NCT03633565

Brief Summary

  1. 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. 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

Trial Health

35
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
45

participants targeted

Target at P25-P50 for phase_4

Timeline
Completed

Started Sep 2018

Typical duration for phase_4

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 12, 2018

Completed
4 days until next milestone

First Posted

Study publicly available on registry

August 16, 2018

Completed
16 days until next milestone

Study Start

First participant enrolled

September 1, 2018

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2021

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2021

Completed
Last Updated

August 16, 2018

Status Verified

August 1, 2018

Enrollment Period

3 years

First QC Date

August 12, 2018

Last Update Submit

August 15, 2018

Conditions

Myopathy

Outcome Measures

Primary Outcomes (1)

  • 6 Minute Walk Distance (6MWD)

    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

    6 month

Study Arms (3)

Steroid

ACTIVE COMPARATOR

prednisolone 20 mg tablet by mouth taken once daily for 10 days each month for 2 years

Drug: Prednisolone (Steroids)

Phosphodiestrase inhibitors

ACTIVE COMPARATOR

sildenafil 25 mg tablet by mouth once daily for 2 years

Drug: Sildenafil (Phosphodiesterase inhibitors)

Mesenchymal stem cell transplantation

EXPERIMENTAL

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

Interventions

Sildenafil (Phosphodiesterase inhibitors)DRUG

tablet 25mg

Also known as: sildenafil, viagra
Phosphodiestrase inhibitors
Prednisolone (Steroids)DRUG

tablet 20 mg

Also known as: Prednisolone 20 mg
Steroid
Mesenchymal stem cell transplantationPROCEDURE

stem cell transplantation intramuscular

Mesenchymal stem cell transplantation

Eligibility Criteria

Age5 Years - 15 Years
Sexmale(Gender-based eligibility)
Gender Eligibility Detailsonly male
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (18)

  • Mercuri E, Muntoni F. Muscular dystrophies. Lancet. 2013 Mar 9;381(9869):845-60. doi: 10.1016/S0140-6736(12)61897-2.

    PMID: 23465426BACKGROUND

  • Nallamilli BR, Ankala A, Hegde M. Molecular diagnosis of Duchenne muscular dystrophy. Curr Protoc Hum Genet. 2014 Oct 1;83:9.25.1-29. doi: 10.1002/0471142905.hg0925s83.

    PMID: 25271841BACKGROUND

  • Lapidos KA, Kakkar R, McNally EM. The dystrophin glycoprotein complex: signaling strength and integrity for the sarcolemma. Circ Res. 2004 Apr 30;94(8):1023-31. doi: 10.1161/01.RES.0000126574.61061.25.

    PMID: 15117830BACKGROUND

  • Emery AE. The muscular dystrophies. Lancet. 2002 Feb 23;359(9307):687-95. doi: 10.1016/S0140-6736(02)07815-7.

    PMID: 11879882BACKGROUND

  • Wong BL, Christopher C. Corticosteroids in Duchenne muscular dystrophy: a reappraisal. J Child Neurol. 2002 Mar;17(3):183-90. doi: 10.1177/088307380201700306.

    PMID: 12026233BACKGROUND

  • Khan MA. Corticosteroid therapy in Duchenne muscular dystrophy. J Neurol Sci. 1993 Dec 1;120(1):8-14. doi: 10.1016/0022-510x(93)90017-s.

    PMID: 8289083BACKGROUND

  • Kojima S, Takagi A, Watanabe T. [Effect of prednisolone on apoptosis and cellular infiltration in mdx mouse muscle]. Rinsho Shinkeigaku. 1999 Nov;39(11):1109-13. Japanese.

    PMID: 10689931BACKGROUND

  • Fukudome T, Shibuya N, Yoshimura T, Eguchi K. Short-term effects of prednisolone on neuromuscular transmission in the isolated mdx mouse diaphragm. Tohoku J Exp Med. 2000 Nov;192(3):211-7. doi: 10.1620/tjem.192.211.

    PMID: 11249150BACKGROUND

  • Wang YX, Rudnicki MA. Satellite cells, the engines of muscle repair. Nat Rev Mol Cell Biol. 2011 Dec 21;13(2):127-33. doi: 10.1038/nrm3265.

    PMID: 22186952BACKGROUND

  • Heslop L, Morgan JE, Partridge TA. Evidence for a myogenic stem cell that is exhausted in dystrophic muscle. J Cell Sci. 2000 Jun;113 ( Pt 12):2299-308. doi: 10.1242/jcs.113.12.2299.

    PMID: 10825301BACKGROUND

  • Galli R, Borello U, Gritti A, Minasi MG, Bjornson C, Coletta M, Mora M, De Angelis MG, Fiocco R, Cossu G, Vescovi AL. Skeletal myogenic potential of human and mouse neural stem cells. Nat Neurosci. 2000 Oct;3(10):986-91. doi: 10.1038/79924.

    PMID: 11017170BACKGROUND

  • Toma JG, Akhavan M, Fernandes KJ, Barnabe-Heider F, Sadikot A, Kaplan DR, Miller FD. Isolation of multipotent adult stem cells from the dermis of mammalian skin. Nat Cell Biol. 2001 Sep;3(9):778-84. doi: 10.1038/ncb0901-778.

    PMID: 11533656BACKGROUND

  • Qu-Petersen Z, Deasy B, Jankowski R, Ikezawa M, Cummins J, Pruchnic R, Mytinger J, Cao B, Gates C, Wernig A, Huard J. Identification of a novel population of muscle stem cells in mice: potential for muscle regeneration. J Cell Biol. 2002 May 27;157(5):851-64. doi: 10.1083/jcb.200108150. Epub 2002 May 20.

    PMID: 12021255BACKGROUND

  • Shafritz DA, Oertel M, Menthena A, Nierhoff D, Dabeva MD. Liver stem cells and prospects for liver reconstitution by transplanted cells. Hepatology. 2006 Feb;43(2 Suppl 1):S89-98. doi: 10.1002/hep.21047.

    PMID: 16447292BACKGROUND

  • Price FD, Kuroda K, Rudnicki MA. Stem cell based therapies to treat muscular dystrophy. Biochim Biophys Acta. 2007 Feb;1772(2):272-83. doi: 10.1016/j.bbadis.2006.08.011. Epub 2006 Sep 6.

    PMID: 17034994BACKGROUND

  • Keating A. Mesenchymal stromal cells: new directions. Cell Stem Cell. 2012 Jun 14;10(6):709-716. doi: 10.1016/j.stem.2012.05.015.

    PMID: 22704511BACKGROUND

  • Mendell JR, Clark KR. Challenges for gene therapy for muscular dystrophy. Curr Neurol Neurosci Rep. 2006 Jan;6(1):47-56. doi: 10.1007/s11910-996-0009-8.

    PMID: 16469271BACKGROUND

  • Partridge TA. Stem cell therapies for neuromuscular diseases. Acta Neurol Belg. 2004 Dec;104(4):141-7.

    PMID: 15742603BACKGROUND

MeSH Terms

Conditions

Muscular Diseases

Interventions

Sildenafil CitratePhosphodiesterase InhibitorsPrednisoloneSteroidsMesenchymal Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Musculoskeletal DiseasesNeuromuscular DiseasesNervous System Diseases

Intervention Hierarchy (Ancestors)

SulfonamidesAmidesOrganic ChemicalsSulfonesSulfur CompoundsPiperazinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsPurinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingEnzyme InhibitorsMolecular Mechanisms of Pharmacological ActionPharmacologic ActionsChemical Actions and UsesPregnadienetriolsPregnadienesPregnanesFused-Ring CompoundsPolycyclic CompoundsStem Cell TransplantationCell TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, Operative

Study Officials

  • Emad EL Daly, Professor

    Assiut University

    STUDY DIRECTOR

Central Study Contacts

Duaa Mahmoud, Assistant professor

CONTACT

01223112124duaa-raafat@hotmail.com

Mervat Youssef, Lecturer

CONTACT

01142606221mamuosif2000@gmail.com

Study Design

Study Type
interventional
Phase
phase 4
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
principle investigator

Study Record Dates

First Submitted

August 12, 2018

First Posted

August 16, 2018

Study Start

September 1, 2018

Primary Completion

September 1, 2021

Study Completion

November 1, 2021

Last Updated

August 16, 2018

Record last verified: 2018-08

Data Sharing

IPD Sharing
Will not share