Parkinson's DiseasePhase 1/2NCT03119636
Safety and Efficacy Study of Human ESC-derived Neural Precursor Cells in the Treatment of Parkinson's Disease
A Phase 1/2 study testing NPC transplantation and Levodopa in people with Parkinson's Disease, run by Chinese Academy of Sciences at 1 site in China.
Trial summary
This study will evaluate the safety and efficacy of intracerebral transplantation of human embryonic stem cells-derived neural precursor cells in patients with Parkinson's Disease. Read more about this study
- Who is it for?
- Ages 50 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- NPC transplantation and Levodopa
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- May 2017to Dec 2020
- Phase
- Phase 1/2
- 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 50 to 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Primary Parkinson's disease patients，a history over 5 years，females or males;
- Cannot effectively control the PD or tolerate the side effects of drugs;
- Hoehn and Yahr Stage 3 or 4 in the off state at screening
- Age between 50 and 80 years;
- Dopamine is effective or once;
- Sign the informed consent
Who cannot join
- Atypical Parkinsonian syndrome or only having tremor syndrome;
- Having been done pallidotomy, DBS, striatum or extrapyramidal surgery;
- Subjects are using apomorphine or anticoagulant;
- Subjects used immunosuppressant or antipsychotic drugs in last 3 months;
- Subjects used botulinum toxin, phenol, or other drugs for the treatment of dystonia or muscle cramps in last 6 months;
- During the period of active epilepsy preventing epilepsy with antiepileptic;
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- Coagulant function abnormality or other obviously abnormal laboratory test results;
- Having skin basal cell carcinoma or cervical cancer and other pre-cancerous lesions;
- Subject has a history of chronic alcohol or drug abuse ;
- Pregnancy or lactation;
- Subjects participated in other clinical trials in recent 3 months;
- Subject is considered as dementia, a serious mental disorder (depression or mania), and personality or behavioral disorders through cognitive and behavioral test;
- Cannot cooperate on the research;
- Severe brain atrophy, or existing brain injury such as cerebral infarction, cerebral vascular malformation or trauma;
- Severe systemic diseases;
- Severe dyskinesia or frequent "OFF" or "ON" states
- Severe infectious diseases (eg HIV, HCV, HBV, syphilis positive)
- Not suitable to participate in this clinical trial assessed by other physicians
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
The first affiliated hospital of Zhengzhou university
Zhengzhou, Henan, 450052
About this study
Official title: A Phase I/II, Open-Label Study to Assess the Safety and Efficacy of Striatum Transplantation of Human Embryonic Stem Cells-derived Neural Precursor Cells in Patients With Parkinson's Disease
This study will evaluate the safety and efficacy of intracerebral transplantation of human embryonic stem cells-derived neural precursor cells in patients with Parkinson's Disease.
Read the detailed description
This study is a Phase I/II, open-label, non randomized clinical trial. The study will enroll 50 patients for cell injection, administering a single dose of neural precursor cells by stereotaxic intra-striatal injection.
- Conditions
- Parkinson's Disease
- Being tested
- NPC transplantation, Levodopa
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NPC transplantationBiological
The cells are stereotactically implanted in the striatum.
LevodopaDrug
Levodopa is used depending on the patient's condition
| Study group | Role | What they receive |
|---|---|---|
| NPC transplantation | Experimental | The patients will receive Levodopa combined with a regular neural precursor cell (NPC) transplantation Receives: Biological: NPC transplantation; Drug: Levodopa |
| HLA-matched NPC transplantation | Experimental | The patients will receive Levodopa combined with a HLA-matched neural precursor cell (NPC) transplantation Receives: Biological: NPC transplantation; Drug: Levodopa |
| HLA-non-matched NPC transplantation | Experimental | The patients will receive Levodopa combined with a HLA-non-matched neural precursor cell (NPC) transplantation Receives: Biological: NPC transplantation; Drug: Levodopa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (TEAEs), severe TEAEs as assessed by head MRI and blood examination
Number of subjects with adverse events such as the evidence of graft failure or rejection
Measured: 6 months
Secondary outcomes (3)
Change in Unified Parkinson's Disease Rating Scale (UPDRS) score from baseline
The total Unified Parkinson's Disease Rating Scale (UPDRS) score is derived from Part I (Mentation, Behavior and Mood), Part II (Activities of Daily Living) and Part III (Motor Examination). Part I assesses 4 functions; Part II assesses 13 activities of daily living; Part III assesses 14 motor symptoms. Each item is rated on a scale from 0 (normal) to 4 (severe). The minimum total score possible is 0 and the maximum total score possible is 176. Each subject was independently rated by two observers at each study visit and a mean score was calculated for analysis.
Measured: Baseline and 12 months
Change in DATscan from baseline
DATscan is an imaging technology that uses small amounts of a radioactive drug to help determine how much dopamine is available in a person's brain.Its principle is based on using of radiopharmaceutical, which bind to dopamine transporters (DAT).
Measured: Baseline and 12 months
Change in Hoehn and Yahr Stage from baseline
The Hoehn and Yahr scale is a commonly used system for describing how the symptoms of Parkinson's disease progress mainly by observing balance and walk.
Measured: Baseline and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2017
- Study startMay 2017 (estimated)
- Primary completionNov 2018 (estimated)
- Study completionDec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chinese Academy of Sciences
- Sponsor type
- Other government
- Responsible party
- Principal investigator, Qi Zhou, Chinese Academy of Sciences
- Investigators
- Zhou Qi, Doctor, Principal investigator, Institute of Zoology, Chinese Academy of Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03119636
- Sponsor's protocol ID
- ChineseASZQ-003
- Status last verified
- Sep 2016
- First submitted
- 6 Apr 2017
- Last updated
- 18 Apr 2017
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Wang YK, Zhu WW, Wu MH, Wu YH, Liu ZX, Liang LM, Sheng C, Hao J, Wang L, Li W, Zhou Q, Hu BY. Human Clinical-Grade Parthenogenetic ESC-Derived Dopaminergic Neurons Recover Locomotive Defects of Nonhuman Primate Models of Parkinson's Disease. Stem Cell Reports. 2018 Jul 10;11(1):171-182. doi: 10.1016/j.stemcr.2018.05.010. Epub 2018 Jun 14.
PubMed 29910127
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II, Open-Label Study to Assess the Safety and Efficacy of Striatum Transplantation of Human Embryonic Stem Cells-derived Neural Precursor Cells in Patients With Parkinson's Disease
- ClinicalTrials.gov ID
- NCT03119636
- Acronym / sponsor ID
- ChineseASZQ-003
- Conditions
- Parkinson's Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Chinese Academy of SciencesOther government
- Collaborators
- The First Affiliated Hospital of Zhengzhou University
- Study start
- May 2017 (estimated)
- Primary completion
- Nov 2018 (estimated)
- Study completion
- Dec 2020 (estimated)
- First posted
- 18 Apr 2017 (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.
This study will evaluate the safety and efficacy of intracerebral transplantation of human embryonic stem cells-derived neural precursor cells in patients with Parkinson's Disease.
Detailed description
This study is a Phase I/II, open-label, non randomized clinical trial. The study will enroll 50 patients for cell injection, administering a single dose of neural precursor cells by stereotaxic intra-striatal injection.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| NPC transplantation | Experimental | Biological: NPC transplantation, Drug: LevodopaThe patients will receive Levodopa combined with a regular neural precursor cell (NPC) transplantation |
| HLA-matched NPC transplantation | Experimental | Biological: NPC transplantation, Drug: LevodopaThe patients will receive Levodopa combined with a HLA-matched neural precursor cell (NPC) transplantation |
| HLA-non-matched NPC transplantation | Experimental | Biological: NPC transplantation, Drug: LevodopaThe patients will receive Levodopa combined with a HLA-non-matched neural precursor cell (NPC) transplantation |
Who may qualify
- Ages
- 50 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Primary Parkinson's disease patients，a history over 5 years，females or males;
- Cannot effectively control the PD or tolerate the side effects of drugs;
- Hoehn and Yahr Stage 3 or 4 in the off state at screening
- Age between 50 and 80 years;
- Dopamine is effective or once;
Key exclusion criteria (18 listed)
- Atypical Parkinsonian syndrome or only having tremor syndrome;
- Having been done pallidotomy, DBS, striatum or extrapyramidal surgery;
- Subjects are using apomorphine or anticoagulant;
- Subjects used immunosuppressant or antipsychotic drugs in last 3 months;
- Subjects used botulinum toxin, phenol, or other drugs for the treatment of dystonia or muscle cramps in last 6 months;
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 |
|---|---|---|
| NPC transplantationThe cells are stereotactically implanted in the striatum. | Biological | HLA-matched NPC transplantation, HLA-non-matched NPC transplantation, NPC transplantation |
| LevodopaLevodopa is used depending on the patient's condition | Drug | HLA-matched NPC transplantation, HLA-non-matched NPC transplantation, NPC transplantation |
Primary outcome
Incidence of treatment-emergent adverse events (TEAEs), severe TEAEs as assessed by head MRI and blood examination
Time frame: 6 months
Number of subjects with adverse events such as the evidence of graft failure or rejection
3 secondary outcomes
Change in Unified Parkinson's Disease Rating Scale (UPDRS) score from baseline
Time frame: Baseline and 12 months
Change in DATscan from baseline
Time frame: Baseline and 12 months
Change in Hoehn and Yahr Stage from baseline
Time frame: Baseline and 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2017.
- Derived from this study: PubMed 29910127Wang YK, Zhu WW, Wu MH, Wu YH, Liu ZX, Liang LM, Sheng C, Hao J, Wang L, Li W, Zhou Q, Hu BY. Human Clinical-Grade Parthenogenetic ESC-Derived Dopaminergic Neurons Recover Locomotive Defects of Nonhuman Primate Models of Parkinson's Disease. Stem Cell Reports. 2018 Jul 10;11(1):171-182. doi: 10.1016/j.stemcr.2018.05.010. Epub 2018 Jun 14.
- 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.
Zhou Qi, Doctor principal investigator
Institute of Zoology, Chinese Academy of Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing NPC transplantation and Levodopa in people with Parkinson's Disease, run by Chinese Academy of Sciences at 1 site 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 6 inclusion and 18 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 18 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Wang Liu, Doctor (+86-01064807858, wangliu@ioz.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (TEAEs), severe TEAEs as assessed by head MRI and blood examination (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03119636, which also lists 1 linked publication.
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 18 Apr 2017.