Mesenchymal Stem Cells for Multiple Sclerosis

NCT01730547 | Completed Phase 1 DMC

• 1 other identifier: MSC-MS
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of the study is to evaluate the safety and efficacy of autologous mesenchymal stromal cells as treatment for Multiple Sclerosis.

Conditions

Multiple Sclerosis

Keywords

Mesenchymal stem cells; Multiple Sclerosis; Autoimmune diseases

Outcome Measures

Primary Outcomes (1)

• To assess the safety of IV therapy with autologous Mesenchymal Stem Cells (MSCs) in MS patients.

  The primary objective of the study is to assess the safety of IV therapy with autologous MSCs in MS. Number of participants with adverse events will be documented at week 0,4,8,12,16,20,24,28,32,36,40,44,48 post treatment. Co-primary objective of the study is to evaluate the activity of autologous MSCS in MS patients, in terms of reduction as compared to placebo in the total number of contrast-enhancing lesions (GEL) at MRI acquired on conventional 1,5 T MRI scans over 24 weeks.

  48 weeks

Secondary Outcomes (1)

• To gather preliminary information of the efficacy of the experimental treatment in terms of combined MRI activity and clinical efficacy (incidence of relapses and disability progression).

  48 weeks

Study Arms (2)

Early treatment with mesenchymal stem cells

ACTIVE COMPARATOR

Patients receive a single intravenous dose of autologous bone marrow-derived MSCs followed by placebo at week 24. Fommow up at week 48

Biological: Autologous mesenchymal stem cells

Delayed treatment with mesenchymal stem cells

ACTIVE COMPARATOR

Patients receive placebo for 24 weeks followed by autologous MSCs at week 24, with a follow-up visit at week 48.

Biological: Autologous mesenchymal stem cells

Interventions

Autologous mesenchymal stem cells BIOLOGICAL

Delayed treatment with mesenchymal stem cells; Early treatment with mesenchymal stem cells

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Diagnosis of MS
• a. Relapsing remitting MS (RRMS) not responding to at least a year of attempted therapy with one or more of the approved therapies (beta-interferon, glatiramer acetate, natalizumab, mitoxantrone, fingolimod) as evidenced by one or more of the following: i. ≥1 clinically documented relapse in past 12 months ii. ≥2 clinically documented relapses in last 24 months iii. ≥1 GEL at MRI performed within the last 12 months
• b. Secondary progressive MS (SPMS) not responding to at least a year of attempted therapy with one or more of the approved therapies (beta-interferon, glatiramer acetate, natalizumab, mitoxantrone, fingolimod) as evidenced by both: i. an increase of ≥1 EDSS point (if at randomization EDSS ≤ 5.0) or 0.5 EDSS point (if at randomization EDSS ≥ 5.5) in the last 12 months ii. ≥1 clinically documented relapse or ≥ 1 GEL at MRI within the last twelve months.
• c. Primary progressive MS (PPMS) patients with all the following features: i. an increase of ≥1 EDSS point (if at randomization EDSS ≤ 5.0) or 0.5 EDSS point (if at randomization EDSS ≥5.5), in the last twelve months ii. ≥ 1 GEL at MRI performed within the last 12 months iii. positive cerebrospinal fluid (CSF) (oligoclonal banding)
• Age 18 to 50 years
• Disease duration 2 to 10 years (included)
• EDSS 3.0 to 6.5

You may not qualify if:

• Any active or chronic infection including infection with HIV1-2 or chronic Hepatitis B or Hepatitis C
• Treatment with any immunosuppressive therapy, including natalizumab and fingolimod, within the 3 months prior to randomization
• Treatment with interferon-beta or glatiramer acetate within the 30 days prior to randomization
• Treatment with corticosteroids within the 30 days prior to randomization
• Relapse occurred during the 60 days prior to randomization
• 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
• Severely limited life expectancy by another co-morbid illness
• History of previous diagnosis of myelodysplasia or previous hematologic disease or current clinically relevant abnormalities of white blood cell counts
• Pregnancy or risk or pregnancy (this includes patients that are unwilling to practice active contraception during the duration of the study)
• eGFR \< 60 mL/min/1.73m2 or known renal failure or inability to undergo MRI examination.
• Inability to give written informed consent in accordance with research ethics board guidelines

Sponsors & Collaborators

• Karolinska Institutet: lead

Study Sites (1)

Karolinska Institute, Karolinska University Hospital Solna

Stockholm, 171 76, Sweden

Location

Related Publications (1)

• Uccelli A, Laroni A, Brundin L, Clanet M, Fernandez O, Nabavi SM, Muraro PA, Oliveri RS, Radue EW, Sellner J, Soelberg Sorensen P, Sormani MP, Wuerfel JT, Battaglia MA, Freedman MS; MESEMS study group. MEsenchymal StEm cells for Multiple Sclerosis (MESEMS): a randomized, double blind, cross-over phase I/II clinical trial with autologous mesenchymal stem cells for the therapy of multiple sclerosis. Trials. 2019 May 9;20(1):263. doi: 10.1186/s13063-019-3346-z.

  PMID: 31072380 DERIVED

MeSH Terms

Conditions

Multiple Sclerosis; Autoimmune Diseases

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD

Study Record Dates

• First Submitted: November 9, 2012
• First Posted: November 21, 2012
• Study Start: February 1, 2013
• Primary Completion: May 9, 2019
• Study Completion: November 20, 2021
• Last Updated: June 1, 2023

Record last verified: 2023-05

Locations

SE (1)