Ensayo aleatorizado controlado con placebo de fase IIa: Células madre mesenquimales como terapia modificadora de la enfermedad para la iPD
11 de agosto de 2026 actualizado por: Mya Schiess
Un ensayo aleatorizado, doble ciego, controlado con placebo de células madre mesenquimales derivadas de médula ósea alogénica como terapia modificadora de la enfermedad de Parkinson idiopática
El propósito de este estudio es seleccionar el número más seguro y efectivo de dosis repetidas de infusiones de células madre mesenquimales (MSC) derivadas de médula ósea alogénicas para retrasar la progresión de la enfermedad de Parkinson (EP).
Descripción general del estudio
Estado
Terminado
Condiciones
Intervención / Tratamiento
Descripción detallada
Estudio de fase IIa en un solo sitio de CMM alogénicas en un ensayo de control aleatorizado doble ciego como terapia modificadora de la enfermedad para la EP.
El diseño incluye tres brazos de tratamiento con 45 pacientes.
Tipo de estudio
Intervencionista
Inscripción (Actual)
45
Fase
- Fase 2
Contactos y Ubicaciones
Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.
Ubicaciones de estudio
-
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Texas
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Houston, Texas, Estados Unidos, 77030
- The University of Texas Health Science Center at Houston
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Criterios de participación
Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.
Criterio de elegibilidad
Edades elegibles para estudiar
50 años a 79 años (Adulto, Adulto Mayor)
Acepta Voluntarios Saludables
No
Descripción
Criterios de inclusión:
- Diagnóstico de la enfermedad de Parkinson según los criterios del banco de cerebros del Reino Unido, incluida la presencia de 2 signos cardinales de la EP más bradicinesia.
- Microsomía leve a anosmia.
- Una etapa modificada de Hoehn y Yahr de 3 o menos.
- Fecha de diagnóstico de EP entre 3 a 10 años
- Respuesta robusta a la terapia dopaminérgica.
Criterio de exclusión:
- Parkinsonismo atípico, vascular o inducido por fármacos.
- Una tomografía DAT atípica o una resonancia magnética que respaldan una explicación alternativa de los síntomas de la EP.
- Paciente que no toma medicamentos que contienen levodopa.
- Características clínicas de psicosis o alucinaciones refractarias.
- Una puntuación de Evaluación Cognitiva de Montreal (MoCA) de menos de 25.
- Trastorno convulsivo no controlado.
- Función anormal del riñón y del hígado.
- Presencia de hipotensión ortostática clínicamente refractaria en la visita de selección o de referencia.
- Índice de masa corporal mayor o igual a 35.
- Enfermedad cardíaca: antecedentes de insuficiencia cardíaca congestiva, bradicardia clínicamente significativa, presencia de bloqueo auriculoventricular de segundo o tercer grado.
- Enfermedad pulmonar: EPOC con requerimiento de oxígeno en reposo o con deambulación; o asma moderada a severa.
- Neoplasia maligna activa o diagnóstico de neoplasia maligna dentro de los 5 años anteriores al inicio de la detección
- Cualquier ideación o comportamiento suicida actual.
- Cualquier diagnóstico de enfermedad autoinmune o estado inmunocomprometido
- Antecedentes de accidentes cerebrovasculares de vasos medianos o grandes.
- Antecedentes de lesión cerebral traumática con pérdida de conciencia y síntomas neurológicos residuales.
- Cirugía mayor en los 3 meses anteriores o prevista en los 6 meses siguientes.
- Historial de uso de un fármaco en investigación dentro de los 90 días anteriores a la visita de selección.
- Historia de la cirugía cerebral para la EP.
- Trastorno por abuso de sustancias.
- Tratamiento anticoagulante activo y/o INR anormal.
Plan de estudios
Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: Aleatorizado
- Modelo Intervencionista: Asignación paralela
- Enmascaramiento: Triple
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
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Experimental: Células madre mesenquimales y placebo
2 infusiones de 10 X 10^6 MSC/kg y 1 infusión de placebo, todas las dosis administradas con 4 meses de diferencia.
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1 dosis es 10 X 10^6 MSC/kg
Otros nombres:
El placebo será idéntico al producto en investigación pero no contendrá células madre mesenquimales (MSC).
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Experimental: Células madre mesenquimales
3 infusiones de 10 X 10^6 MSC/kg administradas cada 4 meses.
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1 dosis es 10 X 10^6 MSC/kg
Otros nombres:
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Comparador de placebos: Placebo
3 dosis de placebo administradas cada 4 meses.
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El placebo será idéntico al producto en investigación pero no contendrá células madre mesenquimales (MSC).
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Bayesian Mean Estimate of the Proportion of Participants Achieving a ≥5-point Improvement on MDS-UPDRS Part III From Screening to Week 62, Compared Between Each Active MSC Dose Arm and Placebo
Periodo de tiempo: From Baseline to Week 62
|
The Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III assesses motor symptoms of Parkinson's disease. .
A responder was defined as a participant achieving a ≥5-point improvement (decrease of at least 5 points from screening) at Week 62. Reported are the Bayesian mean estimates of the proportion of participants achieving this response.
The 95% confidence interval represents the Bayesian 95% credible interval.
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From Baseline to Week 62
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Number of Participants With New-onset Organ Failure
Periodo de tiempo: Baseline through week 88
|
New-onset organ failure defined as a significant acute change in the kidney or liver function, leukocytosis, leukopenia or anemia sustained over 3 months; > 75 % reduction in GFR compared to baseline; altered liver function as defined by ALT >150 U/L and or total bilirubin >1.6 mg/dl; or leukopenia defined as < 4K WBC count or anemia as defined by Hgb < 12 for men and < 11 for women.
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Baseline through week 88
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Number of Participants Developing Donor-Specific Anti-HLA Antibodies
Periodo de tiempo: Baseline through week 88
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Baseline through week 88
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Motor Function as Measured by the Timed-Up-and-Go (TUG) Scale
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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This test uses the time that a person takes to rise from a chair, walk 7 meters, turn around, walk back to the chair, and sit down.
Time is recorded in seconds, a longer duration of time indicates a worse outcome.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Number of Participants With Lifetime Suicidal Ideation (C-SSRS)
Periodo de tiempo: Baseline
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The Columbia-Suicide Severity Rating Scale (C-SSRS) is an assessment tool that evaluates suicidal ideation and behavior.
Reported is the number of participants with a positive response to the lifetime suicidal ideation question, "Have you actually had any thoughts of killing yourself?"
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Baseline
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Parkinson's Disease Severity as Assessed by the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Total Score
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) assesses the severity of Parkinson's disease.
The total score ranges from 0 to 265, with higher scores indicating greater Parkinson's disease severity and disability.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Motor Symptoms of Parkinson's Disease as Assessed by the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III Motor Examination Score
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III Motor Examination assesses motor symptoms of Parkinson's disease.
The total score ranges from 0 to 132, with higher scores indicating greater motor impairment and more severe Parkinson's disease motor symptoms.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Global Measurement of Disability as Measured by the Change in the Screening "Off" Modified Hoehn and Yahr (H&Y)
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Modified Hoehn and Yahr (H&Y) Scale described the progress of Parkinson's disease.
Total score ranges from 0 to 5, with higher scores indicating a worse outcome.
The assessment was performed in the "off" medication state, and the score will be reported.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Quality of Life as Measured by the Modified Schwab and England Activities of Daily Living Scale (ADL)
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Schwab and England Activities of Daily Living (ADL) Scale assesses functional independence in individuals with Parkinson's disease.
Scores range from 0% to 100%, with higher percentages indicating greater independence and better functional ability in activities of daily living.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Quality of Life as Measured by the Parkinson's Disease Questionnaire-39 (PDQ-39)
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Parkinson's Disease Questionnaire-39 (PDQ-39) assesses health-related quality of life in individuals with Parkinson's disease.
Total scores range from 0 to 100, with higher scores indicating poorer quality of life.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Quality of Life as Measured by the EuroQol- 5 Dimension (EQ-5D) Index Score
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The EuroQol- 5 Dimension (EQ-5D) assesses quality of life.
The total score ranges between 0 to 1, a higher score indicating better quality of life.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Non- Motor Symptoms as Measured by the Non-Motor Symptoms Questionnaire (NMSQ)
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Non-Motor Symptoms Questionnaire (NMSQ) assesses the presence of non-motor symptoms associated with Parkinson's disease.
Total score ranges from 0 to 30, with a higher score indicating a greater number of non-motor symptoms.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Olfactory Function as Assessed by the University of Pennsylvania Smell Identification Test (UPSIT-40) Score
Periodo de tiempo: Baseline, Week 49, Week 88
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The University of Pennsylvania Smell Identification Test (UPSIT-40) assesses olfactory function.
The total score ranges from 0 to 40 with a higher score indicating better olfactory function.
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Baseline, Week 49, Week 88
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Cognitive Function as Measured by the Montreal Cognitive Assessment (MoCA)
Periodo de tiempo: Baseline, Week 49, Week 88
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The Montreal Cognitive Assessment (MoCA) assesses cognitive function.
The total score ranges from 0 to 30, a higher score indicates better cognitive function.
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Baseline, Week 49, Week 88
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Anxiety as Assessed by the Parkinson Anxiety Scale (PAS) Score
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Parkinson Anxiety Scale (PAS) assesses anxiety symptoms in individuals with Parkinson's disease.
The total score ranges from 0 to 48, with higher scores indicating greater anxiety severity.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Depressive Symptoms as Assessed by the Geriatric Depression Scale-Short Form (GDS-SF) Score
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Geriatric Depression Scale-Short Form (GDS-SF) assesses depressive symptoms.
The total score ranges from 0 to 15, with higher scores indicating greater depressive symptom severity.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Number of Participants Reporting Suicidal Ideation Since Last Visit
Periodo de tiempo: Week 9, Week 27, Week 49, Week 62, Week 88
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The Columbia-Suicide Severity Rating Scale (C-SSRS) assesses suicidal ideation and behavior.
Reported is the number of participants with a positive response to the suicidal ideation question, "Have you actually had any thoughts of killing yourself?"
since the previous study visit.
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Week 9, Week 27, Week 49, Week 62, Week 88
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Otras medidas de resultado
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Concentration of Growth Factors
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Concentration of Chemokines in Patient Blood Sample.
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Concentration of Neurotransmitters
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Examples of these are homovanillic acid and 5-hydroxytryptamine.
These will be measured in CSF and blood by ELISA.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Alpha-synuclein Oligomers in the Blood (Serum or Plasma)
Periodo de tiempo: Baseline, Week 40, Week 49
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Baseline, Week 40, Week 49
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Alpha-synuclein Oligomers in the Cerebral Spinal Fluid
Periodo de tiempo: Baseline,week 49
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Baseline,week 49
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Concentration of Cytokines in Patient Blood Sample.
Periodo de tiempo: Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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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.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Measurement of Putative Paracrine Mechanism of MSCs Using Neuroimaging
Periodo de tiempo: Baseline, week 40,week 88
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Baseline, week 40,week 88
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Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Mya C Schiess, MD, The University of Texas Health Science Center, Houston
Publicaciones y enlaces útiles
La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.
Publicaciones Generales
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- Nagatsu T, Mogi M, Ichinose H, Togari A. Cytokines in Parkinson's disease. J Neural Transm Suppl. 2000;(58):143-51.
- 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.
- 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.
- 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.
Fechas de registro del estudio
Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.
Fechas importantes del estudio
Inicio del estudio (Actual)
9 de noviembre de 2020
Finalización primaria (Actual)
30 de julio de 2023
Finalización del estudio (Actual)
30 de julio de 2023
Fechas de registro del estudio
Enviado por primera vez
16 de julio de 2020
Primero enviado que cumplió con los criterios de control de calidad
6 de agosto de 2020
Publicado por primera vez (Actual)
10 de agosto de 2020
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
2 de septiembre de 2026
Última actualización enviada que cumplió con los criterios de control de calidad
11 de agosto de 2026
Última verificación
1 de agosto de 2026
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- HSC-MS-20-0150
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
NO
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Sí
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
No
Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .