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

2 maj 2026 uppdaterad 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.

Studieöversikt

Detaljerad beskrivning

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.

Studietyp

Interventionell

Inskrivning (Beräknad)

10

Fas

  • Fas 1

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Studieorter

      • Tehran, Iran
        • Rekrytering
        • Royan Institute
        • Kontakt:

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

  • Vuxen
  • Äldre vuxen

Tar emot friska volontärer

Nej

Beskrivning

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

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: N/A
  • Interventionsmodell: Enskild gruppuppgift
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: 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

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Safety
Tidsram: Baseline to 12 Months Post-Transplant
Incidence and severity of treatment-emergent adverse events (TEAEs)
Baseline to 12 Months Post-Transplant
Safety
Tidsram: 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
Tidsram: 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

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Efficacy
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: Baseline to 60 Months Post-Transplant
Continuous Safety: The number and severity of treatment-emergent adverse events
Baseline to 60 Months Post-Transplant

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

1 augusti 2025

Primärt slutförande (Beräknad)

1 augusti 2028

Avslutad studie (Beräknad)

1 augusti 2030

Studieregistreringsdatum

Först inskickad

7 april 2026

Först inskickad som uppfyllde QC-kriterierna

2 maj 2026

Första postat (Faktisk)

7 maj 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

7 maj 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

2 maj 2026

Senast verifierad

1 april 2026

Mer information

Termer relaterade till denna studie

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Nej

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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