Comparative Study of Strategies for Management of Duchenne Myopathy (DM)

• Status: Unknown status
• Trial end date: 2021-11-01
• Target enrollment: 0
• Participant gender: Male

Summary

1. Comparing different lines of treatment of Duchenne Myopathy (DM) and assessment of new lines of treatment (mesenchymal stem cell, phosphodiesterase inhibitors) in reducing the impact of disability in the patients with Duchenne Myopathy and slowing the progression of cardiomyopathy

2. Upsetting and implementation of the best treatment plan for those children with Duchenne myopathy which is suitable for the available resources in Assiut University Children Hospital

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assiut University
• Treatments: Methylprednisolone; Methylprednisolone Acetate; Methylprednisolone Hemisuccinate; Phosphodiesterase Inhibitors; Prednisolone; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate; Sildenafil Citrate

Criteria

Inclusion Criteria:

• Diagnosis of DMD confirmed by electromyogram (EMG) , Creatine phosphokinase (CPK) level and/ or DNA analysis or muscle biopsy.

• Male patients

• Age 5-15y.

• Ambulatory (loss of ambulation was only seen in those with baseline 6 Minute Walk Distance {6MWD} <325 meters.)

• No clinical evidence of heart failure.

Exclusion Criteria:

• Female patients

• Any injury which may impact functional testing, e.g. upper or lower limb fracture.

• hypertension, diabetes,

• Wheelchair bound.

• Cardiac rhythm disorder, specifically: rhythm other than sinus, supraventricular tachycardia (SVT), atrial fibrillation, ventricular tachycardia.or heart failure (left ventricle ejection fraction {LVEF < 50%}.

• Continuous ventilatory support.

• Liver disease (acute, chronic liver disease)

• Renal impairment