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Safety and Feasibility of Bilateral Striatal Transplantation of DopaCell in Parkinson's Disease
2 mei 2026 bijgewerkt door: 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.
Studie Overzicht
Toestand
Werving
Conditie
Interventie / Behandeling
Gedetailleerde beschrijving
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.
Studietype
Ingrijpend
Inschrijving (Geschat)
10
Fase
- Fase 1
Contacten en locaties
In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.
Studiecontact
- Naam: Sarvenaz Salahi, MD
- Telefoonnummer: 021-23562000
- E-mail: salahi13639@gmail.com
Studie Locaties
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Tehran, Iran
- Werving
- Royan Institute
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Contact:
- Sarvenaz Salahi, PhD
- Telefoonnummer: 021-2356200
- E-mail: salahi13639@gmail.com
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Deelname Criteria
Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Volwassen
- Oudere volwassene
Accepteert gezonde vrijwilligers
Nee
Beschrijving
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.
Studie plan
Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: NVT
- Interventioneel model: Opdracht voor een enkele groep
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
|---|---|
|
Experimenteel: Transplantation
Experimental: DopaCell Biological: DopaCell, 5 million cells per putamen Immunosuppressive Regimen: Basiliximab, Tacrolimus, Prednisolone Device: Customized microinjection device
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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
|
Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Safety
Tijdsspanne: Baseline to 12 Months Post-Transplant
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Incidence and severity of treatment-emergent adverse events (TEAEs)
|
Baseline to 12 Months Post-Transplant
|
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Safety
Tijdsspanne: Baseline to 12 Months Post-Transplant
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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
Tijdsspanne: Baseline to 12 Months Post-Transplant
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Successful completion of a single-session intraputaminal transplantation with of the intended cell dose
|
Baseline to 12 Months Post-Transplant
|
Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Efficacy
Tijdsspanne: 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
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Baseline to 12 Months Post-Transplant
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Efficacy
Tijdsspanne: Baseline to 12 Months Post-Transplant
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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.)
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Baseline to 12 Months Post-Transplant
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Efficacy
Tijdsspanne: Baseline to 12 Months Post-Transplant
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Graft survival following intracerebral DopaCells transplantation based on TRODAT SPECT imaging findings.
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Baseline to 12 Months Post-Transplant
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Continuous Safety
Tijdsspanne: Baseline to 60 Months Post-Transplant
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Continuous Safety: The number and severity of treatment-emergent adverse events
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Baseline to 60 Months Post-Transplant
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Medewerkers en onderzoekers
Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.
Studie record data
Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.
Bestudeer belangrijke data
Studie start (Werkelijk)
1 augustus 2025
Primaire voltooiing (Geschat)
1 augustus 2028
Studie voltooiing (Geschat)
1 augustus 2030
Studieregistratiedata
Eerst ingediend
7 april 2026
Eerst ingediend dat voldeed aan de QC-criteria
2 mei 2026
Eerst geplaatst (Werkelijk)
7 mei 2026
Updates van studierecords
Laatste update geplaatst (Werkelijk)
7 mei 2026
Laatste update ingediend die voldeed aan QC-criteria
2 mei 2026
Laatst geverifieerd
1 april 2026
Meer informatie
Termen gerelateerd aan deze studie
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- 402000063
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Nee
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
Nee
Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .
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