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Multiple SclerosisPhase 1/2MESEMSNCT02403947

MEsenchymal StEm Cells for Multiple Sclerosis

A Phase 1/2 study testing Mesenchymal stem cells and Suspension media in people with Multiple Sclerosis, run by University Hospital, Toulouse at 1 site in France.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

Multiple sclerosis (MS) is a chronic inflammatory disease of the central nervous system (CNS), which ultimately leads to myelin damage and axonal loss. The disease is complex and multifactorial, but the key pathogenic event appears to be an uncontrolled response of components of the immune system (T and B lymphocytes)… Read more about this study

Who is it for?
Ages 18 to 50 years
Conditions the study is for
Multiple Sclerosis
What is being tested?
Mesenchymal stem cells and Suspension media
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
1 participants
Where
1 siteFrance
Study dates
Feb 2015to Dec 2017
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 50 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • Age 18 to 50 years
  • Disease duration 2 to 10 years (included)
  • Diagnosis of MS
  • 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:
  • more or egal 1 clinically documented relapse in past 12 months
  • more or egal 2 clinically documented relapses in last 24 months
Show all 16Show fewer
  • more or egal 1 GEL at MRI performed within the last 12 months
  • 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::
  • With more or egal 1 clinically documented relapse in the last twelve months
  • Without on-going relapses, but with more or egal 1 GEL at MRI performed within the last 12 months.
  • Primary progressive MS (PPMS) patients with all the following features:
  • an increase ofmore or egal 1 EDSS point (if at inclusion EDSS inferior or egal 5.0) or 0.5 EDSS point (if at inclusion EDSS more or egal 5.5), in the last twelve months
  • more or egal 1 GEL at MRI performed within the last 12 months
  • Positive cerebrospinal fluid (CSF) (oligoclonal banding).
  • EDSS (Expanded Disability Status Scale) 3.0 to 6.5
  • Women of childbearing age with an effective contraception.

Who cannot join

  • RRMS not fulfilling inclusion criteria
  • SPMS not fulfilling inclusion criteria
  • PPMS not fulfilling inclusion criteria
  • Inferior to 3 months since treatment with any immunosuppressive therapy including natalizumab and fingolimod
  • Inferior or egal to 1 month since last treatment with interferon-beta or glatiramer acetate
  • Corticosteroid treatment Inferior or egal to 30 days
Show all 14Show fewer
  • Relapse inferior or egal to 60 days
  • Any active or chronic infection including infection with HIV1-2 (Human Immunodeficiency Virus 2) or HTLV I-II (Human T-lymphotropic virus I-II) or Syphilis or chronic Hepatitis B or Hepatitis C inferior to 1 month
  • 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 (estimated Glomerular Filtration Rate ) inferior to 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.

Trial locations and contacts

1 site in 1 country.

France1 site

  • Purpan Hospital

    Toulouse, 31059

Other trials in France

About this study

Official title: Treatment of Multiple Sclerosis With Mesenchymal Stem Cells: Phase I/II Study

Multiple sclerosis (MS) is a chronic inflammatory disease of the central nervous system (CNS), which ultimately leads to myelin damage and axonal loss. The disease is complex and multifactorial, but the key pathogenic event appears to be an uncontrolled response of components of the immune system (T and B lymphocytes) to myelin proteins. No definitive treatment is available for MS, however immunomodulatory and immunosuppressant drugs act as disease-modifying agents (DMDs).

Unfortunately, the current treatments demonstrate partial efficacy in targeting the deleterious immune reactions. According to the present knowledge of the pathophysiology of MS, an ideal therapeutic strategy would be to modulate or suppress the aggressive immune process, to protect axons and neurons from degeneration, and to enhance repair and facilitate remyelination.

A specific form of stem cells, called adult mesenchymal stem cells (MSCs), has shown remarkable ability to modulate the immune response. This study will evaluate the safety of injecting MSCs in people with MS.

Read the detailed description

MSCs have the remarkable ability to modulate the immune response mainly by inhibiting proliferation of T cells and to protect injured tissues through paracrine mechanisms. There is an urgent need to evaluate the real efficacy of MSC transplantation, and its possible position in the current therapeutic armamentarium. An international panel of MS neurology and stem cell experts, as well as immunologists formed an "International Mesenchymal Stem Cells Transplantation" (MSCT) Study Group with the aim to derive a consensus on what cells should be used for transplantation and develop a treatment protocol and an experimental program that will eventually attest to the efficacy of MSC transplantation and understand the mechanism that underlie the benefit. 12 patients with MS will be treated with IV injections of autologous isolated and expanded mesenchymal stem cells. Clinical and objective evaluations will be performed at baseline and during 12 months follow-up.

Being tested
Mesenchymal stem cells, Suspension media
Keywords
Multiple Sclerosis, Bone-marrow, Stem cells, Cell therapy
Medical subject headings
Multiple Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Mesenchymal stem cellsDrug

    After the sampling of bone-marrow and culture of MSCs by the French Blood Establishment (Midi-Pyrénées), patients will receive an IV injection of MSCs.

  • Suspension mediaDrug

    injection suspension media

Study groups in NCT02403947
Study groupRoleWhat they receive
Mesenchymal stem cellsExperimentalAt week 0 a single infusion of either ex-vivo expanded autologous MSC or suspension media will be administered intravenously at a dose of 1-2 x 1000000 MSC/Kg body weight. At week 24, another infusion will be performed for cross-over re-treatment: at week 24 treatments will be reversed compared to week 0
Receives: Drug: Mesenchymal stem cells
Suspension mediaPlacebo comparatorAt week 0 a single infusion of either ex-vivo expanded autologous MSC or suspension media will be administered intravenously at a dose of 1-2 x 1000000 MSC/Kg body weight. At week 24, another infusion will be performed for cross-over re-treatment: at week 24 treatments will be reversed compared to week 0
Receives: Drug: Suspension media

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety of MSCs infusion, number, timeframe of occurrence and severity of Adverse Events

    Safety assessed by number, timeframe of occurrence and severity of Adverse Events

    Measured: 24 weeks from the first infusion

  2. efficacy: number of contrast-enhancing lesions (GEL) at MRI scan

    total number of contrast-enhancing lesions (GEL) at MRI scan

    Measured: 24 weeks from the first infusion

Secondary outcomes (5)

  1. Efficacy of the experimental treatment in term of combined MRI activity

    Number of GEL counted over week 28, 36 and 48 compared with the number of GEL counted over 4, 12 and 24 weeks.

    Measured: 48 weeks from the first infusion

  2. Efficacy assessed by combined unique MRI activity, volume of GEL, and volume of BH

    Combined unique MRI activity (number of new or enlarging T2, or enhancing or re-enhancing lesions), volume of GEL and volume of black holes (BH) over 4, 12, 24 weeks compared between treatment groups.

    Measured: 24 weeks from the first infusion

  3. Efficacy assessed by combined unique MRI activity, volume of GEL and volume of BH

    Combined unique MRI activity, volume of GEL and volume of BH over week 28, 36 and 48 compared with the same outcomes over 4, 12 and 24 weeks.

    Measured: 48 weeks from the first infusion

  4. Efficacy: Number of relapses

    Number of relapses in MSC treatment group vs. placebo group in the first 24 weeks and after cross-over re-treatment in the two groups.

    Measured: 24 weeks from the first infusion

  5. Efficacy: Time to sustained progression of disability

    Time to sustained progression of disability compared between treatment groups during the first 24 weeks and after cross-over

    Measured: 24 weeks from the first infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Mar 2015 (estimated)
  2. Study startFeb 2015
  3. Primary completionDec 2017
  4. Study completionDec 2017

Why it stopped: inclusion default

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
1 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02403947
Sponsor's protocol ID
12 394 03
Acronym
MESEMS
Status last verified
Nov 2018
First submitted
9 Mar 2015
Last updated
3 Dec 2018
Results posted
No
Data monitoring committee
Yes

Publications

  • 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.

    PubMed 31072380

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Mesenchymal stem cells and Suspension media in people with Multiple Sclerosis, run by University Hospital, Toulouse at 1 site in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 16 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 3 Dec 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: inclusion default). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety of MSCs infusion, number, timeframe of occurrence and severity of Adverse Events (time frame: 24 weeks from the first infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02403947, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Dec 2018.

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