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Assessment of Efficacy of umbilical cord derived mesenchymal Stem Cells transplantation on movement improvement in patients with neuromuscular disorders

Protocol summary

Study aim
Efficacy of umbilical cord derived Mesenchymal Stem Cells transplantation on movement improvement in patients with neuromuscular disorders
Design
This study is phase 3 of clinical trial. In this study, there are four categories of inherited neuromuscular diseases. Each category includes 30 people of two 15 people of test and control.Blinding has not been done. Random allocation to two groups of test and control is using block randomization with four blocks. Sample size is 30 people in each category, with 4 categories, total number of patients is 60 people in control group and 60 people in test group.
Settings and conduct
The site of transplantation is in Farhikhtegan hospital. For each patient, four injections are given at the first injection, two weeks later, one month later, and again three months intervals. 3 million per kilogram body weight mesenchymal stem cell are transplanted into the patient's weak muscles under sedation conditions and under ultrasound guidance. After the cell injection, the patient is hospitalized and monitored for 24 hours.
Participants/Inclusion and exclusion criteria
Inclusion Criteria: Patients 4 to 35 yers old affected with genetic neuromusculae disorders. Exclusion Criteria: cancer; chromosomal defects; Patients with HIV, HCV, HBV, HTLV1,2; Dissatisfaction for entering the study.
Intervention groups
120 patients with four inherited neuromuscular diseases entered in this study. These patients were classified into 4 groups of 30 people. Each group consists of two groups of 15 test and 15 control. In patients of test group, mesenchymal stem cells are injected in weak muscles. There is no intervention in the control group. After 9 months, the clinical sign and symptoms, including the distance of walking for 6 minutes, were compared between the two groups.
Main outcome variables
Muscle strength enhancement with umbilical cord mesenchymal stem cell transplantation

General information

Reason for update
Acronym
IRCT registration information
IRCT registration number: IRCT20200426047213N1
Registration date: 2020-05-14, 1399/02/25
Registration timing: registered_while_recruiting

Last update: 2020-05-14, 1399/02/25
Update count: 0
Registration date
2020-05-14, 1399/02/25
Registrant information
Name
MOHAMMADKAZEM BAKHSHANDEH
Name of organization / entity
Country
Iran (Islamic Republic of)
Phone
+98 21 2640 0779
Email address
drbakhshandeh.com@gmail.com
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2019-10-23, 1398/08/01
Expected recruitment end date
2020-06-21, 1399/04/01
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Assessment of Efficacy of umbilical cord derived mesenchymal Stem Cells transplantation on movement improvement in patients with neuromuscular disorders
Public title
Efficacy of umbilical cord Mesenchymal Stem Cells on patients with neuromuscular disorders
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Genetic confirmed patients with Dystrophinopathy(Duchenne muscular dystrophy, Becker muscular dystrophy) Genetic confirmed patients with Neuropathy(Spinal muscular atrophy, Charcot-Marie-Tooth) Genetic confirmed patients with other Muscular dystrophy(Limb girdle muscular dystrophy, Congenital muscular dystrophy) Genetic confirmed patients with other Myopathy (Congenital myopathy, Congenital myasthenic syndrome)
Exclusion criteria:
Patients with cancer Patients with chromosomal defects Patients with HIV, HCV, HBV, HTLV1,2 Dissatisfaction for entering the study
Age
From 4 years old to 35 years old
Gender
Both
Phase
3
Groups that have been masked
No information
Sample size
Target sample size: 120
Randomization (investigator's opinion)
Randomized
Randomization description
In order to randomly assign to the two groups of test and control, the block random division method with four blocks was used. In this way, the letter A was assigned to the test group and the letter B to the control group. In four blocks with letters A and B, the six modes AABB, BBAA, ABAB, BABA, ABBA, BAAB were written on separate sheets and thrown inside a container and accidentally removed one of these sheets from the container. And the composition written on it was noted and again "that sheet was thrown into the container." Because the sample size in this study was 30 patients, this operation was repeated eight times, and each time the composition written on each sheet was written in the sequence of the composition written on the previous sheet. Then, one letter from one to thirty letters was assigned to each of the letters in order, and each letter was placed in an envelope and the number was written on the envelope. Each time the disease was selected, one of the envelopes was opened in order of the number written on the envelope, and it was determined that the patient should be in the test or control group.
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Parallel
Other design features
Patients are divided into 4 groups of 30 patients (15 for Test group and 15 for control patients).The first group of patients with Duchenne and Becker muscular dystrophy based on genetic testing, the second group of neuropathic patients such as Spinal Muscular Atrophy and Charcot-Marie-Thoth disease based on genetic testing, the third group of patients with other types of muscular dystrophy such as Limb girdle Muscular Dystrophy and Congenital muscular dystrophy Based on genetic testing, the fourth group are other inherited myopathies such as congenital myopathy and congenital myasthenic syndrome.

Secondary Ids

empty

Ethics committees

1

Ethics committee
Name of ethics committee
Research ethics committee of Tehran Islamic Azad university of Medical Sciences
Street address
Dr. MohammadKazem Bakhshandeh, Unit 6, Second floor, Mehr Afarin Doctors' Building, End of Morshedi Dead End, In Front of IranMehr Hospital, After Zafar St., Shariati St., Theran, Iran
City
Tehran
Province
Tehran
Postal code
1913776638
Approval date
2019-09-02, 1398/06/11
Ethics committee reference number
IR.IAU.TMU.REC.1398.104

Health conditions studied

1

Description of health condition studied
Dystrophinopathy(Duchenne muscular dystrophy, Becker muscular dystrophy)
ICD-10 code
G71.0
ICD-10 code description
Muscular dystrophy

2

Description of health condition studied
Spinal muscular atrophy
ICD-10 code
G12
ICD-10 code description
Spinal muscular atrophy and related syndromes

3

Description of health condition studied
other Myopathy (Congenital myopathy, Congenital myasthenic syndrome)
ICD-10 code
G71.2
ICD-10 code description
Congenital myopathies

4

Description of health condition studied
Charcot-Marie-Tooth)
ICD-10 code
G60.0
ICD-10 code description
Hereditary motor and sensory neuropathy

Primary outcomes

1

Description
Muscle power
Timepoint
Before study, 6 and 9 and 12 months after Mesenchymal stem cell transplantation
Method of measurement
Walking distance in 6 minutes and muscular force examination

Secondary outcomes

empty

Intervention groups

1

Description
Intervention group: In intervention group or test group the transplantation or injection of mesenchymal stem cell will be done. In this study the site of transplantation is in operation room of Farhikhtegan subspecialty hospital. Before the stem cell injection, the patient needs to be hospitalized for one day. For each patient, four injections are given at the first injection, two weeks later, one month later, and again three months intervals. 3 million per kilogram body weight mesenchymal stem cell are transplanted into the patient's weak muscles under sedation conditions and under ultrasound guidance. After the umbilical cord mesenchymal cell injection, the patient is hospitalized and monitored for 24 hours.
Category
Treatment - Other

2

Description
Control group: For each patient in Test group, there is a patient with same genetic mutation in control group. There is no intervention in control group. After determined intervention the clinical feature of these two group will be compared.
Category
Treatment - Other

Recruitment centers

1

Recruitment center
Name of recruitment center
Farhikhtegan subspecialty hospital
Full name of responsible person
Mohammad Kazem Bakhshandeh
Street address
Dr. MohammadKazem Bakhshandeh, Unit 6, Second floor, Mehr Afarin Doctors' Building, End of Morshedi Dead End, In Front of IranMehr Hospital, After Zafar St., Shariati St., Theran, Iran
City
Tehran
Province
Tehran
Postal code
1913776638
Phone
+98 21 2640 0779
Email
drbakhshandeh.com@gmail.com
Web page address
https://www.irpnc.com/

Sponsors / Funding sources

1

Sponsor
Name of organization / entity
Tehran Islamic Azad university of Medical Sciences
Full name of responsible person
Mohammad Kazem Bakhshandeh
Street address
Dr. Mohammad Kazem Bakhshandeh, Unit 6, Second floor, Mehr Afarin Doctors' Building, End of Morshedi Dead End, In Front of IranMehr Hospital, After Zafar St., Shariati St., Theran, Iran
City
Tehran
Province
Tehran
Postal code
1913776638
Phone
+98 21 2640 0779
Email
drbakhshandeh.com@gmail.com
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
No
Title of funding source
Tehran Islamic Azad university of Medical Sciences
Proportion provided by this source
20
Public or private sector
Private
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 Islamic Azad university of Medical Sciences
Full name of responsible person
Mohammad Kazem Bakhshandeh
Position
Assistant professor
Latest degree
Subspecialist
Other areas of specialty/work
Pediatric neurologist, neuromuscular fellowship
Street address
Dr. MohammadKazem Bakhshandeh, Unit 6, Second floor, Mehr Afarin Doctors' Building, End of Morshedi Dead End, In Front of IranMehr Hospital, After Zafar St., Shariati St., Theran, Iran
City
Tehran
Province
Tehran
Postal code
1913776638
Phone
+98 21 2640 0779
Email
drbakhshandeh.com@gmail.com

Person responsible for scientific inquiries

Contact
Name of organization / entity
Tehran Islamic Azad university of Medical Sciences
Full name of responsible person
Mohammad Kazem Bakhshandeh
Position
Assistant professor
Latest degree
Subspecialist
Other areas of specialty/work
pediatric neurologist, neuromuscular fellowship
Street address
Dr. MohammadKazem Bakhshandeh, Unit 6, Second floor, Mehr Afarin Doctors' Building, End of Morshedi Dead End, In Front of IranMehr Hospital, After Zafar St., Shariati St., Theran, Iran
City
Tehran
Province
Tehran
Postal code
1913776638
Phone
+98 21 2640 0779
Email
drbakhshandeh.com@gmail.com
Web page address
https://www.irpnc.com/

Person responsible for updating data

Contact
Name of organization / entity
Tehran Islamic Azad university of Medical Sciences
Full name of responsible person
Mohammad Kazem Bakhshandeh
Position
Assistant professor
Latest degree
Subspecialist
Other areas of specialty/work
Pediatric neurologist, neuromuscular fellowship
Street address
Dr. MohammadKazem Bakhshandeh, Unit 6, Second floor, Mehr Afarin Doctors' Building, End of Morshedi Dead End, In Front of IranMehr Hospital, After Zafar St., Shariati St., Theran, Iran
City
Tehran
Province
Tehran
Postal code
1913776638
Phone
+98 21 2640 0779
Email
drbakhshandeh.com@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
After 12 months we will publish the analysis of datas
When the data will become available and for how long
After 12 months we will publish the analysis of datas
To whom data/document is available
All interested researchers
Under which criteria data/document could be used
After 12 months we will publish the analysis of datas
From where data/document is obtainable
Mohammadkazem Bakhshandeh
What processes are involved for a request to access data/document
Official request from research department
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