Intermittent vs Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne Muscular Dystrophy

NCT07787689 | Not Yet Recruiting

• 1 other identifier: Dr-Nida-LHR
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• Treatment efficacy

  Increase in the post-treatment North Star Ambulatory Assessment (NSAA) scores will be noted.

  6 month after start of treatment

Secondary Outcomes (2)

• Weight gain

  6 month after start of treatment

• Hyperglycemia

  6 month after start of treatment

Study Arms (2)

Alternate-Day Prednisolone-Group

EXPERIMENTAL

Patients will be given alternate-day administration of prednisolone at a dose of 0.75 mg/kg/day.

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.

Drug: Intermittent prednisolone

Interventions

Alternate-day prednisolone DRUG

Patients will be given alternate-day administration of prednisolone at a dose of 0.75 mg/kg/day.

Alternate-Day Prednisolone-Group

Intermittent prednisolone DRUG

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.

Intermittent Prednisolone-Group

Eligibility Criteria

• Age: 4 Years - 10 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male patients
• Aged 4-10 years
• \. Confirmed diagnosis of DMD (via genetic testing and significantly elevated creatine kinase (CK) levels \>1000 U/L with typical clinical presentation)

You may not qualify if:

• 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

Sponsors & Collaborators

• Muhammad Aamir Latif: lead

Study Sites (1)

Children's Hospital/University of Child Health Sciences

Lahore, Punjab Province, 54600, Pakistan

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Nida Imdad

  Children's Hospital/University of Child Health Sciences, Lahore

  PRINCIPAL INVESTIGATOR

• Tipu Sultan, FCPS

  Children's Hospital/University of Child Health Sciences, Lahore

  STUDY DIRECTOR

Central Study Contacts

Nida Imdad

CONTACT

+923217840636; nidaimdad50@yahoo.com

Tipu Sultan, FCPS

CONTACT

+923334086668; drtipusultan@gmail.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Research Consultant

Study Record Dates

• First Submitted: August 22, 2026
• First Posted: August 26, 2026
• Study Start (Estimated): September 15, 2026
• Primary Completion (Estimated): March 15, 2027
• Study Completion (Estimated): March 15, 2027
• Last Updated: August 26, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

PA (1)