Evaluation of therapeutic effects and complications of treatment of Duchenne muscular dystrophy with stem cells
Design
The clinical trial is performed on 10 patients who have referred for treatment and are followed up for one year.
Settings and conduct
After 300 to 600 cc of subcutaneous adipose tissue is extracted from the patient's abdomen and 100 to 150 cc of SVF is prepared, the resulting product is injected intravenously into the patient.
Participants/Inclusion and exclusion criteria
Inclusion criteria: age 7 to 30 years old; genetic evidence; the patient's ability to walk is not completely lost and the use of assistive devices is not permanent; the patient's muscle strength is decreasing; willingness and satisfaction to participate in the study.
Exclusion criteria: advanced motor impairment leading to permanent use of crutches, walkers and wheelchairs; history of known malignancy; history of associated genetic and non-genetic degenerative diseases; having blood and internal diseases that increase the risk of liposuction and anesthesia required; history of any other acute or chronic illness that, at the discretion of the clinician responsible for implementing the plan, prevents the patient from entering the study; insufficient volume of subcutaneous fat or the presence of concomitant injuries in the abdomen.
Intervention groups
100 to 150 cc of SVF product containing 20% mesenchymal cells, 60% progenitor cells and 20% other autologous cells in albumin medium will be injected intravenously through peripheral vein once in 10 to 30 minutes.
Main outcome variables
Respiratory Function(FVC); Muscular Function(physical examination and lab study); Cardiac Function(EF)
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20150206020981N3
Registration date:2022-06-07, 1401/03/17
Registration timing:registered_while_recruiting
Last update:2022-06-07, 1401/03/17
Update count:0
Registration date
2022-06-07, 1401/03/17
Registrant information
Name
Khadijeh Hajinaghitehrani
Name of organization / entity
Azad Medical University
Country
Iran (Islamic Republic of)
Phone
+98 21 2257 1228
Email address
khtehrani@iautmu.ac.ir
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2022-05-31, 1401/03/10
Expected recruitment end date
2023-05-31, 1402/03/10
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Evaluation of safety and efficacy of autologous adipose-derived mesenchymal cells therapy in Duchenne muscular dystrophy patients
Public title
Efficacy of autologous adipose tissue-derived mesenchymal stem cell therapy in Duchenne muscular dystrophic patients
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Genetic evidence of Duchenne disease as a cause of myopathy
The patient's ability to walk is not completely lost and the use of assistive devices such as canes and walkers is not permanent.
The patient's muscle strength is decreasing
Willingness and satisfaction to participate in the study by the patient's legal guardian in decision making and willingness to participate regularly in follow-up courses
Exclusion criteria:
Advanced motor impairment leading to permanent use of crutches, walkers and wheelchairs
A history of known malignancy
History of associated genetic and non-genetic degenerative diseases
Having blood and internal diseases that increase the risk of liposuction and anesthesia required
A history of any other acute or chronic illness that, at the discretion of the clinician responsible for implementing the plan, prevents the patient from entering the study.
Insufficient volume of subcutaneous fat or the presence of concomitant injuries in the abdomen in the initial examination that make it difficult to obtain adipose tissue to prepare SVF.
Age
From 7 years old to 30 years old
Gender
Male
Phase
N/A
Groups that have been masked
No information
Sample size
Target sample size:
10
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
Ethics Committee on Research Islamic Azad University, Tehran Medical Branch
Street address
Attari Moghadam Ave, Khaghani Ave, Shariati Ave, Islamic Azad University, Tehran Medical Branch
City
Tehran
Province
Tehran
Postal code
1936893813
Approval date
2022-04-26, 1401/02/06
Ethics committee reference number
IR.IAU.TMU.REC.1401.038
Health conditions studied
1
Description of health condition studied
Duchenne muscular dystrophy
ICD-10 code
G71.01
ICD-10 code description
Muscular dystrophy
Primary outcomes
1
Description
Respiratory Function
Timepoint
1, 3, 6, 9 and 12 month after injection
Method of measurement
Spirometery
2
Description
Muscular Force
Timepoint
First every 14 days to 1 month, then Every 1 month to 1 year
Method of measurement
Physical Exam
3
Description
Cardiac Function
Timepoint
1, 3, 6, 9 and 12 month after injection
Method of measurement
Echocardiography
Secondary outcomes
1
Description
Muscular Enzymes
Timepoint
1, 3, 6, 9 and 12 month after injection
Method of measurement
Lab Data
Intervention groups
1
Description
Intervention group: 100 to 150 cc of SVF product containing 20% mesenchymal cells, 60% progenitor cells and 20% other autologous cells in albumin medium will be injected intravenously through peripheral vein once in 10 to 30 minutes.
Category
Treatment - Other
Recruitment centers
1
Recruitment center
Name of recruitment center
Iranian Muscular Dystrophy Association
Full name of responsible person
Dr. Khadijeh Hajinaghi Tehrani
Street address
No 27, Montazeri St. Piroozi St. Shohada Square
City
Tehran
Province
Tehran
Postal code
17117
Phone
+98 21 3660 7093
Email
dr_tehrani10@yahoo.com
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Research Deputy of Islamic Azad University, Tehran Medical Branch
Full name of responsible person
Farshad Hashemian
Street address
Attari Moghadam Ave, Khaghani Ave, Shariati Ave, Islamic Azad University, Tehran Medical Branch
City
Tehran
Province
Tehran
Postal code
1936893813
Phone
+98 21 2200 6660
Email
hashemian.f@iaups.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Research Deputy of Islamic Azad University, Tehran Medical Branch
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
Bu-ali Hospital
Full name of responsible person
Khadijeh Hajinaghi Tehrani
Position
Neurologist
Latest degree
Specialist
Other areas of specialty/work
Neurology
Street address
Damavand St. Imam Hosein Sq.
City
Tehran
Province
Tehran
Postal code
17117
Phone
+98 21 3334 8035
Email
Khtehrani@iautmu.ac.ir
Person responsible for scientific inquiries
Contact
Name of organization / entity
Bu-ali Hospital
Full name of responsible person
Khadijeh Hajinaghi Tehrani
Position
Neurologist
Latest degree
Specialist
Other areas of specialty/work
Neurology
Street address
Damavaand St. Imam Hosein Sq.
City
Tehran
Province
Tehran
Postal code
17117
Phone
+98 21 3334 8035
Email
Khtehrani@iautmu.ac.ir
Person responsible for updating data
Contact
Name of organization / entity
Farhikhtegan Hospital
Full name of responsible person
Fatemeh Naseri Atashani
Position
MD
Latest degree
Medical doctor
Other areas of specialty/work
General Practitioner
Street address
Daneshgah Sq. North Sattari highway
City
Tehran
Province
Tehran
Postal code
1477899679
Phone
+98 21 4486 7262
Email
fatemeh_naseriatashani@yahoo.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
Undecided - It is not yet known if there will be a plan to make this available
Analytic Code
Undecided - It is not yet known if there will be a plan to make this available
Data Dictionary
Undecided - It is not yet known if there will be a plan to make this available
Title and more details about the data/document
The only some part of data will be shared.
When the data will become available and for how long
5 months after publish
To whom data/document is available
Only academic researcher
Under which criteria data/document could be used
By official permission
From where data/document is obtainable
By the corresponding author
What processes are involved for a request to access data/document
By the official request to the corresponding author, if she/he agrees to it.