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Evaluation of safety and efficacy of Allogenic Mesenchymal Stem Cell transplantation (core matrix extracted)in Late-Onset Neurometabolic disorders: an open label study

Protocol summary

Study aim
Effective treatment finding for some untreated neurometabolic disorders
Design
Community based and pragmatic,non-randomized single group, clinical trial without blinded postoperative care and outcome assessment
Settings and conduct
Patients that selected for this clinical trial, will be admitted to Labafinejad or Farmanieh Hospital inTehran. Based on CNS involvement,one or much more injections will be planned by intrathecal and/or intravenous rout. Injectable mesenchymal stem cell will be prepared in clean room by Sinacell company, 3-4 million cell per kilogram more than 90% viability in 20-50 milliliter injectable human albumin as preservative in each intravenous injection and one million per kilogram in 5-10 milliliter in each intrathecal injection.Interval of these injections is between two days to two weeks. The hospitalization time will be about 6 hours for intravenous injection and 24 to 24 48 hours for intrathecal injection. Patients will undergo a clinical and laboratory evaluation for a period of 2 years at defined intervals.
Participants/Inclusion and exclusion criteria
Inclusion Criteria: Patients with genetic proven late onset neurometabolic disorders which have developed symptoms but have not yet reached end stage phase. Exclusion criteria: Patients who are susceptible to malignancy, patients with chronic infectious diseases such as AIDS, hepatitis, syphilis and HTLV, pregnancy, vulnerable ages and conditions.
Intervention groups
Wharton jelly derived allogenic mesenchymal stem cells in suitable albumin solution will inject to leukodystrophy, ataxic, myopathic, neuropathic patients by peripheral and/ or intrathecal routs.
Main outcome variables
Qualitative evaluation of ataxia by SARA Questionnaire, Qualitative evaluation of mental and physical functions by MSFC Questionnaire ,Chance of Diabetes Melitus occurrence ,Chance of heart Failure occurence.

General information

Reason for update
Acronym
IRCT registration information
IRCT registration number: IRCT20180228038899N1
Registration date: 2018-07-16, 1397/04/25
Registration timing: registered_while_recruiting

Last update: 2018-07-16, 1397/04/25
Update count: 0
Registration date
2018-07-16, 1397/04/25
Registrant information
Name
Bita Shalbafan
Name of organization / entity
Country
Iran (Islamic Republic of)
Phone
+98 21 2271 8565
Email address
shalbafan.b@gmail.com
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2017-02-21, 1395/12/03
Expected recruitment end date
2020-09-21, 1399/06/31
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Evaluation of safety and efficacy of Allogenic Mesenchymal Stem Cell transplantation (core matrix extracted)in Late-Onset Neurometabolic disorders: an open label study
Public title
Effect of Allogenic Stem Cell in Neurometabolic disorders
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
The definitive diagnosis of neurometabolic diseases leads to ataxia, leukodystrophy,Myopathy or Neuropathy based on laboratory findings of molecular genetics Age between 18-40 years old Written consent of the patient and parents to attend the study Normal routine biochemistry, hematology and negative serology and virology tests Negative Tumor survey includes abdominal and pelvic ultrasonography and prostatic and breast and thyroid ,and occult blood in the stool test
Exclusion criteria:
A pregnant woman (positive pregnancy test) or a nursing or illness who is planning a pregnancy during the study A disease that is in addition to the involvement of a nervous system with another serious illness, such as hemodynamic disorders, homeostasis disorders, diabetes, cardiovascular / pulmonary disease, etc. Having a serious psychiatric illness or having a history of suicide Treatment with cytotoxic drugs within a month before starting the study The presence of any suspected malignancy mass Serum creatinine more than 1.7 Rised liver enzyme tests more than three times White blood cell count lower than 3000 Positive response to each of the serum tests of HTLV1,2 Ab, HIV1,2Ab, HBcAb, HBsAg,HCVAb
Age
From 18 years old to 40 years old
Gender
Both
Phase
2
Groups that have been masked
No information
Sample size
Target sample size: 25
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 Shahid Beheshti University of Medical Sciences
Street address
Research Management office, 6th floor, building No.2, Shahid Beheshti Medical University, Arabi Ave., Daneshjoo Blvd., Velenjak Town
City
Tehran
Province
Tehran
Postal code
1983963113
Approval date
2017-02-21, 1395/12/03
Ethics committee reference number
Ir.sbmu.rec.1395.82

Health conditions studied

1

Description of health condition studied
Progressive Ataxia
ICD-10 code
G11
ICD-10 code description
Hereditary ataxia

2

Description of health condition studied
Myopathy
ICD-10 code
M62.5
ICD-10 code description
Muscle wasting and atrophy, not elsewhere classified, unspecified site

3

Description of health condition studied
Neuropathy
ICD-10 code
G60
ICD-10 code description
Hereditary and idiopathic neuropathy

4

Description of health condition studied
Leukodystrophy
ICD-10 code
E75.2
ICD-10 code description
Other sphingolipidosis

Primary outcomes

1

Description
Ataxia in Ataxic patients
Timepoint
At first, 1,3,6,12,18,24 months after intervention
Method of measurement
Scale of Assesment and Rating of Ataxia in Ataxic patients

2

Description
Functional Capacity in paretic patients with or without ataxia and mental problems
Timepoint
At first, 1,3,6,12,18,24 months after intervention
Method of measurement
Multiple Sclerosis Functional Capacity scoring

3

Description
Blood Sugar assessment in patients with Friedreich Ataxia diagnosis
Timepoint
At first, 1,3,6,12,18,24 months after intervention
Method of measurement
Laboratory measurements of Fasting Blood Sugar and Hemoglobin َA1C

4

Description
Heart failure assessment in patients with Friedreich Ataxia diagnosis
Timepoint
At first, 1,3,6,12,18,24 months after intervention
Method of measurement
Ejection Fraction by Echocardiography

Secondary outcomes

empty

Intervention groups

1

Description
Intervention group:َAfter patient selection ,one or much more injections will be planned by intrathecal and/or intravenous rout based on CNS involvement . Injectable mesenchymal stem cell will be prepared in clean room by Sinacell company, 3-4 million cell per kilogram more than 90% viability in 20-50 milliliter injectable human albumin as preservative in each intravenous injection and one million per kilogram in 5-10 milliliter in each intrathecal injection.Interval of these injections is between two days to two weeks. The hospitalization time will be about 6 hours for intravenous injection and 24 to 24 48 hours for intrathecal injection. Patients will undergo a clinical and laboratory evaluation for a period of 2 years at defined intervals.
Category
Treatment - Drugs

Recruitment centers

1

Recruitment center
Name of recruitment center
Labafinejad Hospital
Full name of responsible person
Bita Shalbafan
Street address
No. 133,9th Boustan Ave.,Pasdaran Ave.
City
Tehran
Province
Tehran
Postal code
1666659534
Phone
+98 21 2278 3140
Email
shalbafan.b@gmail.com

Sponsors / Funding sources

1

Sponsor
Name of organization / entity
Pasture Institute of Iran
Full name of responsible person
Dr. Mohsen Asouri
Street address
North Institute, Pastor Institute of Iran, Km 5 Babol old road,
City
Amol
Province
Mazandaran
Postal code
4619332976
Phone
+98 11 4319 8074
Fax
+98 11 4319 8651
Email
mohsen.asouri@yahoo.com
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Pasture Institute of Iran
Proportion provided by this source
100
Public or private sector
Private
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Other

Person responsible for general inquiries

Contact
Name of organization / entity
Iran Social Security Organization
Full name of responsible person
Bita Shalbafan
Position
consultant
Latest degree
Specialist
Other areas of specialty/work
Neurology
Street address
No.133,9th Boustan Ave.,Pasdaran Ave.
City
Tehran
Province
Tehran
Postal code
1666659534
Phone
+98 21 2278 3140
Email
shalbafan.b@gmail.com

Person responsible for scientific inquiries

Contact
Name of organization / entity
iran social security Organization
Full name of responsible person
Bita Shalbafan
Position
consultant
Latest degree
Specialist
Other areas of specialty/work
Neurology
Street address
No.133,9th Boustan Ave.,Pasdaran Ave.
City
Tehran
Province
Tehran
Postal code
1666659534
Phone
+98 21 2278 3140
Email
shalbafan.b@gmail.com

Person responsible for updating data

Contact
Name of organization / entity
Iran Social Security Organization
Full name of responsible person
Bita Shalbafan
Position
consultant
Latest degree
Specialist
Other areas of specialty/work
Neurology
Street address
No.133,9th Boustan Ave.,Pasdaran Ave.
City
Tehran
Province
Tehran
Postal code
1666659534
Phone
+98 21 2278 3140
Email
Shalbafan.b@gmail.com

Sharing plan

Deidentified Individual Participant Data Set (IPD)
Yes - There is 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
Yes - There is 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
Not applicable
Data Dictionary
Not applicable
Title and more details about the data/document
After keeping samples privacy , we will share their demographic data and all of findings.
When the data will become available and for how long
We will publish our findings as an article after 6 months.
To whom data/document is available
Somebody are interesting to research in the field of Stem cell.
Under which criteria data/document could be used
For researchers in the field of stem cells, statistical analyzes can be done.
From where data/document is obtainable
1-Dr. Bita Shalbafan +989161119656 shalbafan.b@gmail.com 2-Dr. Mandana Mohyeddin Bonab +989122024962 mohyeddin@sina.tums.ac.ir
What processes are involved for a request to access data/document
Six months after the final article is published, the applicants will submit their application by email or telephone and receive data within a maximum of one month by email.
Comments
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