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Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy

NCT ID: NCT02285673

Last Updated: 2014-11-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-11-30

Study Completion Date

2015-11-30

Brief Summary

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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.

Detailed Description

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Conditions

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Duchenne Muscular Dystrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Umbilical Cord Mesenchymal Stem Cell

Group Type EXPERIMENTAL

Umbilical Cord Mesenchymal Stem Cell

Intervention Type BIOLOGICAL

Interventions

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Umbilical Cord Mesenchymal Stem Cell

Intervention Type BIOLOGICAL

Other Intervention Names

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umbilical cord mesenchymal stem cells

Eligibility Criteria

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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
Minimum Eligible Age

7 Years

Maximum Eligible Age

20 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Acibadem University

OTHER

Sponsor Role lead

Responsible Party

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Ercument Ovali

Specialist, Haematology

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Acibadem Labcell

Istanbul, Uskudar, Turkey (Türkiye)

Site Status RECRUITING

Countries

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Turkey (Türkiye)

Central Contacts

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Ercument Ovali, Prof.Dr.

Role: CONTACT

[email protected]
+905325729174

Cengiz Yakicier, Prof.Dr.

Role: CONTACT

[email protected]
05362998743

Facility Contacts

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Ercument Ovali, Prof. Dr.

Role: primary

[email protected]
+905325729174

References

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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.

Reference Type BACKGROUND
PMID: 15117830 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 9891887 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 21784508 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 23907995 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 21428760 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 18598348 (View on PubMed)

Other Identifiers

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DMD-UC-MSC-1

Identifier Type: -

Identifier Source: org_study_id