Evaluate the safety of simultaneous intrathecal and intravenous administration of umbilical cord-derived mesenchymal stem cells (MSCs) in patients with MS.
Design
This study is designed as a phase I, randomized clinical trial aimed primarily at evaluating the safety of simultaneous intrathecal and intravenous administration of umbilical cord-derived mesenchymal stem cells in patients with MS. The study sample size will consist of 5 patients selected according to the predefined inclusion and non-inclusion criteria. Randomization will be performed using a simple randomization method through the RAND function in Microsoft Excel. All participants will receive the same intervention and will be followed for 12 months regarding clinical, laboratory, and imaging safety outcomes.
Settings and conduct
Patients with MS were referred to the Baqiatallah hospital
Participants/Inclusion and exclusion criteria
Patients with a definite diagnosis of Multiple Sclerosis (MS) according to the 2017 McDonald criteria, aged 18–55 years, with mild to moderate disability (EDSS score 1–6), no acute relapse within the previous 3 months, and written informed consent will be included in the study.
Non-inclusion Criteria:
Patients with pregnancy or breastfeeding, concomitant autoimmune or chronic diseases, use of immunosuppressive drugs within the previous 3 months, or unwillingness to continue participation will not be included in the study.
Intervention groups
Just one group
Main outcome variables
Adverse events and treatment safety; changes in Expanded Disability Status Scale (EDSS); timed 25-foot walk test; renal function (BUN, creatinine); liver function (AST, ALT, ALKP, bilirubin); hematological indices (WBC, RBC, platelet); inflammatory markers; number of brain plaques detected on MRI; serum neurofilament light chain (NFL) levels; inflammatory cytokines; vital signs; EKG findings; urinalysis results.
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20251207068238N1
Registration date:2026-05-29, 1405/03/08
Registration timing:prospective
Last update:2026-05-29, 1405/03/08
Update count:0
Registration date
2026-05-29, 1405/03/08
Registrant information
Name
Ramtin Naderian
Name of organization / entity
Country
Iran (Islamic Republic of)
Phone
+98 919 431 3400
Email address
ramtinndn@gmail.com
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2026-06-05, 1405/03/15
Expected recruitment end date
2026-09-22, 1405/06/31
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Safety assessment of intrathecal and intravenous mesenchymal umbilical cord-derived stem cells in multiple sclerosis patients
Public title
Safety assessment of intrathecal and intravenous mesenchymal umbilical cord-derived stem cells in multiple sclerosis patients
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Definite diagnosis of Multiple Sclerosis according to the 2017 McDonald criteria.
Age between 18 and 55 years old.
Mild to moderate disability (Expanded Disability Status Scale [EDSS] score between 1 and 6).
No acute relapse within the previous 3 months.
Provision of written informed consent for participation in the study.
Exclusion criteria:
Age
From 18 years old to 55 years old
Gender
Both
Phase
1
Groups that have been masked
No information
Sample size
Target sample size:
5
Randomization (investigator's opinion)
Randomized
Randomization description
Given the phase I design of the present clinical trial and its primary focus on safety assessment, a very small sample size will be employed. Consistent with several previous phase I studies that included sample sizes ranging from 5 to 8 participants, the sample size for the current study was determined to be five participants. Eligible subjects will be selected from the study population according to the predefined inclusion and exclusion criteria using a randomized double-blind design.
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Crossover
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Ethics committee of Baqiatallah University of Medical Sciences
Street address
تهران ، میدان ونک ، خیابان ملاصدرا , خیابان شیخ بهایی جنوبی
City
Tehran
Province
Tehran
Postal code
۱۴۳۵۹۱۵۳۸۱
Approval date
2026-05-18, 1405/02/28
Ethics committee reference number
IR.BMSU.REC.1405.028
Health conditions studied
1
Description of health condition studied
Multiple Sclerosis
ICD-10 code
ICD-10 code description
Primary outcomes
1
Description
Incidence and severity of adverse events based on CTCAE criteriaChange in EDSS score from baselineChange in Timed 25-Foot Walk Test performanceChange in the number of brain MRI plaquesChange in serum NFL levelsChange in inflammatory cytokine levels
Timepoint
since the first day to 6 months after that
Method of measurement
The following instruments will be used for data collection in this study:Standardized clinical checklist including:* Vital signs (blood pressure, pulse rate, body temperature, respiratory rate, and SpO₂)* Acute post-injection adverse events (headache, nausea, low back pain, seizure, infection)* EDSS assessment performed by an experienced neurologist specialized in MSStandard paraclinical assessments:* Liver function tests (AST, ALT, ALKP, Bilirubin)* Renal function tests (BUN, Creatinine)* Complete blood count (CBC)* ESR and CRP* Fasting blood glucose* Urinalysis (U/A)* Electrocardiography (EKG)Brain MRI with and without gadolinium contrast enhancement:* Interpreted by an expert neuroradiologistSafety monitoring:* Safety Monitoring Form checklist* Monitoring of immediate adverse events (0–24 hours)* Monitoring of delayed adverse events (1 week, 1 month, and 3 months)Validity of Instruments:Not applicable in this study.Reliability and Validity of Instruments:Content Validity:All instruments and assessments (CBC, LFT, RFT, MRI, EDSS, and EKG) are internationally standardized and approved (FDA-approved / CE-marked) and are routinely used in validated clinical safety protocols; therefore, no additional validation is required.Construct Validity:Measures such as EDSS, MRI findings, and inflammatory biomarkers are among the most established tools for evaluating disease status and treatment safety in MS patients.Reliability:* Inter-rater reliability: EDSS assessments will be performed by the same experienced neurologist to ensure consistency.* Test–retest reliability: Laboratory assessments (CBC, LFT, RFT) and MRI evaluations have established technical reliability and will be performed by the same technicians and radiologist throughout the study.Sample Size and Sample Size Calculation:Given the phase I design of the study and its primary focus on safety evaluation, a small sample size will be used. Consistent with previous similar studies that included 5–8 participants, the sample size of the present study was determined to be 5 patients, selected according to the predefined inclusion and non-inclusion criteria.Statistical Analysis:Data will be reported descriptively using mean, standard deviation, and percentages. Quantitative variables before and after intervention will be compared using paired t-test or Wilcoxon signed-rank test if the data are not normally distributed.Qualitative variables will be analyzed using McNemar or Chi-square tests. In the presence of multiple follow-up time points, Repeated Measures ANOVA or Linear Mixed Models will be applied.For biomarkers (NFL, IL-1β, IL-6, IL-10), logarithmic transformation or non-parametric tests will be used if the distribution is skewed. Correlations between biomarker changes and MRI/questionnaire findings will be assessed using Spearman correlation analysis.A p-value <0.05 will be considered statistically significant in all analyses.
Secondary outcomes
empty
Intervention groups
1
Description
Intervention group: Intervention Group:Patients with Multiple Sclerosis (MS) will receive simultaneous administration of human umbilical cord-derived mesenchymal stem cells (UC-MSCs). The cells will be isolated, expanded, and quality-controlled under Good Manufacturing Practice (GMP) conditions, including sterility, viability, and contamination testing.The intervention consists of:1. Intravenous (IV) administration of UC-MSCs2. Intrathecal (IT) administration of UC-MSCsAll injections will be performed at Baghiatallah Hospital by an experienced neurologist. Following administration, patients will be monitored for immediate and delayed adverse events through clinical assessments, laboratory evaluations, and brain MRI imaging, and will be followed for 12 months.
Category
Treatment - Other
Recruitment centers
1
Recruitment center
Name of recruitment center
Baqiatallah Hospital
Full name of responsible person
Ramtin naderian
Street address
Baqiatallah Hospital, Tehran, Iran
City
Tehran
Province
Tehran
Postal code
۱۴۳۵۹۱۵۳۸۱
Phone
+98 996 323 4034
Email
ramtinndn@gmail.com
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Bagheiat-allah University of Medical Sciences
Full name of responsible person
Seyed Javad Hosseininejad
Street address
Baqiyatallah Hospital
City
Tehran
Province
Tehran
Postal code
۱۴۳۵۹۱۵۳۸۱
Phone
+98 915 318 6055
Email
ramtinndn@gmail.com
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?