Duchenne Muscular Dystrophy
Conditions
Brief 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. 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).
Interventions
DRUGAlternate-day prednisolone
Patients will be given alternate-day administration of prednisolone at a dose of 0.75 mg/kg/day.
DRUGIntermittent prednisolone
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.
Sponsors
Muhammad Aamir Latif
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
4 Years to 10 Years
Healthy volunteers
No
Inclusion criteria
* 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)
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment efficacy | 6 month after start of treatment | Increase in the post-treatment North Star Ambulatory Assessment (NSAA) scores will be noted. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Weight gain | 6 month after start of treatment | The frequency of patients who will gain weight as an adverse effect, will be noted |
| Hyperglycemia | 6 month after start of treatment | The frequency of patients who will who will develop hyperglycemia as an adverse effect will be noted. |
Countries
Pakistan
Contacts
CONTACTNida Imdad
CONTACTTipu Sultan, FCPS
PRINCIPAL_INVESTIGATORNida Imdad
Children's Hospital/University of Child Health Sciences, Lahore
STUDY_DIRECTORTipu Sultan, FCPS
Children's Hospital/University of Child Health Sciences, Lahore
Outcome results
None listed