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

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ClinicalTrials.gov Identifier: NCT03633565
Recruitment Status : Not yet recruiting
First Posted : August 16, 2018
Last Update Posted : August 16, 2018
Sponsor:
Information provided by (Responsible Party):
Nancy Alaa, Assiut University

Brief 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

Condition or disease Intervention/treatment Phase
Myopathy Drug: Sildenafil (Phosphodiesterase inhibitors) Drug: Prednisolone (Steroids) Procedure: Mesenchymal stem cell transplantation Phase 4

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 45 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Comparative Study of Strategies for Management of Duchenne Myopathy in Assiut University Children Hospital
Estimated Study Start Date : September 2018
Estimated Primary Completion Date : September 2021
Estimated Study Completion Date : November 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Muscle Disorders

Arm Intervention/treatment
Active Comparator: Steroid
prednisolone 20 mg tablet by mouth taken once daily for 10 days each month for 2 years
Drug: Prednisolone (Steroids)
tablet 20 mg
Other Name: Prednisolone 20 mg

Active Comparator: Phosphodiestrase inhibitors
sildenafil 25 mg tablet by mouth once daily for 2 years
Drug: Sildenafil (Phosphodiesterase inhibitors)
tablet 25mg
Other Names:
  • sildenafil
  • viagra

Experimental: Mesenchymal stem cell transplantation
The cells can be injected intramuscular in several points in the muscle alternatively they can be injected in the motor point of the muscle. A motor point is the point at which the motor branch of the innervating nerve enters the muscle). This injection is repeated every 6 month up to 2 years.
Procedure: Mesenchymal stem cell transplantation
stem cell transplantation intramuscular




Primary Outcome Measures :
  1. 6 Minute Walk Distance (6MWD) [ Time Frame: 6 month ]
    It is used as measure of motor strength in patients with Duchenne Myopathy. A baseline 6MWD of <350 meters was associated with greater functional decline, and loss of ambulation was only seen in those with baseline 6MWD <325 meters



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Ages Eligible for Study:   5 Years to 15 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   only male
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03633565


Contacts
Contact: Duaa Mahmoud, Assistant professor 01223112124 duaa-raafat@hotmail.com
Contact: Mervat Youssef, Lecturer 01142606221 mamuosif2000@gmail.com

Sponsors and Collaborators
Assiut University
Investigators
Study Director: Emad EL Daly, Professor Assiut University

Publications:

Responsible Party: Nancy Alaa, principle investigator, Assiut University
ClinicalTrials.gov Identifier: NCT03633565     History of Changes
Other Study ID Numbers: smdmauch
First Posted: August 16, 2018    Key Record Dates
Last Update Posted: August 16, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Prednisolone acetate
Methylprednisolone acetate
Prednisolone
Methylprednisolone
Methylprednisolone Hemisuccinate
Prednisolone hemisuccinate
Prednisolone phosphate
Sildenafil Citrate
Phosphodiesterase Inhibitors
Anti-Inflammatory Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Gastrointestinal Agents
Neuroprotective Agents
Protective Agents
Vasodilator Agents
Phosphodiesterase 5 Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action