Evaluating the safety and efficacy of mesenchymal stem cell therapy in patients with cerebellar ataxia
Design
Clinical trial with a control group, with parallel groups, unblinded, randomized, phase 1 on 20 patients. The random number table of the software was used for randomization.
Settings and conduct
Ten patients with cerebellar ataxia receive 50-70 million autologous bone marrow-derived MSCs at Pasteur Hospital. In the control group, other common treatments are prescribed for patients.
Participants/Inclusion and exclusion criteria
inclusion criteria : people aged 10-65 years and with a definitive diagnosis of ataxia and no history of other mental illnesses.
Exclusion criteria : pregnancy or a history of other serious mental and non-mental illnesses.
Intervention groups
Intervention group: 10 patients with cerebellar ataxia receiving mesenchymal stem cells
Control group: 10 patients with cerebellar ataxia who will receive routine treatments.
Main outcome variables
*-neurologic sign of patients including: Scale for the Assessment and Rating of Ataxia (SARA)-Quality of life-Depression-Beck Scale-Improve in international Co-Operative Ataxia Rating Scale-Balance Test-Tremor Rating Scale
*-Before the intervention, month 3 and month 6 after the intervention
*-Clinical examination and questionnaire
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20160809029275N3
Registration date:2022-08-31, 1401/06/09
Registration timing:retrospective
Last update:2022-08-31, 1401/06/09
Update count:0
Registration date
2022-08-31, 1401/06/09
Registrant information
Name
Name of organization / entity
Country
Iran (Islamic Republic of)
Phone
+98 51376276015
Email address
tavakolaj@mums.ac.ir
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2021-07-23, 1400/05/01
Expected recruitment end date
2022-07-23, 1401/05/01
Actual recruitment start date
2021-09-04, 1400/06/13
Actual recruitment end date
2022-06-30, 1401/04/09
Trial completion date
2022-06-30, 1401/04/09
Scientific title
Evaluation of safety and motivation status of patients with cerebellar ataxia after intrathecal treatment with mesenchymal stem cells
Public title
cerebellar ataxia treatment with mesenchymal stem cells
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Does not receive stem cells in the last 6 months.
signing the written consent form by the participants.
The age range of 10-65 years without limitation in sex.
Definitive diagnosis of ataxia based on brain and cervical spinal cord MRI.
Presence of cerebellar or brainstem atrophy and/or cervical spinal cord.
Not having other mental illness such as schizophrenia etc.
Exclusion criteria:
Positive pregnancy test.
Heart, kidney and liver failure, epilepsy, lung disease, cardiac arrhythmia, diabetes, leukemia and other diseases of the central nervous system such as Parkinson's.
If the total level of bilirubin is greater than 1.5 times the upper limit of its normal value.
History of chronic or acute alcohol consumption
The presence of evidence of seizures in the brain scan
Any evidence of infection
History of severe drug sensitivity or anaphylaxis to two or more foods or drugs
Other organic brain diseases
HIV positive and tumor markers positive
Patients with severe psychosis, cognitive disorders and inability to understand or sign consent
Other organic or systemic diseases
Uncontrollable high blood pressure
Participation in other clinical trials in the last three months
The presence of thyroid disease
Presence of evidence of encephalitis
Presence of sarcoidosis and Behcet's disease
Vitamin E deficiency
Wilson's disease
Increased blood ammonia
Positive anti-GAD and anti-neuronal antibodies
Lupus and Wegener's disease
Middle ear dysfunction
Age
From 10 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:
20
Randomization (investigator's opinion)
Randomized
Randomization description
A simple randomization method using the random number table method was used to assign patients to groups. In this method, even numbers were placed in the intervention group and odd numbers were randomly placed in the control group.
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Parallel
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Ethics committee of Mashhad University of Medical Sciences
Street address
Mashhad,Daneshgah Street, Qurashi Building, Mashhad University of Medical Sciences
City
Mashhad
Province
Razavi Khorasan
Postal code
9137913316
Approval date
2022-07-02, 1401/04/11
Ethics committee reference number
IR.MUMS.REC.1401.128
Health conditions studied
1
Description of health condition studied
Diagnosis Code G32.81: Cerebellar ataxia
ICD-10 code
G32.81
ICD-10 code description
Cerebellar ataxia in diseases classified elsewhere
Primary outcomes
1
Description
neurologic sign of patients including: Scale for the Assessment and Rating of Ataxia (SARA)-Quality of life-Depression-Beck Scale-Improve in international Co-Operative Ataxia Rating Scale-Balance Test-Tremor Rating Scale
Timepoint
Before the intervention, month 3 and month 6 after the intervention
Method of measurement
Clinical examination and questionnaire
2
Description
Blood markers including: • T4 and TSH• Alpha-fetoprotein• IgG and IgE• OGTT and FBS• ACE• Vitamin E• Electrophoresis of serum proteins• Chol, TG, LDL and HDL• Ceruloplasmin, Cu and 24-hour urine Cu for patients less than 40 years old• Anti-GAD, Anti-dsDNA, P-ANCA and C-ANCA• Vitamin B12 and folic acid
Timepoint
Before the intervention, month 3 and month 6 after the intervention
Method of measurement
Laboratory methods such as electrophoresis, VIDAS and ELISA
3
Description
Investigating the safety of stem cell treatment in patients participating in the study
Timepoint
24-48 hours after receiving injections and the third and sixth months
Method of measurement
Clinical examination of patients to check for fever, headache or any possible allergic reactions.
Secondary outcomes
empty
Intervention groups
1
Description
Intervention group: Patients receiving 50-70 million autologous MSCs derived from bone marrow
Category
Treatment - Drugs
2
Description
Control group: Control group: patients with cerebellar ataxia who will receive routine treatments including vitamin E, CoQ10, physical therapy and occupational therapy.
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Pasteur hospital
Full name of responsible person
Dr. Amir Reza Boroumand
Street address
Ahmad Abad St. Pasteur Av. Pasteur Hospital
City
Mashhad
Province
Razavi Khorasan
Postal code
09194216791
Phone
+98 51 3841 0245
Email
info@pastorno-hospital.com
Web page address
http://pastorno-hospital.com/
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Dr Majid Ghayour Mobarhan
Street address
Azadi SQ. Mashhad
City
Mashhad
Province
Razavi Khorasan
Postal code
3316-913791
Phone
+98 51 3841 3006
Email
Ums@mums.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Mashhad University of Medical Sciences
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
Mashhad University of Medical Sciences
Full name of responsible person
Dr. Amir Reza Boroumand
Position
specialist
Latest degree
Subspecialist
Other areas of specialty/work
Neuroscience
Street address
Daneshgah st. Kafaei1 Av. Sadra building
City
Mashhad
Province
Razavi Khorasan
Postal code
91942167910
Phone
+98 51 3848 2194
Email
boroumandar@mums.ac.ir
Person responsible for scientific inquiries
Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Dr. Seyyed Jalil tavakol afshari
Position
prof.
Latest degree
Ph.D.
Other areas of specialty/work
Immunology
Street address
Bou Ali SQ, Bou Ali Research center
City
Mashhad
Province
Razavi Khorasan
Postal code
٩١٩۶٧٧٣١١٧
Phone
+98 51 3711 2471
Email
tavakolaj@mums.ac.ir
Person responsible for updating data
Contact
Name of organization / entity
Mashhad University of Medical Sciences
Full name of responsible person
Dr.Seyad Jalil Tavakol Afshari
Position
Professor
Latest degree
Ph.D.
Other areas of specialty/work
Immunology
Street address
Bu-Ali square. Bu-Ali Science and Technology Park, Mashhad
City
Mashhad
Province
Razavi Khorasan
Postal code
9196773117
Phone
+98 51 3711 2610
Fax
+98 51 3711 2596
Email
Tavakolaj@mums.ac.ir
Sharing plan
Deidentified Individual Participant Data Set (IPD)
No - There is not a plan to make this available
Justification/reason for indecision/not sharing IPD
there is no more information
Study Protocol
Yes - There is 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
Yes - There is a plan to make this available
Clinical Study Report
Yes - There is a plan to make this available
Analytic Code
No - There is not 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 project protocol will be reported after the end of the study
When the data will become available and for how long
Access time will be 6 moth after results publication
To whom data/document is available
All people without limitation
Under which criteria data/document could be used
any scientific usage
From where data/document is obtainable
Contact the project manager Dr. Tavakal Afshari with the following details.
Address: Ferdowsi Square, Bo Ali Square, Mashhad, Bo Ali Research Institute, Department of Immunogenetics and Cell Culture Phone: 0517112674
tavakolaj@mums.ac.ir
What processes are involved for a request to access data/document
Send a written request by email to the project manager, Dr. Tavakal Afshari, with the mentioned address and contact number