Protocol summary
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Study aim
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Evaluation of therapeutic effects and complications of treatment of Duchenne muscular dystrophy with stem cells
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Design
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The clinical trial is performed on 16 patients who have referred for treatment and are followed up for 9 months.
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Settings and conduct
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Two injections of 1 million/kg stem cells derived from Wharton gel in 4 muscles of 16 children with Duchenne dystrophy
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Participants/Inclusion and exclusion criteria
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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.
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Intervention groups
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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.
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Main outcome variables
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Respiratory Function(FVC); Muscular Function(physical examination and lab study); Cardiac Function(EF)
General information
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Reason for update
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Deletion of one of Inclusion and Exclusion Criteria
Cancellation of visit 12 months after injection (patient non-compliance due to prolonged research project).
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Acronym
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IRCT registration information
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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
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Registration date
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2023-05-27, 1402/03/06
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Registrant information
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Recruitment status
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Recruitment complete
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Funding source
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Expected recruitment start date
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2023-06-02, 1402/03/12
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Expected recruitment end date
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2024-03-01, 1402/12/11
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Actual recruitment start date
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empty
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Actual recruitment end date
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empty
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Trial completion date
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empty
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Scientific title
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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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Public title
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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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Purpose
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Treatment
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Inclusion/Exclusion criteria
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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.
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Age
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From 4 years old to 20 years old
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Gender
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Male
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Phase
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1-2
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Groups that have been masked
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No information
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Sample size
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Target sample size:
16
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Randomization (investigator's opinion)
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N/A
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Randomization description
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Blinding (investigator's opinion)
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Not blinded
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Blinding description
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Placebo
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Not used
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Assignment
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Single
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Other design features
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Ethics committees
1
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Ethics committee
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Approval date
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2023-05-21, 1402/02/31
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Ethics committee reference number
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IR.IAU.FARHIKHTEGANH.REC.1402.001
Health conditions studied
1
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Description of health condition studied
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Duchenne muscular dystrophy
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ICD-10 code
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G71.0
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ICD-10 code description
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Muscular dystrophy
Primary outcomes
1
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Description
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Respiratory Function
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Timepoint
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1, 3, 6, and 9 months after injection
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Method of measurement
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Spirometery
2
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Description
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Muscular Force
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Timepoint
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First every 14 days to 1 month, then Every 1 month to 9 months
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Method of measurement
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Physical Exam and dynamometer
3
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Description
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Cardiac Function
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Timepoint
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1, 3, 6, and 9 months after injection
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Method of measurement
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Echocardiography
Secondary outcomes
1
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Description
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Muscular Enzymes
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Timepoint
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1, 3, 6, and 9 months after injection
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Method of measurement
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Lab Data
Intervention groups
1
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Description
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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.
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Category
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Treatment - Drugs
1
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Sponsor
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Grant name
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Grant code / Reference number
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Is the source of funding the same sponsor organization/entity?
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Yes
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Title of funding source
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Sinacell Company
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Proportion provided by this source
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100
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Public or private sector
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Private
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Domestic or foreign origin
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Domestic
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Category of foreign source of funding
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empty
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Country of origin
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Type of organization providing the funding
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Industry
Sharing plan
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Deidentified Individual Participant Data Set (IPD)
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Yes - There is a plan to make this available
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Study Protocol
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Yes - There is a plan to make this available
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Statistical Analysis Plan
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Yes - There is a plan to make this available
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Informed Consent Form
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Yes - There is a plan to make this available
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Clinical Study Report
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Undecided - It is not yet known if there will be a plan to make this available
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Analytic Code
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Undecided - It is not yet known if there will be a plan to make this available
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Data Dictionary
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Undecided - It is not yet known if there will be a plan to make this available
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Title and more details about the data/document
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The only some part of data will be shared.
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When the data will become available and for how long
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5 months after publish
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To whom data/document is available
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Only academic researcher
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Under which criteria data/document could be used
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Only academic researcher
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From where data/document is obtainable
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By official permission
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What processes are involved for a request to access data/document
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By the official request to the corresponding author, if she/he agrees to it.
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Comments
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