A Confirmatory Study of Allogeneic iPSC-derived Dopaminergic Progenitor Cells (Raguneprocel) in Patients with Parkinson's Disease (Post-marketing Clinical Study)
A Post-marketing Clinical Study of Raguneprocel in Patients with Parkinson's Disease
Yoshida Kenji
Sumitomo Pharma Co., Ltd.
6-8, Doshomachi 2-chome, Chuo-ku, Osaka, Osaka 541-0045, Japan
+81-120-034-389
cr@sumitomo-pharma.co.jp
Product information center
Sumitomo Pharma Co., Ltd.
6-8, Doshomachi 2-chome, Chuo-ku, Osaka, Osaka 541-0045, Japan
+81-120-034-389
cc@sumitomo-pharma.co.jp
Recruiting
July. 24, 2026
35
Interventional
single arm study
open(masking not used)
uncontrolled control
single assignment
treatment purpose
Participants are eligible to be included in the study only if all of the following criteria are met:
1. (Main cohort only) Age 18>= to <=65 years at the time of informed consent
(Sub-cohort only) Age > 65 years at the time of informed consent
2. The patient has a diagnosis of Parkinson's disease (clinically definite or probable) according to the MDS clinical diagnostic criteria for Parkinson's disease (2015) (whether sporadic or genetic Parkinson's disease).
3. The patient has a reduction pattern in the basal ganglia region that is characteristic in Parkinson's disease as indicated by dopamine transporter scan (DAT scan).
4. The patient has Parkinson's disease for at least 5 years at the time of informed consent.
5. The patient has motor symptoms of Parkinson's disease that are not adequately controlled with existing pharmacological therapy
6. The patient is in the ON and OFF states based on MDS-UPDRS Part 3 and/or symptom diary
7. The patient has H&Y scale at OFF state of Stage 2 or higher
8. The patient has H&Y scale at ON state of Stage 3 or lower
9. The patient has a levodopa response of >= 30% at the time of interrupting antiparkinsonian drugs (practically defined off).
10. The patient is able to keep a symptom diary throughout the study period, with the rate of keeping the symptom diary of >= 80% at the screening and baseline.
11. The patient is in good compliance with antiparkinsonian drugs and able to take antiparkinsonian drugs and tacrolimus as instructed throughout the study period.
12. The patient who, in the judgment of the investigators, is able to attend visits and be hospitalized at the study site, undergo specified tests and assessments, and comply with the visit schedule specified in the protocol throughout the study period.
13. Male participants:
- No contraceptive requirements
Female participants:
- Unable to become pregnant as in surgically sterile or postmenopausal.
OR
- Women of childbearing potential must have a negative urine beta-human chorionic gonadotropin (beta-hCG) pregnancy test at screening and baseline, and must be willing to continue their usual sexual abstinence or use effective methods of contraception from the time of signing the informed consent form (ICF) through the final visit.
14. The patient is capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Participants are excluded from the study if any of the following criteria apply:
1. The patient has a symptomatic organic lesion as detected by head MRI or magnetic resonance angiography (MRA).
2. The patient has abnormal immune function.
3. The patient has dementia, a Mini-Mental State Examination (MMSE) score of <=23, or an MMSE score of >= 24 but with suspected dementia. If the patient has an MMSE score of >= 24 and mild cognitive impairment, the patient may be enrolled if the investigators determine that there are no concerns regarding the patient's participation in this study.
4. The patient has persistent dyskinesia that interferes with daily activities with existing drug therapy.
5. The patient has a bleeding tendency or abnormal coagulation function.
6. The patient has any contraindications to tacrolimus or concomitant medications (eg, levodopa, carbidopa, MRI contrast medium) or who cannot discontinue the drugs contraindicated for co-administration.
7. The patient has hypersensitivity to raguneprocel, tacrolimus, concomitant medications (eg, levodopa, carbidopa, MRI contrast medium) or any of their ingredients.
8. The patient is unable to undergo necessary imaging tests such as MRI and positron emission tomography (PET) because of the implantation or placement of a metal medical device (excluding MRI-compatible products), claustrophobia, or other reasons.
9. The patient has allergy, hypersensitivity, or history of hypersensitivity to study related procedures (eg, anesthetics, antiseptics) or ingredients used in the manufacture of raguneprocel (bovine-derived ingredients, swine-derived ingredients, and Chinese hamster-derived ingredients). (However, anesthetics and antiseptics are excluded if alternative products are available.)
10. The patient is complicated with or has a history of any of the following:
- Malignant neoplasm (excluding those judged to have cured more than 5 years before informed consent)
- Epilepsy
- Mental disease (eg, depression, bipolar disorder, schizophrenia) or similar psychiatric symptoms
- Other serious complications (eg, cerebrovascular disorder, heart disease, chronic respiratory disease, poorly controlled hypertension, diabetes mellitus)
11. The patient is undergoing or have a history of stereotactic brain surgery (pallidotomy or thalamotomy), DBS, levodopa - carbidopa or foslevodopa - foscarbidopa continuous therapy using devices, or MR-guided focused ultrasound therapy at the time of informed consent
12. The patient has received cell therapy or gene therapy prior to informed consent.
13. The patient has received systemic treatment with immunosuppressive drugs or steroids within 3 months prior to informed consent.
14. The patient has undergone transplantation of allogeneic iPSC-derived dopaminergic progenitor cells before informed consent.
15. The patient has received another investigational drug (including a post-marketing study drug) or worn another investigational device (including a post-marketing study device) within 3 months before informed consent or has not completed another clinical study by the time of informed consent.
16. The patient is positive for hepatitis B surface (HBs) antigen or hepatitis B core (HBc) antibody.
17. The patient is positive for anti-human immunodeficiency virus (HIV) antibody.
18. The patient is positive for anti-human T-cell leukemia virus type 1 (HTLV-1) antibody.
19. The patient is positive for hepatitis C virus (HCV) RNA.
20. The patient has active infection including syphilis (non-treponemal lipid antibody [STS] and treponemal antibody [TPHA]) and Mycobacterium tuberculosis.
21. The patient has any of the following laboratory test results at screening:
- Neutrophils < 2,000/microL
- Platelets < 5.0 x 10^4/microL
- AST and ALT > 3.0 x upper limit of normal
- Total bilirubin > 1.5 x upper limit of normal
- eGFR < 60 mL/min/1.73 m^2
eGFR (mL/min/1.73 m^2) = 194 x Cr^(-1.094) x (age [years])^(-0.287) (x 0.739 for females)
22. The patient is lactating at the time of informed consent.
23. The patient is considered by the investigator to be inappropriate as a subject of this study.
18age old over
No limit
Both
Parkinson's disease
Transplant Raguneprocel in the bilateral putamen by stereotactic brain surgery, targeting at approximately 5.4 x 10^6 allogeneic iPSC-derived dopaminergic progenitor cells per side.
Change from baseline in the MDS-UPDRS Part 3 total score at OFF state (practically defined off) at Week 96 after Raguneprocel transplantation
Sumitomo Pharma Co.,Ltd.
Kyoto University Hospital Institutional Review Board
