Determination of the safety of using allogeneic mesenchymal stem cells by pilot method in patients with cystic fibrosis referred to the Children's Medical Center
Determining the safety of use of allogeneic mesenchymal stem cell in cystic fibrosis patients
Design
Double-blind randomized clinical trial with control and
parallel groups
Settings and conduct
10 patients with definite diagnosis of cystic fibrosis referred to Tehran Children's Medical Center Hospital will be included in the study according to inclusion and exclusion criteria. Eligible people will be randomly divided into two groups of 5 people. Everyone will be tested for PFT and 6MWT. 5 patients received placebo as a single dose in the peripheral vein and 5 patients with a dose of 20 million cells as a single dose. In the peripheral vein, human allogeneic mesenchymal stem cells will be injected. Patients will be evaluated for side effects up to 30 days after injection. Then, at the end of the year, the tests that were performed at the beginning of the study as basic tests will be repeated.
Participants/Inclusion and exclusion criteria
Inclusion criteria: severe obstructive disease defined as FEV1 / FVC less than 50%; acceptable function of other organs including creatinine level less than 1.5 times normal limit; ALT level less than 135; AST level less than 3 times normal limit; normal heart function on echo cardiogram; patients in the age range of 12 to 18 years old.
Exclusion criteria: the presence of any viral or bacterial infection that has caused pneumonia; patients with Heart Failure; patients with mental and physical disabilities.
Intervention groups
Patients with cystic fibrosis were randomly divided into placebo and intervention groups.Five patients were injected single-dose placebo into a peripheral vein, and another 5 patients were injected into a peripheral human allogeneic mesenchymal stem cell, or Allo-hMSC for short, at a dose of 20 million cells per single dose.
Main outcome variables
Pulmonary function tests
Body Mass Index
six minute walking test
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20211110053033N1
Registration date:2022-01-02, 1400/10/12
Registration timing:registered_while_recruiting
Last update:2022-01-02, 1400/10/12
Update count:0
Registration date
2022-01-02, 1400/10/12
Registrant information
Name
Faezeh Bolboli
Name of organization / entity
Country
Iran (Islamic Republic of)
Phone
+98 11 4422 4526
Email address
faezeh.bolboli@gmail.com
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2021-12-26, 1400/10/05
Expected recruitment end date
2022-03-16, 1400/12/25
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Determination of the safety of using allogeneic mesenchymal stem cells by pilot method in patients with cystic fibrosis referred to the Children's Medical Center
Public title
Determination of safety of allogeneic mesenchymal stem cells by pilot method in patients with cystic fibrosis
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Definitive diagnosis of cystic fibrosis
Severe obstructive disease defined as FEV1 / FVC less than 50%
Acceptable function of other organs including creatinine level less than 1.5 times normal limit in each age group, ALT level less than 135, AST level less than 3 times normal limit in each age group, bilirubin level less than 1.5 times Normal limit in any age group
Normal heart function on echocardiogram
Patients in the age range of 12 to 18 years
Exclusion criteria:
The presence of any viral or bacterial infection that has caused pneumonia
Patients with Heart Failure
Patients with mental and physical disabilities
Age
From 12 years old to 18 years old
Gender
Both
Phase
N/A
Groups that have been masked
Participant
Investigator
Sample size
Target sample size:
10
Randomization (investigator's opinion)
Randomized
Randomization description
Randomized numbers from one to 10 was produced with a calculator, the first five numbers allocated to intervention group and the other five numbers allocated to control group.Then drugs sequentially from 1 to 10, was given to researcher for prescription to the patients.
Blinding (investigator's opinion)
Double blinded
Blinding description
Drugs and placebos are the same in terms of appearance, color, etc., and thus patients and researchers will not know the contents.
Placebo
Used
Assignment
Parallel
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Ethics committee of Tehran University of Medical Sciences
Street address
Children's Medical Center, Keshavarz Blvd.
City
Tehran
Province
Tehran
Postal code
14197 33151
Approval date
2021-07-07, 1400/04/16
Ethics committee reference number
IR.TUMS.CHMC.REC.1400.073
Health conditions studied
1
Description of health condition studied
Cystic fibrosis
ICD-10 code
E84
ICD-10 code description
Cystic fibrosis
Primary outcomes
1
Description
Lung Volume (Forced expiratory volume in one second);(FEV1)
Forced expiratory volume in 1 second to forced vitalcapacity (FEV1/FVC)
Timepoint
At the first visit and follow-up for up to a year
Method of measurement
Spirometry
5
Description
Walking distance upon 6 minute walking distance test(meter)
Timepoint
At the first visit and follow-up for up to a year
Method of measurement
Six minute walking distance test
6
Description
Body Mass Index
Timepoint
At the first visit and follow-up for up to a year
Method of measurement
Weight (kilogram) divided by height squared (centimetre)
Secondary outcomes
empty
Intervention groups
1
Description
Intervention group: In 5 patients with cystic fibrosis in the peripheral vein, human allogeneic mesenchymal stem cells (Allo-hMSC ) will be injected in a single dose of 20 million cells.
Category
Treatment - Drugs
2
Description
Control group: In 5 patients with cystic fibrosis, the placebo will be injected as a single dose into the peripheral vein.
Category
Placebo
Recruitment centers
1
Recruitment center
Name of recruitment center
Children's Medical Center
Full name of responsible person
Mohammadreza Modarresi
Street address
Keshavarz Blvd
City
Tehran
Province
Tehran
Postal code
14197 33151
Phone
+98 21 6147 0000
Email
mohammadreza.modaresi@tums.ac.ir
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Tehran University of Medical Sciences
Full name of responsible person
Akbar Fotouhi
Street address
Central Campus of the University of Tehran, Vice Chancellor for Research, 16 Azar St., Enghelab St., Enghelab Square
City
Tehran
Province
Tehran
Postal code
1417614411
Phone
+98 21 6649 8813
Email
resaerch@ut.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Tehran University of Medical Sciences
Proportion provided by this source
100
Public or private sector
Public
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Academic
Person responsible for general inquiries
Contact
Name of organization / entity
Tehran University of Medical Sciences
Full name of responsible person
Faezeh Bolboli
Position
Lung Specialist Assistant
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
Keshavarz Blvd
City
Tehran
Province
Tehran
Postal code
14197 33151
Phone
+98 21 6147 0011
Email
faezeh.bolboli@gmail.com
Person responsible for scientific inquiries
Contact
Name of organization / entity
Tehran University of Medical Sciences
Full name of responsible person
Faezeh Bolboli
Position
Lung Specialist Assistant
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
Keshavarz Blvd
City
Tehran
Province
Tehran
Postal code
14197 33151
Phone
+98 21 6147 0011
Email
faezeh.bolboli@gmail.com
Person responsible for updating data
Contact
Name of organization / entity
Tehran University of Medical Sciences
Full name of responsible person
Fazezeh Bolboli
Position
Lung Specialist Assistant
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
Keshavarz Blvd
City
Tehran
Province
Tehran
Postal code
14197 33151
Phone
+98 21 6147 0011
Email
faezeh.bolboli@gmail.com
Sharing plan
Deidentified Individual Participant Data Set (IPD)
Yes - There is a plan to make this available
Study Protocol
Yes - There is a plan to make this available
Statistical Analysis Plan
Yes - There is a plan to make this available
Informed Consent Form
Yes - There is a plan to make this available
Clinical Study Report
Yes - There is a plan to make this available
Analytic Code
Yes - There is a plan to make this available
Data Dictionary
Yes - There is a plan to make this available
Title and more details about the data/document
Information on the main outcome or the like can be shared.
When the data will become available and for how long
Available access 9 months after the publication of results
To whom data/document is available
no
Under which criteria data/document could be used
no
From where data/document is obtainable
Dr. Faezeh Bolboli
What processes are involved for a request to access data/document