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Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy

Primary Purpose

Duchenne Muscular Dystrophy

Status

Unknown status

Phase

Phase 1

Locations

Turkey

Study Type

Interventional

Intervention

Umbilical Cord Mesenchymal Stem Cell

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring umbilical cord, mesenchymal stem cell

Eligibility Criteria

7 Years - 20 Years (Child, Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

- Patients with diagnosis of DMD that is proven clinically and genetically Age between 7-20 Patients need partial respiratory support, during the day Patients have less than or equal to stage I NIH, Liver, renal and cardiac function Patients without cancer Patients without allergic disease Patients without bleeding diathesis,

Exclusion Criteria:

Patients need complete respiratory support Patients have more than to stage II NIH, Liver, renal and cardiac function Patients have bleeding diathesis and allergic disease

Sites / Locations

Acibadem LabcellRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Umbilical Cord Mesenchymal Stem Cell

Arm Description

Outcomes

Primary Outcome Measures

Duchenne muscular dystrophy gene expression

Secondary Outcome Measures

Full Information

NCT ID

NCT02285673

First Posted

November 5, 2014

Last Updated

November 6, 2014

Sponsor

Acibadem University

1. Study Identification

Unique Protocol Identification Number

NCT02285673

Brief Title

Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy

Official Title

Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy: Phase 1/2 Study

Study Type

Interventional

2. Study Status

Record Verification Date

November 2014

Overall Recruitment Status

Unknown status

Study Start Date

November 2013 (undefined)

Primary Completion Date

February 2015 (Anticipated)

Study Completion Date

November 2015 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Acibadem University

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

Duchenne muscular dystrophy (DMD) is a genetic disorder caused by an absence of dystrophin and characterized by progressive muscle degeneration. There is no cure for DMD at present but, there are several strategies under-researched for treatment of DMD such as steroid treatment, gene theraphy, exon skipping, stop codon read through and gene repair, cell theraphy and theraphy with drug that help to produce utrophin protein. The aim of this study is investigate the eficacy of human umblical cord mesenchymal stem cells on DMD and understanding if wild type gene can be transfered to the patient.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Duchenne Muscular Dystrophy

Keywords

umbilical cord, mesenchymal stem cell

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Umbilical Cord Mesenchymal Stem Cell

Arm Type

Experimental

Intervention Type

Biological

Intervention Name(s)

Umbilical Cord Mesenchymal Stem Cell

Other Intervention Name(s)

umbilical cord mesenchymal stem cells

Primary Outcome Measure Information:

Title

Duchenne muscular dystrophy gene expression

Time Frame

up to 9 months

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

7 Years

Maximum Age & Unit of Time

20 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: - Patients with diagnosis of DMD that is proven clinically and genetically Age between 7-20 Patients need partial respiratory support, during the day Patients have less than or equal to stage I NIH, Liver, renal and cardiac function Patients without cancer Patients without allergic disease Patients without bleeding diathesis, Exclusion Criteria: Patients need complete respiratory support Patients have more than to stage II NIH, Liver, renal and cardiac function Patients have bleeding diathesis and allergic disease

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Ercument Ovali, Prof.Dr.

Phone

+905325729174

ercument.ovali@acibademlabcell.com.tr

First Name & Middle Initial & Last Name or Official Title & Degree

Cengiz Yakicier, Prof.Dr.

Phone

05362998743

cengiz.yakicier@asg.com.tr

Facility Information:

Facility Name

Acibadem Labcell

City

Istanbul

State/Province

Uskudar

ZIP/Postal Code

34662

Country

Turkey

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Ercument Ovali, Prof. Dr.

Phone

+905325729174

ercument.ovali@acibadem.com.tr

12. IPD Sharing Statement

Citations:

PubMed Identifier

15117830

Citation

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.

Results Reference

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PubMed Identifier

9891887

Citation

Miller JB, Schaefer L, Dominov JA. Seeking muscle stem cells. Curr Top Dev Biol. 1999;43:191-219. doi: 10.1016/s0070-2153(08)60382-8.

Results Reference

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PubMed Identifier

21784508

Citation

Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, Abbs S, Garralda ME, Bourke J, Wells DJ, Dickson G, Wood MJ, Wilton SD, Straub V, Kole R, Shrewsbury SB, Sewry C, Morgan JE, Bushby K, Muntoni F. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet. 2011 Aug 13;378(9791):595-605. doi: 10.1016/S0140-6736(11)60756-3. Epub 2011 Jul 23.

Results Reference

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PubMed Identifier

23907995

Citation

Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM; Eteplirsen Study Group. Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol. 2013 Nov;74(5):637-47. doi: 10.1002/ana.23982. Epub 2013 Sep 10.

Results Reference

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PubMed Identifier

21428760

Citation

Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N, Holling T, Janson AA, Platenburg GJ, Sipkens JA, Sitsen JM, Aartsma-Rus A, van Ommen GJ, Buyse G, Darin N, Verschuuren JJ, Campion GV, de Kimpe SJ, van Deutekom JC. Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med. 2011 Apr 21;364(16):1513-22. doi: 10.1056/NEJMoa1011367. Epub 2011 Mar 23. Erratum In: N Engl J Med. 2011 Oct 6;365(14):1361.

Results Reference

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PubMed Identifier

18598348

Citation

Kerkis I, Ambrosio CE, Kerkis A, Martins DS, Zucconi E, Fonseca SA, Cabral RM, Maranduba CM, Gaiad TP, Morini AC, Vieira NM, Brolio MP, Sant'Anna OA, Miglino MA, Zatz M. Early transplantation of human immature dental pulp stem cells from baby teeth to golden retriever muscular dystrophy (GRMD) dogs: Local or systemic? J Transl Med. 2008 Jul 3;6:35. doi: 10.1186/1479-5876-6-35.

Results Reference

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Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy

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