Mesenchymal Stem Cells for Progressive Multiple Sclerosis_Sweden

To assess the safety of a single dose of IV infusion of bone-marrow derived autologous Mesenchymal Stem Cells (MSCs) in Multiple Sclerosis (MS) with progressive disease status.

Study Overview

• Status: Completed
• Conditions: Multiple Sclerosis; Autologous Mesenchymal Stem Cells
• Intervention / Treatment: Biological: Autologous mesenchymal stem cells

• Study Type: Interventional
• Enrollment (Actual): 7
• Phase: Phase 1

Contacts and Locations

Study Locations

• Sweden
  • Stockholm, Sweden, 171 76
    • Karolinska Institute, Karolinska University Hospital Solna
  • Stockholm, Sweden, 17176
    • Karolinska Institute, Karolinska University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Diagnosis of MS

   1. Active relapsing remitting MS (RRMS) as evidenced by presence of ≥ 1 clinically documented relapse in the past 12 months or ≥2 clinically documented relapses in the last 24 months.
   2. Secondary progressive MS with clinical progression as evidenced by an increase of 1 EDSS point (or 0,5 p if EDSS ≥ 5 at time for study start) in the last year or evidenced by ≥1 relapse or ≥ GEL at MRI performed within the last year.
   3. Primary progressive MS with clinical progression as evidenced by an increase of 1 EDSS point (or 0,5 p if EDSS ≥ 5 at time for study start) in the last year.
2. Age_ 18-65 years
3. Disease duration: 2-20 years
4. EDSS 3,0-7,0

Exclusion Criteria:

1. Subtype of MS not fulfilling inclusion criteria
2. Treatment with any immunosuppressive therapy, including natalizumab and fingolimod, within the 3 months prior to randomization
3. Treatment with interferon-beta or glatiramer acetate within the 30 days prior to randomization
4. Treatment with corticosteroids within the 30 days prior to randomization
5. Relapse occurred during the 60 days prior to randomization
6. Previous history of a malignancy other than basal cell carcinoma of the skin or carcinoma in situ that has been in remission for more than one year
7. Severely limited life expectancy by another co-morbid illness
8. Active or chronic severe infection.
9. History of previous diagnosis of myelodysplasia or previous hematologic disease or current clinically relevant abnormalities of white blood cell counts
10. Pregnancy or risk or pregnancy (this includes patients that are unwilling to practice active contraception during the duration of the study)
11. eGFR < 60 mL/min/1.73m2 or known renal failure or inability to undergo MRI examination.
12. Inability to give written informed consent in accordance with research ethics board guidelines

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Open label single arm study; All patients will be treated with 1 single dose of IV infusion of autologous bone-marrow derived mesenchymal stem cells (1-2 million cells/kg body weight) and their therapeutic response will be followed over 48 weeks.	Biological: Autologous mesenchymal stem cells; IV therapy with autologous bone-marrow derived mesenchymal stem cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate number of participants with an adverse event related to the treatment.	Adverse events is defined as any untoward or undesirable medical occurence in the form of signs, symptoms, abnormal findings or diseases that emerge during the study period, regardless of causal relationship to the study drug.	48 weeks
To evaluate effects on MS disease activity measured by cumulative number of MRI T2 lesions.	Brain MRI examination	48 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate effects on MS disease activity measured by change in EDSS (expanded disability status scale).	EDSS assessed by neurologist.	48 weeks
To evaluate effect on peripheral blood immune cell populations.	Peripheral blood samples.	24 weeks

Collaborators and Investigators

• Sponsor: Karolinska Institutet

Study record dates

Study Major Dates

• Study Start (Actual): December 1, 2012
• Primary Completion (Actual): December 1, 2016
• Study Completion (Actual): December 31, 2016

Study Registration Dates

• First Submitted: January 14, 2015
• First Submitted That Met QC Criteria: December 14, 2018
• First Posted (Actual): December 19, 2018

Study Record Updates

• Last Update Posted (Actual): June 1, 2023
• Last Update Submitted That Met QC Criteria: May 30, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: Multiple Sclerosis; Mesenchymal Stem Cells
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Disease Attributes; Chronic Disease; Multiple Sclerosis; Multiple Sclerosis, Chronic Progressive; Sclerosis
• Other Study ID Numbers: MSC-progressive MS

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No