Mesenchymal Stem Cells for Progressive Multiple Sclerosis_Sweden

• ClinicalTrials.gov Identifier: NCT03778333
• Recruitment Status: Completed
• First Posted: December 19, 2018
• Last Update Posted: December 19, 2018
• Sponsor: Karolinska Institutet
• Information provided by (Responsible Party): Ellen Iacobaeus, Karolinska Institutet

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Autologous Mesenchymal Stem Cells; Multiple Sclerosis	Biological: Autologous mesenchymal stem cells	Phase 1

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 7 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Intervention Model Description: Open-label phase 1, single-center, pre-post comparison study
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Mesenchymal Stem Cells for Progressive Multiple Sclerosis_Sweden
• Actual Study Start Date: December 1, 2012
• Actual Primary Completion Date: December 2016
• Actual Study Completion Date: December 31, 2016

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. To evaluate number of participants with an adverse event related to the treatment. [ Time Frame: 48 weeks ]
   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.
2. To evaluate effects on MS disease activity measured by cumulative number of MRI T2 lesions. [ Time Frame: 48 weeks ]
   Brain MRI examination

Secondary Outcome Measures :

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

Eligibility Criteria

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

Criteria

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

Contacts and Locations

Locations

Sweden

Karolinska Institute, Karolinska University Hospital Solna

Stockholm, Sweden, 171 76

Karolinska Institute, Karolinska University Hospital

Stockholm, Sweden, 17176

Sponsors and Collaborators

Karolinska Institutet

More Information

• Responsible Party: Ellen Iacobaeus, Principal investigator, Karolinska Institutet
• ClinicalTrials.gov Identifier: NCT03778333
• Other Study ID Numbers: MSC-progressive MS
• First Posted: December 19, 2018
• Last Update Posted: December 19, 2018
• Last Verified: December 2018

• 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

Keywords provided by Ellen Iacobaeus, Karolinska Institutet:

Mesenchymal Stem Cells; Multiple Sclerosis

Additional relevant MeSH terms:

Multiple Sclerosis; Multiple Sclerosis, Chronic Progressive; Sclerosis; Pathologic Processes; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases