Evaluation of safety and efficacy of focal intramuscular injection of wharton’s gelly-derived mesenchymal stem cell in Duchenne muscular dystrophy pediatrics between 4 to 20 years old
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Protocol summary
The clinical trial is performed on 16 patients who have referred for treatment and are followed up for one year.
The clinical trial is performed on 16 patients who have referred for treatment and are followed up for 9 months.
The clinical trial is performed on 16 patients who have referred for treatment and are followed up for one year9 months.
کارآزمایی بالینی بر روی 16 نفر از بیماران که به جهت شروع درمان مراجعه کرده اند انجام میشود و به مدت یک سال پیگیری میشوند.
کارآزمایی بالینی بر روی 16 نفر از بیماران که به جهت شروع درمان مراجعه کرده اند انجام میشود و به مدت 9 ماه پیگیری میشوند.
کارآزمایی بالینی بر روی 16 نفر از بیماران که به جهت شروع درمان مراجعه کرده اند انجام میشود و به مدت یک سال9 ماه پیگیری میشوند.
General information
2024-06-01, 1403/03/12
2024-03-01, 1402/12/11
2024-0603-01 00:00:00
Deletion of one of Inclusion and Exclusion Criteria
Deletion of one of Inclusion and Exclusion Criteria
Cancellation of visit 12 months after injection (patient non-compliance due to prolonged research project).
Deletion of one of Inclusion and Exclusion Criteria Cancellation of visit 12 months after injection (patient non-compliance due to prolonged research project).
حذف یکی از موارد شرایط ورود و یکی از موارد عدم ورود
حذف یکی از موارد شرایط ورود و یکی از موارد عدم ورود
حذف ویزیت 12 ماه پس از تزریق (عدم همکاری بیماران به علت طولانی بودن مدت طرح پژوهشی)
حذف یکی از موارد شرایط ورود و یکی از موارد عدم ورود حذف ویزیت 12 ماه پس از تزریق (عدم همکاری بیماران به علت طولانی بودن مدت طرح پژوهشی)
Primary outcomes
#1
1, 3, 6, 9 and 12 month after injection
1, 3, 6, and 9 months after injection
1, 3, 6, and 9 and 12 monthmonths after injection
1، 3، 6، 9 و 12 ماه بعد از تزریق
1، 3، 6 و 9 ماه بعد از تزریق
1، 3، 6، 9 و 129 ماه بعد از تزریق
#2
First every 14 days to 1 month, then Every 1 month to 1 year
First every 14 days to 1 month, then Every 1 month to 9 months
First every 14 days to 1 month, then Every 1 month to 1 year9 months
ابتدا هر 14 روز تا یک ماه، سپس هر 1 ماه تا یک سال
ابتدا هر 14 روز تا یک ماه، سپس هر 1 ماه تا 9 ماه
ابتدا هر 14 روز تا یک ماه، سپس هر 1 ماه تا یک سال9 ماه
#3
1, 3, 6, 9 and 12 month after injection
1, 3, 6, and 9 months after injection
1, 3, 6, and 9 and 12 monthmonths after injection
1، 3، 6، 9 و 12 ماه بعد از تزریق
1، 3، 6 و 9 ماه بعد از تزریق
1، 3، 6، 9 و 129 ماه بعد از تزریق
Secondary outcomes
#1
1, 3, 6, 9 and 12 month after injection
1, 3, 6, and 9 months after injection
1, 3, 6, and 9 and 12 monthmonths after injection
ماه 1، 3، 6، 9 و 12 بعد از تزریق
ماه 1، 3، 6،و 9 ماه بعد از تزریق
ماه 1، 3، 6،و 9 و 12ماه بعد از تزریق
Protocol summary
Study aim
Evaluation of therapeutic effects and complications of treatment of Duchenne muscular dystrophy with stem cells
Design
The clinical trial is performed on 16 patients who have referred for treatment and are followed up for 9 months.
Settings and conduct
Two injections of 1 million/kg stem cells derived from Wharton gel in 4 muscles of 16 children with Duchenne dystrophy
Participants/Inclusion and exclusion criteria
Inclusion criteria: age 5 to 12 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; weight between 15 to 35. 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; 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.
Intervention groups
In each injection, 1 x 106 mesenchymal cell derived from Wharton's gelly is injected per kilogram of body weight. The injection volume of 25 cc includes a suspension containing one million mesenchymal cells, two local injections are performed in 4 to 5 areas under the muscle fascia at an interval of 6 months.
Main outcome variables
Respiratory Function(FVC); Muscular Function(physical examination and lab study); Cardiac Function(EF)
General information
Reason for update
Deletion of one of Inclusion and Exclusion Criteria
Cancellation of visit 12 months after injection (patient non-compliance due to prolonged research project).
Acronym
IRCT registration information
IRCT registration number:IRCT20150206020981N5
Registration date:2023-05-27, 1402/03/06
Registration timing:prospective
Last update:2024-07-23, 1403/05/02
Update count:4
Registration date
2023-05-27, 1402/03/06
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
2023-06-02, 1402/03/12
Expected recruitment end date
2024-03-01, 1402/12/11
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Evaluation of safety and efficacy of focal intramuscular injection of wharton’s gelly-derived mesenchymal stem cell in Duchenne muscular dystrophy pediatrics between 4 to 20 years old
Public title
Evaluation of safety and efficacy of focal intramuscular injection of wharton’s gelly-derived mesenchymal stem cell in Duchenne muscular dystrophy pediatrics between 4 to 20 years old
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Age between 4 to 20
Genetic evidence of Duchenne disease as a cause of myopathy
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
Weight between 14 to 75
Exclusion criteria:
A history of known malignancy
History of associated genetic and non-genetic degenerative diseases
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.
Age
From 4 years old to 20 years old
Gender
Male
Phase
1-2
Groups that have been masked
No information
Sample size
Target sample size:
16
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 of Islamic Azad University of Medical Sciences, Farhikhtegan Hospital
First every 14 days to 1 month, then Every 1 month to 9 months
Method of measurement
Physical Exam and dynamometer
3
Description
Cardiac Function
Timepoint
1, 3, 6, and 9 months after injection
Method of measurement
Echocardiography
Secondary outcomes
1
Description
Muscular Enzymes
Timepoint
1, 3, 6, and 9 months after injection
Method of measurement
Lab Data
Intervention groups
1
Description
Intervention group: Intervention group: In each injection, 1million mesenchymal cell derived from Wharton's gelly is injected per kilogram of body weight. The injection volume of 25 cc includes a suspension containing one million mesenchymal cells, two local injections are performed in 4 to 5 areas under the muscle fascia at an interval of 6 months.