Duchenne Muscular DystrophyNCT07787689
Intermittent vs Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne Muscular Dystrophy
A study testing Alternate-day prednisolone and Intermittent prednisolone in people with Duchenne Muscular Dystrophy, run by Muhammad Aamir Latif at 1 site in Pakistan.
Trial summary
The alternate-day regimen is hypothesized to reduce hypothalamic-pituitary-adrenal (HPA) axis suppression and decrease steroid-related toxicity. Similarly, intermittent regimens aim to provide periodic steroid exposure sufficient for muscle preservation while allowing recovery periods to limit side effects. 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?
- Alternate-day prednisolone and Intermittent prednisolone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 sitePakistan
- Study dates
- 15 Sep 2026to 15 Mar 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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
- Male patients
- Aged 4-10 years
- 2. Confirmed diagnosis of DMD (via genetic testing and significantly elevated creatine kinase (CK) levels >1000 U/L with typical clinical presentation)
Who cannot join
- Patients with pre-existing cardiopulmonary failure
- Patients entirely unable to perform baseline ambulatory assessments (non-ambulatory)
- Patients with clinically suspected muscular dystrophy with normal genetic study for DMD
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Pakistan1 site
Children's Hospital/University of Child Health Sciences
Lahore, Punjab Province, 54600
Nida Imdad, +923217840636, nidaimdad50@yahoo.com
Tipu Sultan, FCPS, +923334086668, drtipusultan@gmail.com
About this study
Official title: Efficacy and Safety of Intermittent vs. Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne Muscular Dystrophy: A Randomized, Open-Label Trial
The alternate-day regimen is hypothesized to reduce hypothalamic-pituitary-adrenal (HPA) axis suppression and decrease steroid-related toxicity. Similarly, intermittent regimens aim to provide periodic steroid exposure sufficient for muscle preservation while allowing recovery periods to limit side effects. There is a critical need for localized, prospective data to determine which protocol offers the optimal balance of efficacy and safety for pediatric patients in Pakistan.
Read the detailed description
The study will provide evidence to support the standardization of corticosteroid therapy in the local population. By directly comparing alternate-day versus intermittent dosing at The University of Child Health Sciences \& Children's Hospital, Lahore, the study aims to identify the regimen that best preserves motor function while minimizing debilitating side effects, thereby improving overall quality of life and treatment compliance among local patients with Duchenne Muscular Dystrophy (DMD).
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Alternate-day prednisolone, Intermittent prednisolone
- 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.
Alternate-day prednisoloneDrug
Patients will be given alternate-day administration of prednisolone at a dose of 0.75 mg/kg/day.
Intermittent prednisoloneDrug
Patients will be administered prednisolone at a dose of 0.75 mg/kg/day for 10 consecutive days, followed by a 10-day steroid-free period.
| Study group | Role | What they receive |
|---|---|---|
| Alternate-Day Prednisolone-Group | Experimental | Patients will be given alternate-day administration of prednisolone at a dose of 0.75 mg/kg/day. Receives: Drug: Alternate-day prednisolone |
| Intermittent Prednisolone-Group | Experimental | Patients will be administered prednisolone at a dose of 0.75 mg/kg/day for 10 consecutive days, followed by a 10-day steroid-free period. Receives: Drug: Intermittent prednisolone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment efficacy
Increase in the post-treatment North Star Ambulatory Assessment (NSAA) scores will be noted.
Measured: 6 month after start of treatment
Secondary outcomes (2)
Weight gain
The frequency of patients who will gain weight as an adverse effect, will be noted
Measured: 6 month after start of treatment
Hyperglycemia
The frequency of patients who will who will develop hyperglycemia as an adverse effect will be noted.
Measured: 6 month after start of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2026
- Study start15 Sep 2026 (estimated)
- Primary completion15 Mar 2027 (estimated)
- Study completion15 Mar 2027 (estimated)
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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Muhammad Aamir Latif
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Muhammad Aamir Latif, RESnTEC, Institute of Research
- Investigators
- Nida Imdad, Principal investigator, Children's Hospital/University of Child Health Sciences, LahoreTipu Sultan, FCPS, Study director, Children's Hospital/University of Child Health Sciences, Lahore
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07787689
- Sponsor's protocol ID
- Dr-Nida-LHR
- Status last verified
- Aug 2026
- First submitted
- 22 Aug 2026
- Last updated
- 26 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Data can be shared on a reasonable request.
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Intermittent vs. Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne Muscular Dystrophy: A Randomized, Open-Label Trial
- ClinicalTrials.gov ID
- NCT07787689
- Acronym / sponsor ID
- Dr-Nida-LHR
- Conditions
- Duchenne Muscular Dystrophy
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Muhammad Aamir LatifUniversity, hospital or other
- Study start
- 15 Sep 2026 (estimated)
- Primary completion
- 15 Mar 2027 (estimated)
- Study completion
- 15 Mar 2027 (estimated)
- First posted
- 26 Aug 2026 (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 alternate-day regimen is hypothesized to reduce hypothalamic-pituitary-adrenal (HPA) axis suppression and decrease steroid-related toxicity. Similarly, intermittent regimens aim to provide periodic steroid exposure sufficient for muscle preservation while allowing recovery periods to limit side effects. There is a critical need for localized, prospective data to determine which protocol offers the optimal balance of efficacy and safety for pediatric patients in Pakistan.
Detailed description
The study will provide evidence to support the standardization of corticosteroid therapy in the local population. By directly comparing alternate-day versus intermittent dosing at The University of Child Health Sciences \& Children's Hospital, Lahore, the study aims to identify the regimen that best preserves motor function while minimizing debilitating side effects, thereby improving overall quality of life and treatment compliance among local patients with Duchenne Muscular Dystrophy (DMD).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Alternate-Day Prednisolone-Group | Experimental | Drug: Alternate-day prednisolonePatients will be given alternate-day administration of prednisolone at a dose of 0.75 mg/kg/day. |
| Intermittent Prednisolone-Group | Experimental | Drug: Intermittent prednisolonePatients will be administered prednisolone at a dose of 0.75 mg/kg/day for 10 consecutive days, followed by a 10-day steroid-free period. |
Who may qualify
- Ages
- 4 to 10 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Male patients
- Aged 4-10 years
- 2. Confirmed diagnosis of DMD (via genetic testing and significantly elevated creatine kinase (CK) levels >1000 U/L with typical clinical presentation)
Key exclusion criteria (3 listed)
- Patients with pre-existing cardiopulmonary failure
- Patients entirely unable to perform baseline ambulatory assessments (non-ambulatory)
- Patients with clinically suspected muscular dystrophy with normal genetic study for DMD
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 |
|---|---|---|
| Alternate-day prednisolonePatients will be given alternate-day administration of prednisolone at a dose of 0.75 mg/kg/day. | Drug | Alternate-Day Prednisolone-Group |
| Intermittent prednisolonePatients will be administered prednisolone at a dose of 0.75 mg/kg/day for 10 consecutive days, followed by a 10-day steroid-free period. | Drug | Intermittent Prednisolone-Group |
Primary outcome
Treatment efficacy
Time frame: 6 month after start of treatment
Increase in the post-treatment North Star Ambulatory Assessment (NSAA) scores will be noted.
2 secondary outcomes
Weight gain
Time frame: 6 month after start of treatment
Hyperglycemia
Time frame: 6 month after start of treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2026.
- Individual participant dataSharing planned: No. Data can be shared on a reasonable request.
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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.
Nida Imdad principal investigator
Children's Hospital/University of Child Health Sciences, Lahore
Tipu Sultan, FCPS study director
Children's Hospital/University of Child Health Sciences, Lahore
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Alternate-day prednisolone and Intermittent prednisolone in people with Duchenne Muscular Dystrophy, run by Muhammad Aamir Latif at 1 site in Pakistan.
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 3 inclusion and 3 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: opening soon, as of the sponsor's last update on 26 Aug 2026. 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 Pakistan. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Nida Imdad (+923217840636, nidaimdad50@yahoo.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment efficacy (time frame: 6 month after start of treatment). 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 26 Aug 2026.