NCT02285673

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.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
10

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Nov 2013

Typical duration for phase_1

Geographic Reach
1 country

1 active site

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

Study Start

First participant enrolled

November 1, 2013

Completed
1 year until next milestone

First Submitted

Initial submission to the registry

November 5, 2014

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 7, 2014

Completed
3 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2015

Completed
9 months until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2015

Completed
Last Updated

November 7, 2014

Status Verified

November 1, 2014

Enrollment Period

1.3 years

First QC Date

November 5, 2014

Last Update Submit

November 6, 2014

Conditions

Duchenne Muscular Dystrophy

Keywords

umbilical cord, mesenchymal stem cell

Outcome Measures

Primary Outcomes (1)

  • Duchenne muscular dystrophy gene expression

    up to 9 months

Study Arms (1)

Umbilical Cord Mesenchymal Stem Cell

EXPERIMENTAL
Biological: Umbilical Cord Mesenchymal Stem Cell

Interventions

Umbilical Cord Mesenchymal Stem CellBIOLOGICAL
Also known as: umbilical cord mesenchymal stem cells
Umbilical Cord Mesenchymal Stem Cell

Eligibility Criteria

Age7 Years - 20 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • \- 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,

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Acibadem Labcell

Istanbul, Uskudar, 34662, Turkey (Türkiye)

RECRUITING

Related Publications (6)

  • 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

  • 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.

    PMID: 9891887BACKGROUND

  • 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.

    PMID: 21784508BACKGROUND

  • 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.

    PMID: 23907995BACKGROUND

  • 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.

    PMID: 21428760BACKGROUND

  • 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.

    PMID: 18598348BACKGROUND

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Ercument Ovali, Prof.Dr.

CONTACT

+905325729174ercument.ovali@acibademlabcell.com.tr

Cengiz Yakicier, Prof.Dr.

CONTACT

05362998743cengiz.yakicier@asg.com.tr

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Specialist, Haematology

Study Record Dates

First Submitted

November 5, 2014

First Posted

November 7, 2014

Study Start

November 1, 2013

Primary Completion

February 1, 2015

Study Completion

November 1, 2015

Last Updated

November 7, 2014

Record last verified: 2014-11

Locations

TU(1)