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| Name | Class |
|---|---|
| Michael J. Fox Foundation for Parkinson's Research | OTHER |
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The purpose of this study is to select the safest and most effective number of repeat doses of allogeneic bone marrow-derived mesenchymal stem cell (MSC) infusions to slow the progression of Parkinson's disease (PD).
Single site phase IIa study of allogeneic MSC in a double blind randomized control trial as disease modifying therapy for PD. The design includes three treatment arms with 45 patients.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Mesenchymal Stem Cells and Placebo | Experimental | 2 infusions of 10 X 10^6 MSC/kg and 1 placebo infusion, all doses administered 4 months apart. |
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| Mesenchymal Stem Cells | Experimental | 3 infusions of 10 X 10^6 MSC/kg administered every 4 months. |
|
| Placebo | Placebo Comparator | 3 placebo doses administered every 4 months. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Mesenchymal Stem Cells | Drug | 1 dose is 10 X 10^6 MSC/kg |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale | Screening | |
| Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale | Week 7, post infusion #1 | |
| Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale | Week 20, post infusion #2 | |
| Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale | Week 29, post-infusion #3 | |
| Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale | Week 39 follow-up | |
| Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale | Week 52 follow-up | |
| Safest number of effective doses of MSC as measured by the Part III of the Movement Disorder Society Unified Parkinson's disease Rating Scale (MDS-UPDRS) scale | Week 78 follow-up |
| Measure | Description | Time Frame |
|---|---|---|
| Safety and tolerability as measured by serious adverse reactions. | Baseline,week 7,week 20,week 29,week 39,week 78 | |
| Safety and tolerability as measured by immunologic responses. | Donor specific antibodies (DSA) in serum of patients will be measured and compared to baseline to determine if there is a development of antibody response to donor HLA (human leukocyte antigen). This will determine if there is a possibility of anti-donor alloimmune response after the first or second infusion of MSC, which might lead to a subsequent antibody-mediated rejection (AMR) with the following infusion. Testing will be done at the these time points to determine if 2nd or 3rd infusions will continue. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Mya C Schiess, MD | The University of Texas Health Science Center, Houston | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The University of Texas Health Science Center at Houston | Houston | Texas | 77030 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30584159 | Background | Dorsey ER, Sherer T, Okun MS, Bloem BR. The Emerging Evidence of the Parkinson Pandemic. J Parkinsons Dis. 2018;8(s1):S3-S8. doi: 10.3233/JPD-181474. | |
| 12498954 | Background | Braak H, Del Tredici K, Rub U, de Vos RA, Jansen Steur EN, Braak E. Staging of brain pathology related to sporadic Parkinson's disease. Neurobiol Aging. 2003 Mar-Apr;24(2):197-211. doi: 10.1016/s0197-4580(02)00065-9. |
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| Placebo | Drug | Placebo will be identical to the investigational product but will not contain mesenchymal stem cells (MSCs). |
|
| Baseline,week 7,week 20,week 29,week 39,week 78 |
| Motor function as measured by the Timed-Up-and-Go (TUG) scale | This test uses the time that a person takes to rise from a chair, walk three meters, turn around, walk back to the chair, and sit down. A longer duration of time indicates a worse outcome | Baseline,week 7,week 20,week 29,week 39,week 52,week 78 |
| Global measurement of disability as measured by the change in the screening "Off" modified Hoehn and Yahr (H&Y) | Baseline,week 7,week 20,week 29,week 39,week 52,week 78 |
| Quality of life as measured by the modified Schwab and England activities of daily living scale (ADL) | Baseline,week 7,week 20,week 29,week 39,week 52,week 78 |
| Quality of life as measured by the Parkinson's Disease Questionnaire 39 (PDQ-39) | Baseline,week 7,week 20,week 29,week 39,week 52,week 78 |
| Quality of life as measured by the EuroQol- 5 Dimension (EQ-5D) | Baseline,week 7,week 20,week 29,week 39,week 52,week 78 |
| Non-motor symtoms as measured by the The University of Pennsylvania Smell Identification Test (UPSIT- 40 odor test booklet). | Baseline,week 29,week 78 |
| Cognitive function as measured by the the change in Montreal Cognitive Assessment (MoCA) | Baseline,week 29,week 78 |
| Behavioral changes as measured by the Columbia Suicide Severity Rating Scale (C-SSRS) | The Columbia-Suicide Severity Rating Scale (C-SSRS) is an assessment tool that evaluates suicidal ideation and behavior. It rates an individual's degree of suicidal ideation on a scale, ranging from "wish to be dead" to "active suicidal ideation with specific plan and intent". Exclusionary criteria views 1 or more positive answers in the last month as not eligible for participation. In follow up, one positive response requires further investigation. | Baseline,week 7,week 20,week 29,week 39,week 52,week 78 |
| Behavioral changes as measured by the Geriatric Depression Scale-Short Form (GDS-SF) | The Geriatric Depression Scale Short form (GDS-SF) is a 15-item screening tool that is used to identify depression in older adults. Answers indicating depression are in bold and italicized; score one point for each bold one selected. A score of 0 to 5 is normal. A score greater than 5 suggests depression and requires evaluation, whereas a score of 10 or greater would require evaluation for treatment . | Baseline,week 7,week 20,week 29,week 39,week 52,week 78 |
| Behavioral changes as measured by the Parkinson Anxiety Scale (PAS) | Baseline,week 7,week 20,week 29,week 39,week 52,week 78 |
| Measurement of putative paracrine mechanism of MSCs using neuroimaging | Baseline,week 29,week 78 |
| Measurement of putative paracrine mechanism of MSCs as measured by concentration of cytokines in patient blood sample. | Examples of these are IL-1beta, IL-6, TNF-alpha, COX-2 and PGE-2. These are measured by Magnetic Bead Panel - Multiplex Assay or ELISA, allowing for specificity in the blood. | Baseline,week 7,week 20,week 29,week 39,week 78 |
| Measurement of putative paracrine mechanism of MSCs as measured by concentration of chemokines in patient blood sample. | Examples of these are CCL2 (MCP-1), CCL7 (MCP-3), CCL11 (Eotaxin) CCL2 (MDC), CXCL10 (IP-10), CX3CL1 (Fractalkine). These will be measured by Magnetic Bead Panel - Multiplex Assay or ELISA, allowing for specificity in the blood. | Baseline,week 7,week 20,week 29,week 39,week 78 |
| Measurement of putative paracrine mechanism of MSCs as measured by concentration of growth factors | Examples of these are Glial Derived Neurotrophic Factor, Brain Derived Neurotrophic Factor and Vascular Epidermal Growth Factor . These will be measured by ELISA specific to blood and CSF. | Baseline,week 7,week 20,week 29,week 39,week 78 |
| Measurement of putative paracrine mechanism of MSCs as measured by concentration of neurotransmitters | Examples of these are homovanillic acid and 5-hydroxytryptamine. These will be measured in CSF and blood by ELISA. | Baseline,week 7,week 20,week 29,week 39,week 78 |
| Measurement of putative paracrine mechanism of MSCs as measured by alpha-synuclein oligomers in the blood (serum or plasma) | Basleline,week 29,week 39,week 78 |
| Measurement of putative paracrine mechanism of MSCs as measured by alpha-synuclein oligomers in the cerebral spinal fluid | Baseline,week 39 |
| 24756517 | Background | Connolly BS, Lang AE. Pharmacological treatment of Parkinson disease: a review. JAMA. 2014 Apr 23-30;311(16):1670-83. doi: 10.1001/jama.2014.3654. |
| 20070435 | Background | Jellinger KA. Basic mechanisms of neurodegeneration: a critical update. J Cell Mol Med. 2010 Mar;14(3):457-87. doi: 10.1111/j.1582-4934.2010.01010.x. Epub 2010 Jan 11. |
| 15668963 | Background | Kortekaas R, Leenders KL, van Oostrom JC, Vaalburg W, Bart J, Willemsen AT, Hendrikse NH. Blood-brain barrier dysfunction in parkinsonian midbrain in vivo. Ann Neurol. 2005 Feb;57(2):176-9. doi: 10.1002/ana.20369. |
| 25757748 | Background | Gray MT, Woulfe JM. Striatal blood-brain barrier permeability in Parkinson's disease. J Cereb Blood Flow Metab. 2015 May;35(5):747-50. doi: 10.1038/jcbfm.2015.32. Epub 2015 Mar 11. |
| 12531233 | Background | Orr CF, Rowe DB, Halliday GM. An inflammatory review of Parkinson's disease. Prog Neurobiol. 2002 Dec;68(5):325-40. doi: 10.1016/s0301-0082(02)00127-2. |
| 11128604 | Background | Nagatsu T, Mogi M, Ichinose H, Togari A. Cytokines in Parkinson's disease. J Neural Transm Suppl. 2000;(58):143-51. |
| 8945728 | Background | Stypula G, Kunert-Radek J, Stepien H, Zylinska K, Pawlikowski M. Evaluation of interleukins, ACTH, cortisol and prolactin concentrations in the blood of patients with parkinson's disease. Neuroimmunomodulation. 1996 Mar-Jun;3(2-3):131-4. doi: 10.1159/000097237. |
| 21082892 | Background | Joyce N, Annett G, Wirthlin L, Olson S, Bauer G, Nolta JA. Mesenchymal stem cells for the treatment of neurodegenerative disease. Regen Med. 2010 Nov;5(6):933-46. doi: 10.2217/rme.10.72. |
| 40993774 | Derived | Martinez-Lemus JD, Molony DA, Suescun J, Tharp E, Thomas TS, Green C, Onuigbo C, Ritter R 3rd, Schiess MC. Allogeneic bone marrow-derived mesenchymal stem cells in the aging kidney: secondary results of a Parkinson's disease clinical trial. Stem Cell Res Ther. 2025 Sep 24;16(1):493. doi: 10.1186/s13287-025-04577-y. |
| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |
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