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

2 mai 2026 mis à jour par: 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.

Aperçu de l'étude

Description détaillée

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.

Type d'étude

Interventionnel

Inscription (Estimé)

10

Phase

  • La phase 1

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Coordonnées de l'étude

Lieux d'étude

      • Tehran, L'Iran
        • Recrutement
        • Royan Institute
        • Contact:

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

  • Adulte
  • Adulte plus âgé

Accepte les volontaires sains

Non

La description

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 d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: N / A
  • Modèle interventionnel: Affectation à un seul groupe
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: 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

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Safety
Délai: Baseline to 12 Months Post-Transplant
Incidence and severity of treatment-emergent adverse events (TEAEs)
Baseline to 12 Months Post-Transplant
Safety
Délai: 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
Délai: 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

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
Efficacy
Délai: 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
Délai: 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
Délai: 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
Délai: Baseline to 60 Months Post-Transplant
Continuous Safety: The number and severity of treatment-emergent adverse events
Baseline to 60 Months Post-Transplant

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

1 août 2025

Achèvement primaire (Estimé)

1 août 2028

Achèvement de l'étude (Estimé)

1 août 2030

Dates d'inscription aux études

Première soumission

7 avril 2026

Première soumission répondant aux critères de contrôle qualité

2 mai 2026

Première publication (Réel)

7 mai 2026

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

7 mai 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

2 mai 2026

Dernière vérification

1 avril 2026

Plus d'information

Termes liés à cette étude

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Non

Étudie un produit d'appareil réglementé par la FDA américaine

Non

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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