Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02285673
• Other study ID #: DMD-UC-MSC-1
• Status: Recruiting
• Phase: Phase 1/Phase 2
• First received: November 5, 2014
• Last updated: November 6, 2014
• Start date: November 2013
• Est. completion date: November 2015

Study information

• Verified date: November 2014
• Source: Acibadem University
• Contact: Ercument Ovali, Prof.Dr.
• Phone: +905325729174
• Email: ercument.ovali[@]acibademlabcell.com.tr
• Is FDA regulated: No
• Health authority: Turkey: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: November 2015
• Est. primary completion date: February 2015
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 7 Years to 20 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Biological:
• Umbilical Cord Mesenchymal Stem Cell

Locations

Country	Name	City	State
Turkey	Acibadem Labcell	Istanbul	Uskudar

Sponsors (1)

Lead Sponsor	Collaborator
Acibadem University

Country where clinical trial is conducted

Turkey, 

References & Publications (6)

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. — View 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. — View 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. — View 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. Review. — View 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. — View Citation

Miller JB, Schaefer L, Dominov JA. Seeking muscle stem cells. Curr Top Dev Biol. 1999;43:191-219. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Duchenne muscular dystrophy gene expression		up to 9 months	Yes