Protocol summary

Summary
Our objective is to apply autologous bone marrow-derived Mesenchymal Stem Cells (MSCs) for refractory Crohn’s disease; then, determine immunomodulatory effects of MSCs into TH1 and TH17 immune responses, and in the treatment of refractory Crohn’s disease patients. Seventeen adult patients with refractory Crohn’s disease will be entered to the study. Criteria for patient inclusion is that patients at least 18 years of age and have moderate to severe Crohn’s disease, as define by a baseline Crohn’s Disease Activity Index (CDAI) score between 220 and 450. Furthermore, patients have to be refractory to the standard treatment options for Crohn’s disease. We define refractory patients as patients that, at some time during the course of the disease, must have received steroids, immunosuppressive agents (eg, azathioprine, 6-mercaptopurine or methotrexate) or anti-TNF therapy which did not result in an adequate response to treatment. Colonoscopy will be performed at baseline to confirm disease activity. All patients will be taken informed consent. Patients will be underwent bone marrow aspiration under local anesthesia, and Bone marrow MSCs will be isolated and will be expanded ex vivo. Two intravenous infusions of 0.5 to 2 ×106 cell/kg a week apart will be used. Gene expression as well as serum levels of TGFβ, IL-17A, IL-10, IL-4,IL-23,and IFN-γ Cytokines will be determined by RT-PCR and ELISA, respectively. Also, TH1, TH17, and Treg Subpopulations will be measured by flowcytometry.

General information

Acronym
CD: Crohn's Disease, MSCs: Mesenchymal Stem Cells
IRCT registration information
IRCT registration number: IRCT2016110630726N1
Registration date: 2017-05-10, 1396/02/20
Registration timing: prospective

Last update:
Update count: 0
Registration date
2017-05-10, 1396/02/20
Registrant information
Name
Jalil Tavakolafshari
Name of organization / entity
Immunogenetic &cell culture department, Immunology Research center,BuAli Research Institue
Country
Iran (Islamic Republic of)
Phone
+98 51376276015
Email address
nazarih931@mums.ac.ir
Recruitment status
Recruitment complete
Funding source
Vice Chancellor for Research and Technology Mashhad University of Medical Scienes
Expected recruitment start date
2017-05-22, 1396/03/01
Expected recruitment end date
2018-09-21, 1397/06/30
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Evaluation TH1 and TH17 immune response mechanisms involved in the pathogenesis of Crohn's disease, following intravenous injection of autologous mesenchymal stem cells in refractory Crohn's disease patients.
Public title
Applying of stem cells to treat crohn's disease
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:Patients at least 18 years of age; Crohn’s Disease Activity Index (CDAI) score between 220 and 450; Colonoscopy performe at baseline to confirm disease activity; Patients have to be refractory to the standard treatment options for Crohn’s disease; such as: steroids, immunosuppressive agents (eg, azathioprine, 6-mercaptopurine or methotrexate) or anti-TNF therapy. Exclusion criteria:History of malignancy during the course of treatment or during the last 5 years.
Age
From 18 years old
Gender
Both
Phase
1-2
Groups that have been masked
No information
Sample size
Target sample size: 17
Randomization (investigator's opinion)
N/A
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Single
Other design features

Secondary Ids

empty

Ethics committees

1

Ethics committee
Name of ethics committee
Regional Ethics Committee Mashhad University of Medical Sciences
Street address
Ghoreishi Building, Next to the cinema Hoveizeh , University Avenue, Mashhad, Iran.
City
Mashhad
Postal code
91735-951
Approval date
2017-02-20, 1395/12/02
Ethics committee reference number
IR.MUMS.REC.1395.493

Health conditions studied

1

Description of health condition studied
Crohn disease
ICD-10 code
K50.9
ICD-10 code description
Crohn disease, unspecified

Primary outcomes

1

Description
Interleukin 23 level
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
ELISA, Pg/ml

2

Description
IFN-γ level
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
ELISA, Pg/ml

3

Description
Interleukin 10 level
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
ELISA, Pg/ml

4

Description
Interleukin 4 level
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
ELISA, Pg/ml

5

Description
TGF-β level
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
ELISA, Pg/ml

6

Description
Th1 cell percentage
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
flow cytometry, percentage

7

Description
Treg cell percentage
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
flow cytometry, percentage

8

Description
Th17 cell percentage
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
flow cytometry, percentage

9

Description
Interleukin 17 level
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
ELISA. Pg/ml

10

Description
Interleukin 17 gene expression
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
QRT-PCR

11

Description
Interleukin 23 gene expression
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
QRT-PCR

12

Description
Interleukin 10 gene expression
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
QRT-PCR

13

Description
Interleukin 4 gene expression
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
QRT-PCR

14

Description
TGF-β gene expression
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
QRT-PCR

15

Description
IFN-γ gene expression
Timepoint
Before intervention, Three months after intervention, six months after intervention
Method of measurement
QRT-PCR

Secondary outcomes

1

Description
CBC
Timepoint
Before intervention
Method of measurement
cells/L ، g/L ، fL (femtoliters)، pg (picograms)

2

Description
Liver enzymes
Timepoint
Before intervention
Method of measurement
U/L

3

Description
Stool Exam
Timepoint
Before intervention
Method of measurement
Without unit

4

Description
Crohn's Disease Activity Index (CDAI)
Timepoint
Before intervention, Six months after intervention
Method of measurement
Standard form, SCORE

5

Description
CRP
Timepoint
Before treatment
Method of measurement
mg/L

6

Description
ESR
Timepoint
Before intervention
Method of measurement
mm/hr

Intervention groups

1

Description
Phase 1/2 Clinical Trial. Intravenous infusion autologous Bone Marrow-derived Mesenchymal Stem Cells (MSCs) for refractory Crohn’s disease. Two dose infusions of 0.5 to 2 ×106 cell/kg with a week interval.
Category
Treatment - Other

Recruitment centers

1

Recruitment center
Name of recruitment center
Gaem hospital, Mashhad University of Medical Sciences, Mashhad, Iran.
Full name of responsible person
Dr. Hasan Vosoughinia
Street address
Gaem Hospital, Shariati Sq, Mashhad, Iran
City
Mashhad

Sponsors / Funding sources

1

Sponsor
Name of organization / entity
Vice Chancellor for Research and Technology University, Mashhad University of Medical Sciences
Full name of responsible person
Dr.Mohsen Tafaghodi
Street address
Vice Chancellor for Research and Technology University, Ghoreishi Building, next to the cinema Hoveize, University Avenue, Mashhad, Iran
City
Mashhad
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Vice Chancellor for Research and Technology University, Mashhad University of Medical Sciences
Proportion provided by this source
100
Public or private sector
empty
Domestic or foreign origin
empty
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
empty

Person responsible for general inquiries

Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Haniyeh Ghaffari Nazari
Position
Ph.D student in Medical immunology
Other areas of specialty/work
Street address
Immunogenetic & Tissue Culture Department, Bu Ali Research Institute, BuAli sq, Mashhad, Iran
City
Mashhad
Postal code
Phone
+98 51 3711 2611
Fax
Email
nazarih931@mums.ac.ir
Web page address

Person responsible for scientific inquiries

Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Jalil Tavakkoli Afshari
Position
M.Sc., Ph.D., MT(ASCP), CLS(NCA) immunology
Other areas of specialty/work
Street address
Immunogenetic & cell culture department, Immunology Research Center, BuAli Research Institue
City
Mashhad
Postal code
Phone
+98 51376276015
Fax
Email
tavakolaj@mums.ac.irtavakolafshari@gmail.com
Web page address

Person responsible for updating data

Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Haniyeh Ghaffari Nazari
Position
Ph.D student in Medical immunology
Other areas of specialty/work
Street address
Immunogenetic & Tissue Culture Department, Bu Ali Research Institute, BuAli sq, Mashhad, Iran
City
Postal code
Phone
00
Fax
Email
nazarih931@mums.ac.ir
Web page address

Sharing plan

Deidentified Individual Participant Data Set (IPD)
empty
Study Protocol
empty
Statistical Analysis Plan
empty
Informed Consent Form
empty
Clinical Study Report
empty
Analytic Code
empty
Data Dictionary
empty
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