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Safety and Feasibility of Bilateral Striatal Transplantation of DopaCell in Parkinson's Disease

2 de mayo de 2026 actualizado por: Royan Institute

Evaluation of the Safety and Feasibility of a Single Transplantation of 10 Million Human Embryonic Stem Cell-Derived Dopaminergic Progenitor Cells Into the Bilateral Striatum of Patients With Moderately Severe Parkinson's Disease: a Multicenter, Open-label, Single-arm Phase I Clinical Trial

Dopason is a phase I, open-label, multicenter, single-arm clinical trial designed to evaluate the safety and feasibility of intraputaminal transplantation of human embryonic stem cell-derived dopaminergic progenitor cells (DopaCells) in patients with moderately severe Parkinson's disease.

Descripción general del estudio

Descripción detallada

During this trial phase, a total of ten participants will be included and distributed between two clinical centers. Each participant will undergo one standardized surgical procedure consisting of bilateral stereotactic intraputaminal transplantation of approximately 10 × 10⁶ cells in total (about 5 × 10⁶ cells per putamen), aiming to achieve an estimated survival of 200,000 neurons. All procedures will be performed using the same surgical delivery system and an almost identical operative protocol across both sites to ensure procedural consistency. After transplantation, patients will receive immunosuppressive therapy for a duration of one year and will be systematically followed for study outcomes for at least 12 months.

Tipo de estudio

Intervencionista

Inscripción (Estimado)

10

Fase

  • Fase 1

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.

Estudio Contacto

  • Nombre: Sarvenaz Salahi, MD
  • Número de teléfono: 021-23562000
  • Correo electrónico: salahi13639@gmail.com

Ubicaciones de estudio

      • Tehran, Irán
        • Reclutamiento
        • Royan Institute
        • Contacto:

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

  • Adulto
  • Adulto Mayor

Acepta Voluntarios Saludables

No

Descripción

Inclusion Criteria:

  • Age: 30-70 years
  • Diagnosis of PD: MDS clinical Diagnostic Criteria for Parkinson's disease
  • The disease duration more than 5 years
  • Moderate Parkinson's disease, defined as a Hoehn and Yahr stage of 2 or 3 during the OFF period.
  • The patient is receiving oral pharmacological therapy, and in the opinion of the Principal Investigator, the patient's symptoms remain inadequately controlled despite optimal medical management, or the patient is experiencing adverse effects related to their current treatment
  • No history or only mild levodopa-induced dyskinesia, defined as a score of 2 or less on the UDysRS scale in any body region during the ON state.
  • The patient demonstrates a clinically meaningful response to a therapeutic dose of levodopa, as determined by the Principal Clinical Investigator or a trained specialist under the supervision of the Principal Investigator.
  • The performance of different organs based on laboratory evaluations:

    • Number of neutrophils ≥2000 / microliter
    • Platelet count ≥100,000 / microliter
    • AST / ALT: less than or equal to three times the maximum normal value at the intervention site
    • Total bilirubin less than or equal to 1.5 times the maximum normal amount at the intervention site
    • eGFR * rate: greater than or equal to 60 ml / min / 1.73 m2 * eGFR (mL / min / 1.73 m2) = 194 X Cr ^ -1.094 X age ^ -0.287 (X 0.739 for females)
  • Informed consent

Exclusion Criteria:

  • The abnormal function of immune system
  • The symptomatic brain injuries (brain atrophy, cerebral Infarct, trauma, vascular malformation) confirmed by brain MRI
  • Markedly reduced or normal signal in the ventral striatum on TRO-DaT SPECT imaging.
  • Any abnormal findings on brain MRI.
  • Positive GBA mutation test.
  • Diagnosis of dementia based on a MoCA score < 24.
  • The abnormality of thrombotic system or high risk of bleeding
  • Positive for any of the following viral markers or active infections: HBsAg, HBsAb, HBcAb, anti-HIV antibodies, anti-HTLV-1&2 antibodies, active hepatitis C infection, syphilis, or active CMV, VZV, EBV, or COVID-19 infection.
  • Impossibility of MRI imaging for patients with metal in the body, pacemaker in the body, claustrophobia, with artificial heart valves that are incompatible with MRI or body weight is not within the tolerable range for MRI.
  • Patients with contraindications to the study drug: Tacrolimus, Prednisolone, Basiliximab, Cotrimoxazole, MRI contrast agent.
  • Patients undergoing other cell transplants, including embryonic stem cell-derived dopaminergic progenitor cells.
  • Patients with a history of PD at the same time and concurrent: Malignant neoplasm, epilepsy, cerebral hemorrhage or a positive history
  • Psychiatric disorders confirmed by a psychiatrist, including major depression, bipolar disorder, or schizophrenia, that are uncontrolled or treatment resistant.
  • Patients with intellectual disability who, in the judgment of a psychiatrist, are unable to fully comprehend the study requirements.
  • History of pallidotomy, thalamotomy, or deep brain stimulation (DBS).
  • Patients considered high-risk candidates for surgery, particularly neurosurgery or DBS implantation, due to significant cardiovascular, pulmonary, or other systemic comorbidities identified during preoperative evaluation.
  • Patients who have a history of taking the following in the three months prior to enrollment: Immunosuppressant, antipsychotic drug, anticonvulsant drugs or anticoagulant therapy (if discontinuation or perioperative adjustment is not feasible), botulinum toxin (within 6 months), phenol injections, or other treatments for dystonia or muscle spasm
  • History of Apomorphine use
  • History of chronic alcohol use or illicit drug abuse.
  • Patients who are pregnant, lactating, or people who did not avoid pregnancy during the study.
  • Patients who, according to the researchers' opinions, are not suitable for safe study.

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: N / A
  • Modelo Intervencionista: Asignación de un solo grupo
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Transplantation
Experimental: DopaCell Biological: DopaCell, 5 million cells per putamen Immunosuppressive Regimen: Basiliximab, Tacrolimus, Prednisolone Device: Customized microinjection device
DopaCells are dopamine-producing progenitor cells generated from human embryonic stem cells through differentiation under GMP-compliant conditions.
Basiliximab, Tacrolimus, Prednisolone
For intraputaminal delivery of DopaCell

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Safety
Periodo de tiempo: Baseline to 12 Months Post-Transplant
Incidence and severity of treatment-emergent adverse events (TEAEs)
Baseline to 12 Months Post-Transplant
Safety
Periodo de tiempo: Baseline to 12 Months Post-Transplant
Evidence of any post-transplantation anatomical changes at the transplant site suggestive of tumor formation, as assessed by MRI imaging.
Baseline to 12 Months Post-Transplant
Feasibility
Periodo de tiempo: Baseline to 12 Months Post-Transplant
Successful completion of a single-session intraputaminal transplantation with of the intended cell dose
Baseline to 12 Months Post-Transplant

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Efficacy
Periodo de tiempo: Baseline to 12 Months Post-Transplant
Changes in the duration of OFF and ON periods following intracerebral DopaCells transplantation as assessed by the Hauser diary
Baseline to 12 Months Post-Transplant
Efficacy
Periodo de tiempo: Baseline to 12 Months Post-Transplant
Changes in the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score from baseline following intracerebral DopaCells transplantation during ON and OFF periods ( MDS-UPDRS Part III ranges from 0 to 132, where higher scores indicate worse motor impairment.)
Baseline to 12 Months Post-Transplant
Efficacy
Periodo de tiempo: Baseline to 12 Months Post-Transplant
Graft survival following intracerebral DopaCells transplantation based on TRODAT SPECT imaging findings.
Baseline to 12 Months Post-Transplant
Continuous Safety
Periodo de tiempo: Baseline to 60 Months Post-Transplant
Continuous Safety: The number and severity of treatment-emergent adverse events
Baseline to 60 Months Post-Transplant

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

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)

1 de agosto de 2025

Finalización primaria (Estimado)

1 de agosto de 2028

Finalización del estudio (Estimado)

1 de agosto de 2030

Fechas de registro del estudio

Enviado por primera vez

7 de abril de 2026

Primero enviado que cumplió con los criterios de control de calidad

2 de mayo de 2026

Publicado por primera vez (Actual)

7 de mayo de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

7 de mayo de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

2 de mayo de 2026

Última verificación

1 de abril de 2026

Más información

Términos relacionados con este estudio

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

No

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. .

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