- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving NCT07572071
Safety and Feasibility of Bilateral Striatal Transplantation of DopaCell in Parkinson's Disease
2. mai 2026 oppdatert av: 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.
Studieoversikt
Status
Rekruttering
Forhold
Intervensjon / Behandling
Detaljert beskrivelse
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
Intervensjonell
Registrering (Antatt)
10
Fase
- Fase 1
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiekontakt
- Navn: Sarvenaz Salahi, MD
- Telefonnummer: 021-23562000
- E-post: salahi13639@gmail.com
Studiesteder
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-
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Tehran, Iran
- Rekruttering
- Royan Institute
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Ta kontakt med:
- Sarvenaz Salahi, PhD
- Telefonnummer: 021-2356200
- E-post: salahi13639@gmail.com
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-
Deltakelseskriterier
Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Voksen
- Eldre voksen
Tar imot friske frivillige
Nei
Beskrivelse
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.
Studieplan
Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
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Eksperimentell: 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
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Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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Safety
Tidsramme: Baseline to 12 Months Post-Transplant
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Incidence and severity of treatment-emergent adverse events (TEAEs)
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Baseline to 12 Months Post-Transplant
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Safety
Tidsramme: 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.
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Baseline to 12 Months Post-Transplant
|
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Feasibility
Tidsramme: Baseline to 12 Months Post-Transplant
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Successful completion of a single-session intraputaminal transplantation with of the intended cell dose
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Baseline to 12 Months Post-Transplant
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Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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Efficacy
Tidsramme: Baseline to 12 Months Post-Transplant
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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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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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Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Studierekorddatoer
Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.
Studer hoveddatoer
Studiestart (Faktiske)
1. august 2025
Primær fullføring (Antatt)
1. august 2028
Studiet fullført (Antatt)
1. august 2030
Datoer for studieregistrering
Først innsendt
7. april 2026
Først innsendt som oppfylte QC-kriteriene
2. mai 2026
Først lagt ut (Faktiske)
7. mai 2026
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
7. mai 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
2. mai 2026
Sist bekreftet
1. april 2026
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- 402000063
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Nei
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
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