Precise Transplantation of hAESCs Into the Ventricle for Parkinson's Disease.

NCT05435755 | Unknown Early Phase 1 DMC

• 1 other identifier: 2020YFA0112600
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the safety of multiple treatments of hAESCs treatments, while exploring the effectiveness of hAESCs treatments for PD.

Conditions

Parkinson's Disease

Keywords

Parkinson's Disease; stem cell

Outcome Measures

Primary Outcomes (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.

  12 Months

Secondary Outcomes (8)

• Changes in Unified Parkinson's Disease Rating Scale (UPDRS) in OFF state between the experimental group and the control group at 12 months after the first hAESCs treatment

  day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.

• Changes in the Hoehn and Yahr scale

  day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.

• Changes in the Schwab and England score

  day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.

• Changes in the Parkinson's Disease Questionnaire (PDQ-39)

  day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.

• Changes in the Hamilton Depression (HAMD) Scale

  day 0, 1 month, 2 month, 3 month, 6 month, 9 month and 12 month.

Study Arms (2)

hAESCs treatment

EXPERIMENTAL

A total of 6 times hAESCs transplants will be performed. 50 million (in 2ml) hAESCs will be transplanted into the ventricle of participants through the Ommaya sac (set as day 0 at the beginning of the trial). Subsequently, hAESCs ventricle transplants will be performed at 1 month ±5 days, 2 months ±5 days, 3 months ±5 days, 6 months ±5 days, and 9 months ±5 days after the first cell transplantation, with a volume of 50 million cells (in 2 ml) each time.

Biological: hAESCs treatment

Control group

PLACEBO COMPARATOR

Four times hAESCs transplants and two times placebo (cell preservation solution) injections will be performed by Ommaya sac. 50 million (in 2ml) hAESCs will be transplanted into the ventricle of participants (set as day 0 at the beginning of the trial). Subsequently, hAESCs will be transplanted into the ventricle for 3 times, respectively, at 1 month ±5 days , 2 months±5 days , and 3 months ±5 days after the first cell transplantation, with a volume of 50 million cells (in 2 ml) each time. Ventricle injection of 2 ml placebo (cell preservation solution) will be performed at 6 months ±5 days and 9 months ±5 days after the first cell transplantation.

Biological: placebo (saline)

Interventions

hAESCs treatment BIOLOGICAL

Precise Transplantation of hAESCs Into the Ventricle for Parkinson's Disease.

hAESCs treatment

placebo (saline) BIOLOGICAL

2 times placebo (normal saline) injections as control group

Control group

Eligibility Criteria

• Age: 30 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• years old, with more than 5 years primary Parkinson's disease history, male or female;
• The "off-age" UPDRS exercise score is between 38 and 70 points, two times before the operation, the "down period" UPDRS exercise score was stable, that is, (high value - low value) / high value ≤ 10%;
• The Parkinson treatment is stable for more than 3 months;
• Levodopa treatment was effective;
• No general anesthesia contraindications, no contraindications to stereotactic surgery and other conditions that interfere with clinical evaluation;
• No abnormalities affecting cell transplantation were found through the cranial MRI;
• Patients shall be properly cared during treatment, and caregivers can provide transplantation situations and assistance to the researcher if necessary.

You may not qualify if:

• Atypical Parkinson's disease, such as Parkinson's syndrome, secondary Parkinson's disease;
• Only having tremor syndrome;
• "off period" or "on period" has serious movement disorders and cannot complete any routine exercise tasks;
• Symptoms of severe neurological deficits caused by other diseases;
• Severe mental symptoms or dementia;
• Patients are unwilling to cooperate or incapable of completing self-evaluation, and cannot complete the evaluator with the help of a doctor;
• History of striatum or extrapyramidal surgery, including deep brain stimulation (DBS);
• Injecting apomorphine treatment;
• Abnormal blood coagulation or ongoing anticoagulation therapy;
• Women of childbearing age who do not take effective contraception;
• Pregnant or lactation;
• Patients who have participated in other clinical studies of drugs or medical devices within 3 months;
• Botox toxin, phenol, subarachnoid injection of baclofen or interventional therapy for the treatment of dystonia or spasticity within 6 months;
• History of seizures or prophylactic anti-epileptic drugs;
• Alcohol or drug abuse;

Sponsors & Collaborators

• Shanghai East Hospital: lead
• Shanghai iCELL Biotechnology Co., Ltd, Shanghai, China: collaborator

Study Sites (1)

Jingwen Wu

Shanghai, Shanghai Municipality, 200000, China

Location

MeSH Terms

Conditions

Parkinson Disease

Interventions

Sodium Chloride

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Officials

• Jingwen Wu, Dr

  Shanghai East Hospital, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jingwen Wu, Dr

CONTACT

18916111526; wujingwendongfang@163.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: The study plans to recruit 12 patients who meet the criteria. 50 millions hAESCs will be transplanted to participants with PD.

Study Record Dates

• First Submitted: March 15, 2022
• First Posted: June 28, 2022
• Study Start: December 2, 2022
• Primary Completion: July 30, 2023
• Study Completion: August 30, 2023
• Last Updated: October 28, 2022

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)