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IRCT20160704028786N2 IRCT 2023-08-12 Iranian academic center for education culture and research Safety and Feasibility of DopaCell transplantation in patients with Parkinson's disease DOPASON study DOPASON project: The Assessment of Safety and Feasibility of Intra-Striatal Transplantation of Human Embryonic stem cell-derived Dopaminergic Progenitors (DopaCell) in Parkinson’s disease: A Multicenter Phase I Clinical Trial 2023-12-22 anticipated 4 Complete https://irct.ir/trial/71062 interventional Randomization: N/A, Blinding: Not blinded, Placebo: Not used, Assignment: Single, Purpose: Treatment. 1 Parkinson's disease. The cell suspension prepared for transplantation includes caudal ventral mesencephalic dopaminergic progenitor cells derived from human embryonic stem cells (DopaCell) with a concentration of 100,000 cells per microliter, which will be prepared under GMP conditions and with clinical quality. The cell suspension will be injected into the striatum of both hemispheres of the patient during one stereotactic surgery. In each hemisphere, three routes inside the striatum (in the putamen and in front, at, and behind the anterior commissure) will be chosen for the injection of cell suspension, and the total volume of injection for each hemisphere will be 50 microliters. The cell suspension will be injected at a rate of one microliter per minute. To perform this surgery, the usual instrument used to move the microelectrodes in DBS will be used. The injection routes will be determined for each patient using imaging and routing software used in DBS surgery.. Undecided - It is not yet known if there will be a plan to make this available Justification or reason for indecision in sharing IPD is There is no further information
public Sarvenaz Salahi
Royan Institute for Stem Cell Biology and Technology, Hafez Alley Nr.2 , Banihashem, Shahid Soleymani highway
Tehran Iran (Islamic Republic of) 16635148 +98 21 2356 2000 salahi13639@gmail.com Iranian academic center for education culture and research scientific Mohammad Roohani; Vahid Reza Ostovan
Royan Institute for Stem Cell Biology and Technology, Hafez Alley Nr.2 , Banihashem, Shahid Soleymani highway
Tehran Iran (Islamic Republic of) 16635148 +98 21 2356 2000 salahi13639@gmail.com Iran University of Medical Sciences; Shiraz University of Medical Sciences Iran (Islamic Republic of) Iran (Islamic Republic of) Definitive diagnosis of PD based on MDS Clinical Diagnostic Criteria for Parkinson's Disease (2015) The duration of the disease must be at least 5 years from the time of diagnosis Patients should have ON- and OFF-period and have Hoehn Yahr stage≥III during OFF-period and Hoehn Yahr stage<III during ON-period. Patients should only receive drug treatment at the time of study entry The patient does not have levodopa-induced dyskinesia The patient must have at least 30% response to levodopa therapeutic dose (based on UPDRS part III). The function of different organs based on laboratory evaluations, within 7 days of entering the study, should include: (1) neutrophil count ≥2000/μl (2) platelet count ≥100,000/μl (3) AST/ALT ≤3 times the maximum normal value at the intervention site (4) total bilirubin value ≤ 1.5 times the maximum normal value at the intervention site (5) eGFR level: greater than or equal to 60 ml/min/1.73 square meters. Before entering the study, the patient must submit a written consent form to participate in the research. (If the patient is unable to write due to illness, the patient's legal guardian must complete the written consent form, or if the legal guardian is not present, the patient must verbally inform the researcher of consent to enter the study.) 30 years 70 years Both Patient with underlying disease (Such as abnormal immune system) Patient with symptomatic brain damage confirmed by MRI Patient with dementia. Patient with a GBA mutation. Patient with an abnormal coagulation system Patient with positive viral markers. Patient considered high-risk for surgical intervention (high risk of cardiovascular disease, pulmonary and other systemic diseases in pre-op evaluation) Patient with concurrent neurological disorder such as malignancy, neoplasm, epilepsy, cerebral hemorrhage or a positive history of it, or uncontrolled mental diseases. Patient with contraindications for MRI (the presence of metal in the body, the presence of a pacemaker in the body, claustrophobia, with artificial heart valves that are incompatible with MRI, body weight not within the tolerable range for the MRI table). Patient cannot tolerate the use of immunosuppressive regimens (including azathioprine, prednisolone, tacrolimus), concomitant medications (such as levodopa, carbidopa, MRI contrast agent), cotrimoxazole, or bovine serum derivatives. Patient that has undergone another cell transplantation. Patient that is pregnant or lactating. Patient who, according to the researcher's opinion, is not suitable for conducting the study safely. Patient with a history of chronic alcohol or drug use. Patient with a history of taking immunosuppressive drugs, antipsychotic drugs, anticonvulsant drugs, anticoagulant drugs, or apomorphine in the last three months. Patient with a history of using botulism toxin, phenol, or any drug related to the treatment of dystonia or muscle cramps in the six months leading to the study. G20 Parkinson's disease Treatment - Other The cell suspension prepared for transplantation includes caudal ventral mesencephalic dopaminergic progenitor cells derived from human embryonic stem cells (DopaCell) with a concentration of 100,000 cells per microliter, which will be prepared under GMP conditions and with clinical quality. The cell suspension will be injected into the striatum of both hemispheres of the patient during one stereotactic surgery. In each hemisphere, three routes inside the striatum (in the putamen and in front, at, and behind the anterior commissure) will be chosen for the injection of cell suspension, and the total volume of injection for each hemisphere will be 50 microliters. The cell suspension will be injected at a rate of one microliter per minute. To perform this surgery, the usual instrument used to move the microelectrodes in DBS will be used. The injection routes will be determined for each patient using imaging and routing software used in DBS surgery. Safety and feasibility. Timepoint: during hospitalization, month 1, 3, 6, 9, 12 after intervention. Method of measurement: Checklist of adverse events based on CTCAE version 4. Clinical presentation in On-period. Timepoint: During hospitalization, 1, 3, 6, 9, 12 months after the intervention. Method of measurement: MDS-UPDRS questionnaire. Clinical presentation in Off-period. Timepoint: Month 1, 6, 12 after the intervention. Method of measurement: MDS-UPDRS questionnaire. Parkinson's disease severity. Timepoint: Months 1, 3, 6, 9, 12 after the intervention. Method of measurement: Hoehn-Yahr scale. Quality of life. Timepoint: Months 1, 3, 6, 9, 12 after the intervention. Method of measurement: PDQ-39 questionnaire. Medication dosage. Timepoint: Months 1, 3, 6, 9, 12 after the intervention. Method of measurement: Levodopa equivalent dose. On- and Off-period with and without dyskinesia. Timepoint: Months 1, 6, 12 after intervention. Method of measurement: Hauser diary checklist. Graft function. Timepoint: Month 12 after intervention. Method of measurement: TRO-DaT SPECT imaging. Psychiatric status. Timepoint: During hospitalization, 1, 3, 6, 9, 12 months after intervention. Method of measurement: Psychiatric interview, Montreal cognitive assessment questionnaires, Beck anxiety inventory, Beck depression inventory, Positive and negative syndrome scale, Brief psychiatric rating scale, Yale-brown obsessive compulsive scale, Hamilton depression rating scale. Iran University of Medical Sciences Shiraz University of Medical Sciences Science and Technology Vice-Presidency Science and Technology Vice-Presidency Science and Technology Vice-Presidency Approved 2023-08-08 Research Ethics Committee of Royan Institute- Academic Center for Education, Culture, and Research No. 12, Hafez St., Banihashem St., Qasem Soleimani Expressway (Resalat Ave). Tehran Tehran Iran (Islamic Republic of)