An Exploratory Clinical Study of UX-DA001 in Subjects With Idiopathic Parkinson's Disease

NCT06778265 | Enrolling By Invitation Phase 1

• 1 other identifier: UX-DA001-101
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

This clinical study is designed to explore the safety and tolerability of UX-DA001. It will also explore if UX-DA001 works to improve motor function in subjects with Parkinson's disease. UX-DA001 manufactured from participant's own cells will differentiate into mature dopaminergic neurons after being transplanted into the brain of the participant.

Conditions

Parkinson Disease, Idiopathic

Keywords

Idiopathic Parkinson´s Disease; autologous iPSC derived mDAPs; Midbrain Dopaminergic Progenitors; Induced pluripotent stem cells; mDAPs; stem cells; iPSC derived product

Outcome Measures

Primary Outcomes (2)

• The incidence and and severity of adverse events (AEs) associated with surgery and/or investigational product

  The incidence and and severity of adverse events (AEs) associated with surgery and/or investigational product during the surgical treatment period and the 4-week postoperative observation period. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.

  within 4 weeks post surgery

• The incidence and severity of AEs/serious adverse events (SAEs)

  The incidence and severity of AEs/serious adverse events (SAEs) during the study, including AEs and SAEs during the surgery treatment period, postoperative observation period, and follow-up period. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.

  From baseline to 2 years post surgery

Secondary Outcomes (7)

• 18F-FP-CIT uptake using positron emission tomography (PET)

  From baseline to 2 years post surgery

• The situation of implantation and overgrowth of transplanted cells using cranial MRI

  From baseline to 2 years post surgery

• Changes in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score, part III, from baseline.

  From baseline to 2 years post surgery

• Changes in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score, part II, from baseline.

  From baseline to 2 years post surgery

• Changes in modified modified Hoehn-Yahr (H&Y) scale from baseline

  From baseline to 2 years post surgery

Study Arms (1)

UX-DA001

EXPERIMENTAL

Biological: UX-DA001

Interventions

UX-DA001 BIOLOGICAL

UX-DA001 (Human Midbrain Dopaminergic Progenitor Cells) is used for treating patients with iPD via implanting into bilateral putamina under stereotactic neurosurgery. Two dose levels will be planned. Each patient only receives one corresponding dose of UX-DA001.

UX-DA001

Eligibility Criteria

• Age: 50 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The subjects or their legally acceptable representative understand and comply with the study procedures, agree to participate in the clinical trial, and sign the ICF;
• Aged between 50-75 years old, male or female;
• Subjects diagnosed with idiopathic PD, with a medical history of 5-20 years;
• Having received standard anti-PD treatment and been given optimal anti-PD treatment under the guidance of the investigator, but the efficacy has significantly declined;
• Good response to levodopa medications; the LCT shows that the maximum improvement rate of the UPDRS part III score exceeds 30%;
• The modified H\&Y scale of clinical "OFF" period is ≥ Stage 3 and ≤ Stage 4;
• Taking a stable dosage of anti-PD medications for at least 4 weeks;
• Good physical condition or stable concomitant diseases;
• With reliable caregivers who can cooperate to complete the assessment items;
• Subjects with good compliance.

You may not qualify if:

• PD Subjects in whom previous genetic testing has found a GBA gene mutation or PD Subjects whom the investigator considers unsuitable for participation in this clinical study due to other gene mutations;
• Subjects with the atypical Parkinson's syndrome or secondary Parkinson's syndrome;
• Subjects with HIV, HBV, HCV, treponema pallidum (TP) infection, or other active infections;
• Subjects infected with HTLV, EBV or CMV whose infection renders their blood cells unsuitable for cell product preparation;
• Subjects with a known hereditary disorder;
• Subjects with any history of malignancy;
• Subjects with other serious systemic diseases or functional disorders;
• Accompanied by other serious central nervous system diseases or serious cognitive and mental disorders;
• Subjects who are currently receiving or have previously received cell therapy or other medicine effecting safety and efficacy assessement;
• Subjects whose prior head CT/MRI examinations indicate brain injury, or Subjects with imaging abnormalities in the striatum and other brain regions leading to a significant increase in surgical risk, or Subjects who have previously undergone brain surgery;
• Subjects with clinically significant abnormal results in coagulation function, or Subjects who have been using anticoagulants for a long time and cannot discontinue use;
• Subjects with a history of severe allergy or hypersensitivity reactions, or a known history of hypersensitivity, or a history of intolerance to the investigational cellular drug or its excipients;
• Subjects who have undergone other surgeries within the past six months that the investigator deems may affect this trial, or Subjects who cannot tolerate general anesthesia or stereotactic surgery;
• Subjects with contraindications to MRI and PET scans;
• Subjects with a history of alcoholism or drug abuse;

Sponsors & Collaborators

• Shanghai UniXell Biotechnology Co., Ltd: lead
• Ruijin Hospital: collaborator

Study Sites (1)

Ruijin Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, 200025, China

Location

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

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

Study Officials

• Jun Liu, MD, PhD

  Ruijin Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: open-label, multi-center, dose-escalation and dose-expansion

Study Record Dates

• First Submitted: December 30, 2024
• First Posted: January 16, 2025
• Study Start: March 1, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: January 12, 2026

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)