Intrathecal Administration of Autologous Mesenchymal Stem Cell-derived Neural Progenitors (MSC-NP) in Progressive Multiple Sclerosis
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|ClinicalTrials.gov Identifier: NCT03355365|
Recruitment Status : Recruiting
First Posted : November 28, 2017
Last Update Posted : May 6, 2020
|Condition or disease||Intervention/treatment||Phase|
|Multiple Sclerosis||Biological: Intrathecal MSC-NP injection Other: Intrathecal saline injection||Phase 2|
The IT-MSC-NP treatments and all clinical assessments will take place at a single center (Tisch MSRCNY). Study subjects will be assigned to blocks stratified by baseline EDSS score (3.0-4.0, 4.5-5.5, 6.0, and 6.5) and disease subtype (SPMS or PPMS). Study subjects are randomized in an equal fashion (1:1) to study treatment and placebo at initial randomization. Subjects in each block will be randomized into placebo or treatment group. In the second year, treated subjects will cross over to the placebo group and placebo subjects will cross over to the treated group.
The total study duration will be 3 years upon enrollment. Each study subject will be required to attend up to 18 study visits, to include 1 screening visit, 1 bone marrow visit, 1 baseline visit, followed by study visits every 2 months during the treatment period of two years (12 treatment/LP procedure visits and 2 outcome visits), and an additional follow-up visit at the end of year 3.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Autologous, Bone Marrow-Derived Mesenchymal Stem Cell-Derived Neural Progenitor Cells (MSC-NP), Expanded Ex Vivo; Administered Intrathecally|
|Actual Study Start Date :||September 21, 2018|
|Estimated Primary Completion Date :||November 2020|
|Estimated Study Completion Date :||November 2023|
Experimental: Intrathecal MSC-NP injection
Patients will receive six autologous stem cell injections through spinal taps every 2 months over a year.
Biological: Intrathecal MSC-NP injection
MSC-NPs represent a neural subpopulation of MSCs from bone marrow with reduced pluripotency and minimized risk of ectopic differentiation, thus are likely to be more suitable for CNS delivery. Importantly, characterization of MSC-NPs demonstrated their immunoregulatory and trophic properties, and MSC-NPs derived from MS and non-MS patients alike were therapeutically viable.
Placebo Comparator: Intrathecal saline injection
Patients will receive six placebo injections through spinal taps every 2 months over a year.
Other: Intrathecal saline injection
- Expanded Disability Status Scale (EDSS) Plus [ Time Frame: Month 36 from first treatment or placebo ]Changes in disability assessed based on composite score of EDSS, timed 25-foot walk (T25FW), and nine hole peg test (9HPT) (EDSS-Plus). Improvement will be defined by at least one of the following three measures: ≥0.5 improvement in EDSS (if EDSS at entry is ≥ 6.0) or ≥ 1.0 improvement in EDSS (if EDSS at entry is ≤5.5), ≥20% improvement in T25FW, and ≥20% improvement in 9HPT in either dominant or non-dominant upper limb. Assessments will be made at baseline, Month 6, 13, 20, 27 & 36 in each group.
- Multiple sclerosis functional composite (MSFC) [ Time Frame: Month 36 from first treatment or placebo ]Changes in disability assessed by MSFC at baseline, Month 6, 13, 20, 27 & 36 in each group.
- Bladder function [ Time Frame: Month 27 from first treatment or placebo ]Degree of bladder dysfunction assessed by urodynamics testing at baseline, Month 13 and 27 in each group.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03355365
|Contact: Saud A Sadiq, MD, FAAN||212 265 firstname.lastname@example.org|
|Contact: Setse Bush, BA||212 265 email@example.com|
|United States, New York|
|Tisch MS Research Center of New York||Recruiting|
|New York, New York, United States, 10019|
|Contact: Setse Bush, BA 212-265-8070 firstname.lastname@example.org|
|Principal Investigator: Saud A. Sadiq, MD|
|Principal Investigator:||Saud A Sadiq, MD, FAAN||Tisch MS Research Center of New York|