1
Clinical Trial Protocol
Iranian Registry of Clinical Trials
05 Nov 2022
Complications of intravenous transplantation of allogeneic tissue-derived mesenchymal stem cells in patients with ALS phase 1 clinical trial
Protocol summary
Study aim
The main aim: The occurrence of side effects following intravenous injection of cells Secondary aim: The effect of cell transplantation on ALS-FRS, FVC, ASLAQ-40 scores, and EMG-NCV
Design
Clinical trial, single-group, not-blinded, phase 1 on 15 patients. Randomization is not applicable to this study.
Settings and conduct
The cell suspension prepared from the adipose tissue cell bank of Royan Research Institute is injected
intravenously into the patients in the hospital. Within 24 hours after the injection, the patient will be monitored for side effects and will be discharged from the hospital if there are no side effects. Test results, MRI, EMG-NCV, spirometry, ALS-FRS, ASLAQ-40, and side effects before evaluation, 2 months and 4 months, 6 months, and one year after injection are evaluated.
Participants/Inclusion and exclusion criteria Inclusion criteria: Age: 18-65 Both gender Duration of disease onset: 6 months Both type of ALS: Limb onset &
Bulbar onset FVC ≥40٪ ALS-FRS≥ 23 Undertreatment with Rilozule Exclusion criteria: Concomitant neurological and psychiatric disease Concomitant systemic disease Treatment with corticosteroid, immunoglobulin, immunosuppressive during 12 months
Intervention groups
Fifteen patients with sporadic ALS who enter the study according to the inclusion and exclusion criteria and refer to the neurology clinic of the Royan Research Institute or the office of a fellow neurologist for treatment.
Main outcome variables
Side effects include a change in systolic and diastolic pressure relative to baseline; heart rate change, grade 2 oxygen saturation based on CTACAE version 4.0, temperature change; itching, hives, erythema, edema, headache, restlessness, nausea, vomiting, loss of consciousness; abnormal tissue formation, worsening of the disease course;
General information
Reason for update Acronym
IRCT registration information
IRCT registration number: IRCT20080728001031N26 Registration date: 2021-01-10, 1399/10/21
Registration timing: retrospective
Last update: 2021-01-10, 1399/10/21 Update count: 0
Registration date 2021-01-10, 1399/10/21 Registrant information
Name
Nasser Aghdami
Name of organization / entity Royan Institute
Country
Iran (Islamic Republic of) Phone
+98 21 2356 2000 Email address
nasser.aghdami@royaninstitute.org Recruitment status
Recruitment complete Funding source
Expected recruitment start date 2014-03-20, 1392/12/29
Expected recruitment end date 2015-08-25, 1394/06/03 Actual recruitment start date
2015-01-07, 1393/10/17 Actual recruitment end date
2016-05-05, 1395/02/16 Trial completion date
2017-09-22, 1396/06/31
2 Scientific title
Complications of intravenous transplantation of allogeneic tissue-derived mesenchymal stem cells in patients with ALS phase 1 clinical trial
Public title
Complications of intravenous transplantation of allogeneic tissue-derived mesenchymal stem cells in patients with ALS
Purpose Treatment
Inclusion/Exclusion criteria Inclusion criteria:
Age: 18-65 Both gender Duration of disease onset: 6 months Both type of ALS: Limb onset & Bulbar onset FVC
≥40٪ ALS-FRS≥ 23 Under treatment with Rilozule Exclusion criteria:
Neurological and psychiatric concomitant disease Concomitant systemic disease Treatment with
corticosteroid ,Ig,immunosuppressive during 12 months Familial ALS
Age
From 18 years old to 65 years old Gender
Both Phase
1
Groups that have been masked No information
Sample size
Target sample size: 20
Actual sample size reached: 17
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
1
Registry name clinicaltrials.gov Secondary trial Id
NCT02492516 Registration date
2015-07-08, 1394/04/17
Ethics committees
1
Ethics committee
Name of ethics committee
Royan Institute, Medical Ethics and Research Ethics Committee
Street address
Tehran, Resalat Highway, at the end of Bani Hashem North Street, Hafez Alley, PO Box 148- 16635 City
Tehran Province
Tehran Postal code
148- 16635 Approval date
2014-10-07, 1393/07/15
Ethics committee reference number EC/93/1084
Health conditions studied
1
Description of health condition studied Amyotrophic lateral sclerosis
ICD-10 code G12.21
ICD-10 code description Amyotrophic lateral sclerosis
Primary outcomes
1
Description
Side effects include systolic pressure greater than 180 mm Hg or less than 90 mm Hg, or more than 30 mm Hg change from baseline, diastolic pressure greater than 100 mm Hg or less than 50 mm Hg, or more than 30 mm Hg change from baseline, heart rate more than 110 or more than 33% increase, or heart rate less than 60 or less than 33% decrease, oxygen saturation (grade 2 according to CTACAE version 4.0) , Temperature more than 38 or more than 1 degree increase, any toxic side effects according to CTACAE version 4.0, itching, urticaria, erythema, edema, headache, restlessness, nausea, vomiting, decreased level of meningitis, encephalitis, formation Mass, the formation of abnormal tissue in the nervous system or other parts of the body, worsening of the disease
Timepoint
One day, two months, four months, six months and one year after injection
Method of measurement
Checklist of side effects, blood test, MRI, EMG-NCV and spirometry and The ALS Functional Rating Scale and Amyotrophic Lateral Sclerosis Assessment Questionnaire -ALSAQ
Secondary outcomes
1
Description
3 Disease progression level by measuring The ALS
Functional Rating Scale Timepoint
2 months, 4 months, 6 months and 12 months after injection of cell suspension
Method of measurement The ALS Functional Rating Scale
2
Description
Disease progression level by Amyotrophic Lateral Sclerosis Assessment Questionnaire
Timepoint
2 months, 4 months, 6 months and 12 months after injection of cell suspension
Method of measurement
Amyotrophic Lateral Sclerosis Assessment Questionnaire
3
Description
Disease progression level by measuring the forced vital capacity
Timepoint
2 months, 4 months, 6 months and 12 months after injection of cell suspension
Method of measurement Spirometry
Intervention groups
1
Description
Intervention group: Patients with sporadic amyotrophic lateral sclerosis receive an allogeneic adipose tissue mesenchymal stem cell suspension with a concentration of 2 million cells in 10 ml of normal saline, after
preparation in the adipose cell bank of Royan Research Institute. The cell suspension is injected once,
intravenously, in the ICU department of the hospital under the supervision of the corresponding specialist and anesthesiologist and by the responsible expert nurse.
After the injection, patients are monitored for 24 hours for side effects and are discharged from the hospital if no side effects occur. Patients are followed up two months, four months, 6 months, and one year after injection.
Category
Treatment - Other
Recruitment centers
1
Recruitment center
Name of recruitment center Royan Institute
Full name of responsible person Nasser Aghdami
Street address
Tehran, Resalat Highway, at the end of Bani Hashem
North Street, Hafez Alley, PO Box 148- 16635 City
Tehran Province
Tehran Postal code
148- 16635 Phone
+98 21 2251 8388 Email
nasseragh@gmail.com
Sponsors / Funding sources
1 Sponsor
Name of organization / entity Royan Institute
Full name of responsible person Abdolhossein Shahverdi
Street address
Royan Alley, Eastern Hafez St, Northern Banihashem St, 45 m Ghasem Soleymani Highway
City Tehran Province
Tehran Postal code
1665664511 Phone
+98 21 2356 2211 Email
shahverdi@royaninstitute.org Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source Royan Institute
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 Nasser Aghdami
Full name of responsible person Nasser Aghdami
Position
Associate Professor
4 Latest degree
Ph.D.
Other areas of specialty/work Immunology
Street address
Royan Alley, Eastern Hafez St, Northern Banihashem St, 45 m Ghasem Soleymani Highway
City Tehran Province
Tehran Postal code
Tehran, Resalat High Phone
+98 21 2251 8388 Email
nasseragh@gmail.com
Person responsible for scientific inquiries
Contact
Name of organization / entity
Iranian academic center for education culture and research
Full name of responsible person Nasser Aghdami
Position
Associate professor Latest degree
Ph.D.
Other areas of specialty/work Immunology
Street address
Tehran, Resalat Highway, at the end of Bani Hashem North Street, Hafez Alley, PO Box 148- 16635 City
Tehran Province
Tehran Postal code
1665664511 Phone
+98 21 2251 8388 Email
nasseragh@gmail.com
Person responsible for updating data
Contact
Name of organization / entity Royan Institute
Full name of responsible person Hoda Madani
Position Researcher Latest degree
Medical doctor
Other areas of specialty/work Cell Science and Application Street address
Royan Alley, Eastern Hafez St, Northern Banihashem St, 45 m Ghasem Soleymani Highway
City Tehran Province
Tehran Postal code
1665664511 Phone
+98 21 2251 8388 Email
hoda62_m@yahoo.com
Sharing plan
Deidentified Individual Participant Data Set (IPD) Undecided - It is not yet known if there will be a plan to make this available
Study Protocol
Undecided - It is not yet known if there will be a plan to make this available
Statistical Analysis Plan
Undecided - It is not yet known if there will be a plan to make this available
Informed Consent Form
Undecided - It is not yet known if there will be 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
