Evaluation of safety and efficacy of repeated intrathecal and/or intravenous transplantations of Wharton’s jelly derived mesenchymal stromal cells in patients with ALS, clinical trial, phase I
Evaluation of safety and efficacy of repeated intrathecal and/or intravenous transplantations of Wharton’s jelly derived mesenchymal stromal cells in patients with ALS
Design
This study, which is a phase 1 clinical trial, includes 12 patients with ALS.
Settings and conduct
The patient was admitted to the VIP room of Mostafa Khomeini Hospital and the cell suspension prepared from Whartocell allogeneic cell bank produced in the center with good manufacturing products (GMP) grade was injected intrathecally by the neurologist in charge of the project in 6 patients and intravenously by the expert nurse in charge in 6 other patients.
Participants/Inclusion and exclusion criteria
Age: 18-70
Both gender
Disease onset at least: 6 months
All patients diagnosed with ALS and R/O of differential diagnosis
Forced Vital Capacity (FVC) ≥40٪
ALS Functional Rating Scale-Revised (ALSFRS-R)≥ 25
Under treatment with rilozule and edaravone
Ability to walk even with aid and ability to tolerate the procedures
Intervention groups
1- Intrathecal transplantations of Wharton’s jelly derived mesenchymal stromal cells
2- Intravenous transplantations of Wharton’s jelly derived mesenchymal stromal cells
Main outcome variables
1- Evaluation of possible side effects after repeated intrathecal transplantation or Intravenous allogeneic cord-derived mesenchymal cells of allogeneic cord in patients with ALS
2- Evaluation of changes in ALSFRS-R score
3- Evaluation of FVC changes
4- Evaluation of year survival
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20080728001031N32
Registration date:2022-05-07, 1401/02/17
Registration timing:prospective
Last update:2022-05-07, 1401/02/17
Update count:0
Registration date
2022-05-07, 1401/02/17
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
2022-05-20, 1401/02/30
Expected recruitment end date
2022-07-06, 1401/04/15
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Evaluation of safety and efficacy of repeated intrathecal and/or intravenous transplantations of Wharton’s jelly derived mesenchymal stromal cells in patients with ALS, clinical trial, phase I
Public title
Evaluation of safety and effectiveness of recurrent umbilical cord-derived stem cell transplantation in patients with ALS
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Age: 18-70
Both gender
Disease onset at least: 6 months
All patients diagnosed with ALS and R/O of differential diagnosis
FVC ≥40٪
ALSFRS-R≥ 25
Under treatment with rilozule and edaravone
Ability to walk even with aid and ability to tolerate the procedures
Exclusion criteria:
Concomitant psychiatric or cognitive disease
Concomitant systemic disease (uncontrolled HTN, uncontrolled DM, uncontrolled kidney diseases, HF, …)
Treatment with corticosteroid , IVIg, immunosuppressive and cytotoxic during 12 months
Dependency to ventilation
History of malignancy
Concomitant other neurodegenerative disorders
Evaluation of possible side effects at intervals of 24 hours, 1 month, 3 months, 6 and 12 months after injection
Method of measurement
side effects questionnaire
Secondary outcomes
1
Description
Changes in ALSFRS-R score
Timepoint
1, 3, 6 and 12 months after transplantation
Method of measurement
ALSFRS-R questionnaire
2
Description
Changes in FVC percent (spirometery)
Timepoint
6 and 12 months after transplantation
Method of measurement
Spirometry
3
Description
One-year survival
Timepoint
one year
Method of measurement
visiting the patients
Intervention groups
1
Description
Intervention groups: Intravenous or intrathecal injection of 60 millions of Wharton’s jelly derived mesenchymal stromal cells in two doses/ 3 months- WJMSCs are suspended in 10 ml N/S for intravenous and 5 ml for intrathecal transplantation. Celltech pharmed Co. provided the WJMSCs for this study.