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| ID | Type | Description | Link |
|---|---|---|---|
| IND 13889 | Other Identifier | FDA |
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The study is an open-label, phase I clinical trial designed to evaluate the safety and tolerability of intrathecal administration of autologous mesenchymal stem cell-derived neural progenitor cells (MSC-NP) in patients with progressive multiple sclerosis. Study participants will receive three intrathecal injections of culture-expanded autologous MSC-NPs at three month intervals.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| autologous MSC-NP | Experimental | intrathecal administration of autologous MSC-NP in three doses at three month intervals |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| intrathecal administration of autologous MSC-NP | Biological | Autologous MSC-NPs administered intrathecally at a dose between 2 and 10 million cells, depending on ex vivo expansion characteristics. Three doses will be administered at 3 month intervals. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with adverse events | The primary objective of the study is to assess the safety and tolerability of intrathecal therapy with autologous MSCNPs in MS. Number of participants with adverse events will be documented 1 day, 1 week, 1 month and 2 months post treatment for three treatments (followup for third treatment is 3 months instead of 2 months). | 9 months |
| Number of participants with adverse events . | The co-primary objective will be to observe long term safety of the treatment 6 months and 30 months following the last treatment. | 30 months |
| Measure | Description | Time Frame |
|---|---|---|
| Preliminary evaluation of efficacy | The secondary objective is to observe trends in efficacy over the course of the experimental treatment. Outcome measures include evoked potentials (baseline and 3 months post 3rd dose) ,quality of life questionnaire, EDSS, and MSFC (baseline, at the time of each dose, and 3 and 6 months after third dose), and MRI (baseline, 2 months after first dose, and 3 and 24 months after third dose). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Saud A Sadiq, MD | Tisch MS Research Center of New York | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Tisch MS Research Center of New York | New York | New York | 10019 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23197858 | Background | Harris VK, Faroqui R, Vyshkina T, Sadiq SA. Characterization of autologous mesenchymal stem cell-derived neural progenitors as a feasible source of stem cells for central nervous system applications in multiple sclerosis. Stem Cells Transl Med. 2012 Jul;1(7):536-47. doi: 10.5966/sctm.2012-0015. Epub 2012 Jun 28. | |
| 21962795 | Background |
| Label | URL |
|---|---|
| Tisch MS Research Center of New York | View source |
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| 9 months |
| Harris VK, Yan QJ, Vyshkina T, Sahabi S, Liu X, Sadiq SA. Clinical and pathological effects of intrathecal injection of mesenchymal stem cell-derived neural progenitors in an experimental model of multiple sclerosis. J Neurol Sci. 2012 Feb 15;313(1-2):167-77. doi: 10.1016/j.jns.2011.08.036. Epub 2011 Oct 1. |
| 29449193 | Result | Harris VK, Stark J, Vyshkina T, Blackshear L, Joo G, Stefanova V, Sara G, Sadiq SA. Phase I Trial of Intrathecal Mesenchymal Stem Cell-derived Neural Progenitors in Progressive Multiple Sclerosis. EBioMedicine. 2018 Mar;29:23-30. doi: 10.1016/j.ebiom.2018.02.002. Epub 2018 Feb 3. |
| 33277427 | Derived | Harris VK, Stark JW, Yang S, Zanker S, Tuddenham J, Sadiq SA. Mesenchymal stem cell-derived neural progenitors in progressive MS: Two-year follow-up of a phase I study. Neurol Neuroimmunol Neuroinflamm. 2020 Dec 4;8(1):e928. doi: 10.1212/NXI.0000000000000928. Print 2021 Jan. |
| ID | Term |
|---|---|
| D009103 | Multiple Sclerosis |
| ID | Term |
|---|---|
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D003711 | Demyelinating Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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