Safety and Efficacy of Intravenous Autologous Mesenchymal Stem Cells for MS: a Phase 2 Proof of Concept Study

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02239393
• Date of registration: 10/09/2014
• Prospective Registration: Yes
• Primary sponsor: Ottawa Hospital Research Institute
• Public title: Safety and Efficacy of Intravenous Autologous Mesenchymal Stem Cells for MS: a Phase 2 Proof of Concept Study MESCAMS
• Scientific title: MEsenchymal Stem Cell Therapy for CAnadian MS Patients
• Date of first enrolment: June 2015
• Target sample size: 31
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02239393
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Canada

Contacts

• Name: Mark S. Freedman, MSc MD FRCPC
• Affiliation: Ottawa Hospital Research Institute

Key inclusion & exclusion criteria

Inclusion Criteria:

- 1) Males and females with a diagnosis of MS

1. Relapsing remitting MS (RRMS) not responding to at least 1 year of attempted
therapy with one or more of the approved therapies (beta-interferon, glatiramer
acetate, natalizumab, mitoxantrone, fingolimod, dimethyl fumarate, teriflunomide,
alemtuzumab) as evidenced by at least one of the following:

- i) =1 clinically documented relapse in past 12 months

- ii) =2 clinically documented relapses in past 24 months

- iii) =1 gadolinium-enhancing lesion (GEL) at MRI performed within the past
12 months

2. 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, dimethylfumarate, teriflunomide,
alemtuzumab) 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 past 12 months

- ii) =1 clinically documented relapse or = 1 gadolinium-enhancing lesion
(GEL) at MRI within the past 12 months

3. 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 past 12 months

- ii) = 1 gadolinium-enhancing lesion (GEL) at MRI performed within the past
12 months

- iii) positive cerebrospinal fluid (CSF) (oligoclonal banding)

- 2) Age 18 to 50 years old, inclusive at time of informed consent

- 3) Disease duration 2 to 15 years (inclusive)

- 4) EDSS 2.5 to 6.5

- 5) Able and willing to sign informed consent prior to any study-related activities

Exclusion Criteria:

- 1) RRMS not fulfilling inclusion criteria

- 2) SPMS not fulfilling inclusion criteria

- 3) PPMS not fulfilling inclusion criteria

- 4) A history of active or chronic infection including infection with HIV1-2, chronic
Hepatitis B or Hepatitis C

- 5) Treatment with any immunosuppressive therapy, including natalizumab and fingolimod,
within the 3 months prior to randomization

- 6) Previous treatment with cladribine or alemtuzumab

- 7) Treatment with interferon-beta, glatiramer acetate, teriflunomide or dimethyl
fumarate within the 30 days prior to randomization (all teriflunomide patients will be
required to have followed a wash-out with either cholestyramine or activated charcoal
as indicated in the product monograph)

- 8) Treatment with corticosteroids within the 30 days prior to randomization

- 9) Relapse occurred during the 60 days prior to randomization

- 10) Previous history of a malignancy (patient reported) other than basal cell
carcinoma of the skin or carcinoma in situ that has been in remission for more than
one year

- 11) Severely limited life expectancy by any other co-morbid illness

- 12) History of previous diagnosis of myelodysplasia or previous hematologic disease
(patient reported) or current clinically relevant abnormalities of white blood cell
counts

- 13) Pregnancy or risk of pregnancy (this includes participants that are not willing to
practice active contraception for the duration of the study)

- 14) eGFR < 60 mL/min/1.73m2 or known renal failure or inability to undergo MRI
examination

- 15) Known allergy to gentamicin or related aminoglycosides

- 16) Inability to give written informed consent in accordance with research ethics
board guidelines

- 17) Concomitant participation in another clinical trial

- 18) Inability to adhere to protocol according to the investigator's medical judgement



Age minimum: 18 Years
Age maximum: 50 Years
Gender: All

Health Condition(s) or Problem(s) studied

Multiple Sclerosis

Intervention(s)

Biological: Mesenchymal Stem Cells

Primary Outcome(s)

Efficacy [Time Frame: 24 weeks from first infusion]

Safety [Time Frame: 24 weeks from first infusion]

Secondary Outcome(s)

Efficacy [Time Frame: 48 weeks from first infusion]

Efficacy [Time Frame: 24 weeks from first infusion]

Secondary ID(s)

20140368

Source(s) of Monetary Support

Please refer to primary and secondary sponsors