
About this study
Official title: CINRG0513: A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disease for which no curative treatment has yet been identified, making it important to slow progression and improve the quality of life among affected boys and young men. Treatment with corticosteroids is standard of care for patients with DMD five years old and older, due to the robust observation that this intervention lengthens the interval prior to loss of ambulation but is associated with many side effects. This clinical trial will be conducted in the youngest age group able to receive corticosteroids orally and on whom study outcomes are measurable, ages 3 to 7 years. This is a randomized, double blinded, double masked, placebo-controlled clinical trial that will explore whether better synchronization of corticosteroid administration with the circadian rhythm will provide improved tolerability and at least comparable efficacy to current standards in which corticosteroids are always given in the morning. Furthermore, the trial provides a unique opportunity to rigorously evaluate corticosteroid effects in the young DMD patient, both for efficacy as compared to placebo and as a study of the impact of corticosteroid chronotherapy, or delayed release, on increased tolerability over standard therapy. The main hypothesis is that synchronization of the timing of corticosteroid dosing will improve medication tolerability in children, while maintaining (non-inferiority) the efficacy of corticosteroid. The study also offers a unique opportunity to measure several biomarkers as well as novel genetic modifiers that may further impact the response to corticosteroid in DMD.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- Prednisone, Placebo
- Keywords
- Duchenne Muscular Dystrophy, Corticosteroids
- 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.
PrednisoneDrug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Immediate Release Prednisone | Active comparator | During the entire 18 months of the protocol, these subjects will receive immediate release prednisone as a morning dose. All observations and measurements are performed the same as the other study groups. Receives: Drug: Prednisone |
| Delayed Release Prednisone | Experimental | During the entire 18 months of the protocol, these subjects will receive delayed release prednisone as an evening dose. All observations and measurements are performed the same as the other study groups. Receives: Drug: Prednisone |
| Placebo-Delayed Release Prednisone | Placebo comparator | During the first 6 months of the protocol, these subjects will receive placebo. After 6 months, this half of the placebo group was re-randomized to receive the delayed release prednisone medication. All observations and measurements are performed the same as the other study groups. Receives: Drug: Prednisone; Drug: Placebo |
| Placebo-Immediate Release Prednisone | Placebo comparator | During the first 6 months of the protocol, these subjects will receive placebo. After 6 months, this half of the placebo group was re-randomized to receive the immediate release corticosteroid medication. All observations and measurements are performed the same as the other study groups. Receives: Drug: Prednisone; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety
The primary outcome will measure safety and tolerability by tabulating number of adverse events occuring in patients in each treatment group. Adverse events are specified in the protocol and relate to excess weight gain, inadequate linear growth, elevated blood pressure, worsening scores on behavior scales, declining heart rate variability and abnormalities of circadian rhythm of sympathetic tone.
Measured: 18 months
Secondary outcomes (2)
Time to walk/run 50 meters
This test will measure the time it will take to run/walk 50 meters. It has not been typically used in clinical trials as a timed test measure, however, may be a more sensitive test measure in the very young cohort to assess functional strength as it measures a longer distance to run compared to the 10 meter walk. Preliminary analysis in a small pilot cohort indicates that it is better correlated with other functional assessments such as the North Star Ambulatory Assessment.
Measured: 18 months
North Star Ambulatory Assessment (NSAA)
The NSAA is a clinician rated 17-item functional scale originally designed for ambulant boys with DMD who are able to ambulate at least 10 meters. This evaluation tool assesses functional activities including standing, getting up from the floor, negotiating steps, hopping, and running. The assessment is based on a 3-point rating scale of 2= ability to perform the test normally, 1= modified method or assistance to perform test, 0=unable to perform the test. Thus, total score can range from 0 (completely non-ambulant) to 34 (no impairment) on these assessments. NSAA has shown good reliability and validity in multi-center studies as well as good clinical validity demonstrated with Rasch analysis.
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2014 (estimated)
- Study startNov 2014
- Primary completionFeb 2015
- Study completionFeb 2015
Why it stopped: No funding for project.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies 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
- Supportive care
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Collaborators
- Children's National Research Institute
- Responsible party
- Principal investigator, Nancy Kuntz, MD, Ann & Robert H Lurie Children's Hospital of Chicago
- Investigators
- Nancy Kuntz, MD, Study chair, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02036463
- Sponsor's protocol ID
- CINRG0513
- Other IDs
- IND #121239
- Status last verified
- Feb 2015
- First submitted
- 6 Jan 2014
- Last updated
- 18 Feb 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- CINRG0513: A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02036463
- Acronym / sponsor ID
- CINRG0513
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Duchenne Muscular Dystrophy, Corticosteroids
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: No funding for project.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Collaborators
- Children's National Research Institute
- Study start
- Nov 2014
- Primary completion
- Feb 2015 (actual)
- Study completion
- Feb 2015 (actual)
- First posted
- 15 Jan 2014 (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.
Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disease for which no curative treatment has yet been identified, making it important to slow progression and improve the quality of life among affected boys and young men. Treatment with corticosteroids is standard of care for patients with DMD five years old and older, due to the robust observation that this intervention lengthens the interval prior to loss of ambulation but is associated with many side effects. This clinical trial will be conducted in the youngest age group able to receive corticosteroids orally and on whom study outcomes are measurable, ages 3 to 7 years. This is a randomized, double blinded, double masked, placebo-controlled clinical trial that will explore whether better synchronization of corticosteroid administration with the circadian rhythm will provide improved tolerability and at least comparable efficacy to current standards in which corticosteroids are always given in the morning. Furthermore, the trial provides a unique opportunity to rigorously evaluate corticosteroid effects in the young DMD patient, both for efficacy as compared to placebo and as a study of the impact of corticosteroid chronotherapy, or delayed release, on increased tolerability over standard therapy. The main hypothesis is that synchronization of the timing of corticosteroid dosing will improve medication tolerability in children, while maintaining (non-inferiority) the efficacy of corticosteroid. The study also offers a unique opportunity to measure several biomarkers as well as novel genetic modifiers that may further impact the response to corticosteroid in DMD.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Immediate Release Prednisone | Active comparator | Drug: PrednisoneDuring the entire 18 months of the protocol, these subjects will receive immediate release prednisone as a morning dose. All observations and measurements are performed the same as the other study groups. |
| Delayed Release Prednisone | Experimental | Drug: PrednisoneDuring the entire 18 months of the protocol, these subjects will receive delayed release prednisone as an evening dose. All observations and measurements are performed the same as the other study groups. |
| Placebo-Delayed Release Prednisone | Placebo comparator | Drug: Prednisone, Drug: PlaceboDuring the first 6 months of the protocol, these subjects will receive placebo. After 6 months, this half of the placebo group was re-randomized to receive the delayed release prednisone medication. All observations and measurements are performed the same as the other study groups. |
| Placebo-Immediate Release Prednisone | Placebo comparator | Drug: Prednisone, Drug: PlaceboDuring the first 6 months of the protocol, these subjects will receive placebo. After 6 months, this half of the placebo group was re-randomized to receive the immediate release corticosteroid medication. All observations and measurements are performed the same as the other study groups. |
Who may qualify
- Ages
- 3 to 6 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Genetically confirmed dystrophin mutation compatible with DMD phenotype. Specifically, 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' OR showing complete absence of dystrophin by muscle biopsy.
- Ages between 3 years and < 7 years
- Steroid-naïve
- Signed informed consent
Key exclusion criteria (7 listed)
- Treatment with CoenzymeQ10, creatine, amino acid supplements within 3 months of study entry
- Treatment with cardiac medications: beta-blockers, digoxin, and carvedilol
- Existing medical condition or physical disability that would alter subject's motor development
- Existing medical condition that precludes the use of corticosteroids
- Inability to swallow sample tablet in bite of soft food*
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 | Delayed Release Prednisone, Immediate Release Prednisone, Placebo-Delayed Release Prednisone, Placebo-Immediate Release Prednisone |
| Placebo | Drug | Placebo-Delayed Release Prednisone, Placebo-Immediate Release Prednisone |
Primary outcome
Safety
Time frame: 18 months
The primary outcome will measure safety and tolerability by tabulating number of adverse events occuring in patients in each treatment group. Adverse events are specified in the protocol and relate to excess weight gain, inadequate linear growth, elevated blood pressure, worsening scores on behavior scales, declining heart rate variability and abnormalities of circadian rhythm of sympathetic tone.
2 secondary outcomes
Time to walk/run 50 meters
Time frame: 18 months
North Star Ambulatory Assessment (NSAA)
Time frame: 18 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Feb 2015.
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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.
Nancy Kuntz, MD study chair
Ann & Robert H Lurie Children's Hospital of Chicago
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 Prednisone in people with Duchenne Muscular Dystrophy (DMD), run by Ann & Robert H Lurie Children's Hospital of Chicago 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 4 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: withdrawn, as of the sponsor's last update on 18 Feb 2015. 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. Some participants in this study may receive a placebo. 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 was withdrawn before enrolling participants, so there are no results.
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?
Safety (time frame: 18 months). 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 18 Feb 2015.