Parkinson's DiseasehiPSC-DAPNCT06145711
A Clinical Trial of Parkinson's Disease Treatment by HiPSCs Derived Dopaminergic Neural Precursor Cells
A study testing hiPSC-DAP in people with Parkinson's Disease, run by Shanghai East Hospital at 2 sites in China.
Trial summary
XellSmart Biomedical (Suzhou) Co., Ltd. has closely collaborated with Shanghai East Hospital(East Hospital Affiliated To Tongji University) , using iPSC seed cells, on the development and establishment of a dopaminergic neural precursor cell production system. Read more about this study
- Who is it for?
- Ages 30 to 70 years
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- hiPSC-DAP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 2 sitesChina
- Study dates
- 23 Nov 2023to 22 Dec 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 30 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- The patient or their legally authorized guardian consents to participate in this study and signs the Informed Consent Form (ICF) in writing.
- Patients with primary Parkinson's disease, aged between 30 and 70 years, of any gender, with a disease duration of more than 5 years.
- Hohen-Yahr stage for Parkinson's disease (Appendix V) is between stage 3 and stage 4. Patients with a Hohen-Yahr stage below 3 who refuse other treatments may also be included.
- The MDS-UPDRS-III score for "OFF" periods in Parkinson's disease is greater than 38 points, and the MDS-UPDRS-III score for the two "OFF" periods before surgery is stable, i.e., (high value - low value) / high value is less than or equal to 10%.
- Patients who have stably received anti-Parkinson drug therapy for more than 3 months.
- Previous effective treatment with levodopa, followed by significant drug resistance and wearing-off effect.
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- The patient who is in stable condition, well-controlled complications, no contraindications for general anesthesia, no contraindications for stereotactic surgery, and no other conditions that interfere with clinical assessment.
- Head MRI does not reveal structural abnormalities that would affect stem cell transplantation, such as severe brain atrophy or space-occupying lesions.
- Subjects have a caregiver and are able to provide information about their condition before and after stem cell transplantation and can assist the investigator if necessary.
Who cannot join
- Patients with atypical Parkinson's disease, such as Parkinson's syndrome or secondary Parkinson's disease.
- Patients with Parkinson's disease with only tremors.
- Patients with severe motor impairment preventing the completion of routine motor tasks.
- Patients with severe neurological deficits caused by other diseases.
- Patients with severe psychiatric symptoms or dementia.
- The Patient who is unwilling or unable to cooperate, incapable of self-assessment, and unable to complete assessments even with the help of a physician.
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- Patients who have history of pallidotomy or thalamotomy or deep brain stimulation (DBS) surgery.
- Patients with apomorphine treatment currently .
- Coagulation abnormalities (prothrombin time (PT) or international normalized ratio (INR) > 1.5×ULN; activated partial thromboplastin time (APTT) > 1.5×ULN) or receiving anticoagulation therapy.
- Pregnant or lactating female patients and male and female patients who cannot use effective contraception within 1 year after the last study drug use.
- Patients who have participated in other drug or medical device clinical studies within the last 3 months.
- Patients who have used botulinum toxin, phenol, subarachnoid injection of baclofen, or intervention therapy for treating muscle tone disorders or spasticity within the last 6 months.
- Patients who have history of seizures or prophylactic use of antiepileptic drugs.
- Patients who have contraindications for general anesthesia or stereotactic surgery, such as sleep apnea or chronic obstructive pulmonary disease, etc.
- Patients with any other unstable systemic diseases, including active infections, poorly controlled hypertension (systolic blood pressure >160 mmHg and/or diastolic blood pressure >100 mmHg after treatment), unstable angina, congestive heart failure (New York Heart Association NYHA class II or above), or metabolic diseases, etc.
- Patients with alcohol or drug abuse.
- Patients who have used a high dose of benzodiazepines within the last 3 months.
- Patients with severe cognitive impairment, depression, or behavioral disorders, defined as a Mini-Mental State Examination (MMSE) score less than 26 and a Hamilton Depression Rating Scale (HAMD) score greater than 35.
- History of any malignant tumors.
- Patients with hepatitis A, active hepatitis B (HBsAg positive with HBV DNA higher than the upper limit of normal, and excluding hepatitis caused by drugs or other reasons), active hepatitis C (anti-HCV antibody positive with HCV RNA higher than the upper limit of normal), hepatitis E, human immunodeficiency virus (HIV) antibody positive, or syphilis (TP antibody positive).
- Patients with abnormal liver and kidney function laboratory tests during the screening period, such as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3×ULN, serum creatinine >1.5×ULN, or total bilirubin >1.5×ULN.
- Other situations in which the investigator believes that the subject is not suitable for inclusion or cannot tolerate stem cell transplantation surgery.
- PD subjects found to have gene mutations by genetic testing.
Trial locations and contacts
2 sites in 1 country.
China2 sites, 2 recruiting
- Recruiting
Shanghai East Hospital
Shanghai, Shanghai Municipality, 200120
wu jingwen Shanghai East Hospital, +13810835976, songjiwen001@qq.com
- Not yet recruiting
Shanghai East Hospital
Shanghai, Shanghai Municipality
Jingwen Wu, 13585517271, wujingwendongfang@163.com
About this study
Official title: A Clinical Trial of Parkinson's Disease Treatment by Human-induced Pluripotent Stem Cells (hiPSCs) Derived Dopaminergic Neural Precursor Cells
XellSmart Biomedical (Suzhou) Co., Ltd. has closely collaborated with Shanghai East Hospital(East Hospital Affiliated To Tongji University) , using iPSC seed cells, on the development and establishment of a dopaminergic neural precursor cell production system.
This study is conducted under collaboration between Shanghai East Hospital and XellSmart Biomedical (Suzhou) Co., Ltd. It is a distinctive stem cell therapy research targeting Parkinson's disease patients, characterized by the following:
Read more
- The first instance in China of using autologous iPSC-derived subtype-specific dopaminergic neural precursor cells for alternative transplantation therapy in Parkinson's disease.
- Before the initiation of this study, there was only one reported case around the world involving the use of laboratory-grade autologous iPSC-induced dopaminergic neural precursor cells for Parkinson's disease treatment in an American Caucasian individual. Positive preliminary clinical research results were obtained and published in the New England Journal of Medicine in 2020. Since then, no similar studies have been reported.
It has great significance to conduct this research. It will fill two critical clinical research gaps in stem cell therapy for Parkinson's disease in the world:
- The use of clinical-grade autologous iPSCs differentiated into dopaminergic neural cells, and treat Parkinson's disease.
- Evaluating the safety of using autologous iPSC-differentiated dopaminergic neural cells for Parkinson's disease treatment in individuals of Asian descent or Chinese ethnicity.
Read the detailed description
This study is a non-randomized, open-label, investigator-initiated interventional clinical trial aimed at exploring the safety of stem cell investigational drugs. The study plans to recruit 3 eligible PD subjects and prepare dopaminergic neural precursor cells from autologous iPSCs in advance. The PD subjects will undergo general anesthesia and, with the assistance of stereotactic technology, receive an injection of autologous iPSC-derived dopaminergic neural precursor cells into the putamen of striatum on one side, transplanted evenly at a linear distance of 1 centimeter. A Sofia drug pouch will also be installed to regularly monitor changes in neurotransmitter metabolism, including dopamine, in the brain.
Following the surgery, there will be regular observations for 6 months, during which the safety of this treatment modality will be examined and analyzed. If the test results within the first 6 months post-surgery indicate both good safety and some level of efficacy, the trial will continue for an additional 6 months. However, if the test results within the first 6 months post-surgery demonstrate good safety (i.e., no adverse events related to stem cell transplantation of grade 3 or higher occurring within 28 days after stereotactic transplantation of dopaminergic neural precursor cells) but insufficient efficacy (evaluated based on subject self-assessment, clinical symptoms, rating scales, PET/MR, changes in the dosage of levodopa, and other medications), autologous iPSC-derived dopaminergic neural precursor cells will be injected into the putamen of striatum on the other side (using the same method). Subsequently, the subjects will be regularly observed for an additional 18 months.
Postoperative observation and follow-up for unilateral stem cell transplantation will be conducted for 12 months, while bilateral stem cell transplantation will be observed and followed for 24 months (with the first postoperative observation and follow-up at 6 months and the second at 18 months). During the study, there will be periodic reviews and summaries every 6 months, generating interim clinical reports to optimize and guide the next stage of data collection and research.
- Conditions
- Parkinson's Disease
- Being tested
- hiPSC-DAP
- Keywords
- Parkinson's disease;stem cell
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hiPSC-DAPOther
Using hiPSC-DAP for the treatment of the patients of Parkinson's diease
| Study group | Role | What they receive |
|---|---|---|
| to use cell | Experimental | Not described Receives: Other: hiPSC-DAP |
What is being measured
The primary outcome is the main question the study is designed to answer.
1.Number of participants with adverse event (AE), serious adverse event(SAE) and adverse events of special interest(AESI)
AE occurring throughout the study period will be evaluated using the CTCAE V5.0 standard. AE/SAE and AESI were evaluated after Ommaya sac installation by laboratory examination, measurement of vital signs, physical examination, and subjects' symptoms to detect new abnormalities and/or deterioration of previous conditions and evaluate the safety of the study.
AESI is defined as intracranial infection, hemorrhage, rejection, and edema within 7 days after Ommaya sac installation, as well as acute allergic reactions and ectopic mass formation associated with study therapy.
[Time Frame: 18 Months]
Measured: 18 Months
Secondary outcomes (6)
Changes in Unified Parkinson's Disease Rating Scale (UPDRS) in OFF state between the experimental group and the control group at 18 months after the first hiPSCs-DAP treatment
UPDRS is used for evaluating the impairment and disability associated with Parkinson's Disease (PD). It consists of four sections: (1) Mentation, behavior, and mood; (2) Activities of daily living (ADLs); (3) Motor; and (4) Complications.
The UPDRS score ranges from 0 to 199, with higher score indicating greater disability [Time Frame: day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month.]
Measured: day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month
Changes in the Hoehn and Yahr scale
Hoehn and Yahr scale is used to provide a general estimate of clinical function of PD patients, combining functional deficits (disability) and objective signs (impairment). The Hoehn and Yahr score ranges from 0 to 5, with higher score indicating higher dysfunction of PD patients
Measured: day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month
Changes in the Schwab and England score
The Schwab and England score develop a scale that describes the capacity of daily living shown by a PD patients. It measures the following three areas: dependence, abilities, and awareness. The Schwab and England score range from o% to 100%, with higher scores indicating greater healthy status.
Measured: day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month
Changes in the Parkinson's Disease Questionnaire (PDQ-39)
PDQ-39 provides the evidence of the quality of life of a PD patient. The higher the score, the lower the quality of life of PD patients.
Measured: day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month
Hamilton Depression Rating Scale (HAM-D)
HAMD is the most widely used scale in clinical evaluation of depression in PD patients. This project takes 8 points as the critical value of depression, and the total score over 35 points is considered as severe depression, more than 20 points is likely to be diagnosed with depression, less than 8 points is not depressed.
Measured: day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month
Cranial dopamin transporter measured by positron emission tomography (PET)- magnetic resonance imaging (MRI)
Changes of cranial expression of dopamine transporter investigated by PET-M
Measured: [Time Frame: 3 month, 6 month, 18 month]
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2023
- Study start23 Nov 2023
- Primary completion22 Dec 2025 (estimated)
- Study completion22 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai East Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06145711
- Sponsor's protocol ID
- XS2022001
- Acronym
- hiPSC-DAP
- Status last verified
- Oct 2024
- First submitted
- 13 Nov 2023
- Last updated
- 15 Oct 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Trial of Parkinson's Disease Treatment by Human-induced Pluripotent Stem Cells (hiPSCs) Derived Dopaminergic Neural Precursor Cells
- ClinicalTrials.gov ID
- NCT06145711
- Acronym / sponsor ID
- hiPSC-DAP / XS2022001
- Conditions
- Parkinson's Disease
- Keywords
- Parkinson's disease;stem cell
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- Shanghai East HospitalUniversity, hospital or other
- Study start
- 23 Nov 2023 (actual)
- Primary completion
- 22 Dec 2025 (estimated)
- Study completion
- 22 Dec 2025 (estimated)
- First posted
- 24 Nov 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
XellSmart Biomedical (Suzhou) Co., Ltd. has closely collaborated with Shanghai East Hospital(East Hospital Affiliated To Tongji University) , using iPSC seed cells, on the development and establishment of a dopaminergic neural precursor cell production system.
This study is conducted under collaboration between Shanghai East Hospital and XellSmart Biomedical (Suzhou) Co., Ltd. It is a distinctive stem cell therapy research targeting Parkinson's disease patients, characterized by the following:
- The first instance in China of using autologous iPSC-derived subtype-specific dopaminergic neural precursor cells for alternative transplantation therapy in Parkinson's disease.
- Before the initiation of this study, there was only one reported case around the world involving the use of laboratory-grade autologous iPSC-induced dopaminergic neural precursor cells for Parkinson's disease treatment in an American Caucasian individual. Positive preliminary clinical research results were obtained and published in the New England Journal of Medicine in 2020. Since then, no similar studies have been reported.
It has great significance to conduct this research. It will fill two critical clinical research gaps in stem cell therapy for Parkinson's disease in the world:
- The use of clinical-grade autologous iPSCs differentiated into dopaminergic neural cells, and treat Parkinson's disease.
- Evaluating the safety of using autologous iPSC-differentiated dopaminergic neural cells for Parkinson's disease treatment in individuals of Asian descent or Chinese ethnicity.
Detailed description
This study is a non-randomized, open-label, investigator-initiated interventional clinical trial aimed at exploring the safety of stem cell investigational drugs. The study plans to recruit 3 eligible PD subjects and prepare dopaminergic neural precursor cells from autologous iPSCs in advance. The PD subjects will undergo general anesthesia and, with the assistance of stereotactic technology, receive an injection of autologous iPSC-derived dopaminergic neural precursor cells into the putamen of striatum on one side, transplanted evenly at a linear distance of 1 centimeter. A Sofia drug pouch will also be installed to regularly monitor changes in neurotransmitter metabolism, including dopamine, in the brain.
Following the surgery, there will be regular observations for 6 months, during which the safety of this treatment modality will be examined and analyzed. If the test results within the first 6 months post-surgery indicate both good safety and some level of efficacy, the trial will continue for an additional 6 months. However, if the test results within the first 6 months post-surgery demonstrate good safety (i.e., no adverse events related to stem cell transplantation of grade 3 or higher occurring within 28 days after stereotactic transplantation of dopaminergic neural precursor cells) but insufficient efficacy (evaluated based on subject self-assessment, clinical symptoms, rating scales, PET/MR, changes in the dosage of levodopa, and other medications), autologous iPSC-derived dopaminergic neural precursor cells will be injected into the putamen of striatum on the other side (using the same method). Subsequently, the subjects will be regularly observed for an additional 18 months.
Postoperative observation and follow-up for unilateral stem cell transplantation will be conducted for 12 months, while bilateral stem cell transplantation will be observed and followed for 24 months (with the first postoperative observation and follow-up at 6 months and the second at 18 months). During the study, there will be periodic reviews and summaries every 6 months, generating interim clinical reports to optimize and guide the next stage of data collection and research.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| to use cell | Experimental | Other: hiPSC-DAP |
Who may qualify
- Ages
- 30 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- The patient or their legally authorized guardian consents to participate in this study and signs the Informed Consent Form (ICF) in writing.
- Patients with primary Parkinson's disease, aged between 30 and 70 years, of any gender, with a disease duration of more than 5 years.
- Hohen-Yahr stage for Parkinson's disease (Appendix V) is between stage 3 and stage 4. Patients with a Hohen-Yahr stage below 3 who refuse other treatments may also be included.
- The MDS-UPDRS-III score for "OFF" periods in Parkinson's disease is greater than 38 points, and the MDS-UPDRS-III score for the two "OFF" periods before surgery is stable, i.e., (high value - low value) / high value is less than or equal to 10%.
- Patients who have stably received anti-Parkinson drug therapy for more than 3 months.
Key exclusion criteria (23 listed)
- Patients with atypical Parkinson's disease, such as Parkinson's syndrome or secondary Parkinson's disease.
- Patients with Parkinson's disease with only tremors.
- Patients with severe motor impairment preventing the completion of routine motor tasks.
- Patients with severe neurological deficits caused by other diseases.
- Patients with severe psychiatric symptoms or dementia.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| hiPSC-DAPUsing hiPSC-DAP for the treatment of the patients of Parkinson's diease | Other | to use cell |
Primary outcome
1.Number of participants with adverse event (AE), serious adverse event(SAE) and adverse events of special interest(AESI)
Time frame: 18 Months
AE occurring throughout the study period will be evaluated using the CTCAE V5.0 standard. AE/SAE and AESI were evaluated after Ommaya sac installation by laboratory examination, measurement of vital signs, physical examination, and subjects' symptoms to detect new abnormalities and/or deterioration of previous conditions and evaluate the safety of the study. AESI is defined as intracranial infection, hemorrhage, rejection, and edema within 7 days after Ommaya sac installation, as well as acute allergic reactions and ectopic mass formation associated with study therapy. [Time Frame: 18 Months]
6 secondary outcomes
Changes in Unified Parkinson's Disease Rating Scale (UPDRS) in OFF state between the experimental group and the control group at 18 months after the first hiPSCs-DAP treatment
Time frame: day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month
Changes in the Hoehn and Yahr scale
Time frame: day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month
Changes in the Schwab and England score
Time frame: day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month
Changes in the Parkinson's Disease Questionnaire (PDQ-39)
Time frame: day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month
Hamilton Depression Rating Scale (HAM-D)
Time frame: day 0, 1 month, 3 month, 6 month, 9 month,12 month,15 month and 18 month
Cranial dopamin transporter measured by positron emission tomography (PET)- magnetic resonance imaging (MRI)
Time frame: [Time Frame: 3 month, 6 month, 18 month]
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2024.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing hiPSC-DAP in people with Parkinson's Disease, run by Shanghai East Hospital at 2 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 15 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact WU jingwen Shanghai East Hospital (+13810835976, songjiwen001@qq.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
1.Number of participants with adverse event (AE), serious adverse event(SAE) and adverse events of special interest(AESI) (time frame: 18 Months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2024.