The Safety, Tolerability and Efficacy of NouvNeu001 for Parkinson's Disease

October 30, 2024 updated by: iRegene Therapeutics Co., Ltd.

A Phase I/II Study to Assess the Safety, Tolerability and Efficacy of NouvNeu001 Injection for Mid- to Late-stage Parkinson's Disease

This clinical trial is designed to evaluate the safety, tolerability and preliminary efficacy of a single injection of NouvNeu001 (Human Dopaminergic Progenitor Cells Injection) in patients with Parkinson's disease.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This is a multi-center, single arm, and open label trial. The NouvNeu001 will be transplanted into bilateral putamen/striatum using stereotactic neurosurgery. Subjects will take immunosuppressants to prevent potential immune rejection for 24 to 36 weeks.

Study Type

Interventional

Enrollment (Estimated)

40

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
    • Beijing
      • Beijing, Beijing, China, 100730
    • Hubei
      • Wuhan, Hubei, China, 430071
        • Not yet recruiting
        • Zhongnan Hospital of Wuhan University
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Age 50-75 years old, male or female
  • Able to understand the rationale of the clinical trial and sign informed consent form (ICF)
  • Diagnosis of Parkinson's Disease in accordance with the MDS clinical diagnostic criteria for Parkinson's disease (2015)
  • Diagnosis of Parkinson's Disease made between 4 to 20 years ago
  • Medically suitable for neurosurgery under anesthesia and able to participate in Magnetic Resonance Imaging (MRI) and Positron Emission Tomography (PET) scan.
  • Hoehn-Yahr staging for "off" episodes is 2.5 to 4
  • The MDS-UPDRS Part III (MDS-UPDRS-III) score in the "off" state > 35, and positive for the Acute Levodopa Challenge Test (ALCT)
  • Acceptable laboratory test results during screening and prior to transplantation

Exclusion Criteria:

  • Atypical Parkinsonism
  • Patients who have had previous pallidotomy, deep brain stimulation (DBS) surgery, striatal or extrapyramidal surgery, brain stereotaxy or other brain surgery; as well as other surgical procedures that are judged by the investigator to affect patient's participation in this study
  • Patients who have a previous head CT/MRI examination showing cerebral trauma, vascular malformation, hydrocephalus, brain tumor, etc., and patients who have brain imaging abnormalities in the striatum or other brain areas leading to a significantly increased risk for surgery.
  • Patients with a history of severe cardiovascular and cerebrovascular diseases
  • Patients with a history of malignant tumors
  • Patients who have had previous cellular therapy
  • Patients with active disseminated intravascular coagulation and significant hemorrhagic tendency within 3 months prior to screening, or who cannot temporarily suspend anti-platelet agents or other anti-coagulant medications for at least 5 days before surgery
  • Patients with long-term, heavy use of glucocorticoids or immunosuppressive drugs within 3 months prior to signing the ICF
  • Patients with a history of mental illness who are deemed unfit to participate in the study by the investigator; or a history of suicidal ideation or suicide attempts within the past year or currently;
  • Patients who have used botulinum toxin within 6 months prior to signing the ICF
  • Patients with active epilepsy or currently on anti-epileptic drugs
  • Patients with a history of dementia or severe cognitive disorder, or the score of MDS-UPDRS 1.1 during screening is > 3; poor compliance, inability to accurately keep diary, and/or inability to sign ICF due to dementia
  • Patients with severe depression or with severe anxiety during screening;
  • Patients with the following abnormalities during screening, including: Abnormal coagulation; Abnormal immunological tests, and assessed by the investigator it is not suitable to participate in the trial; Hypertensive patients with poorly controlled blood pressure and patients with severe postural hypotension; Diabetic patients with poorly controlled blood glucose
  • Patients with surgical contraindications, or with other surgical procedures within 6 months before screening which, in the opinion of the investigator, have an impact on this trial; Patients with other neurosurgical contraindications
  • Patients with other combined severe systemic diseases, such as pulmonary heart disease, moderate to severe asthma, severe chronic obstructive pulmonary disease (COPD)
  • Presence of one of the following: positive for human immunodeficiency virus (HIV) antibody, treponema pallidum antibody, hepatitis C virus (HCV) antibody and HCV RNA; Hepatitis B virus (HBV) surface antigen positive and HBV DNA copy number > detection of normal values; Tuberculosis is in the active stage; Other active infections that the investigator believes may affect Patients' participation in the study or affect study outcomes;
  • Patients with alcohol addiction or positive for drug of abuse testing
  • Patients with a history of contraindication or allergy to the drugs used during the study or any of its components, or are allergic to the same drugs or other macrolides, or have allergies;
  • Female of childbearing potential who are not surgically sterilized/premenopausal/unwilling to use medically approved effective contraception with 2 years after administration of investigational drug and lactating women; men who are not surgically sterilized/unwilling to use medically approved effective contraception with 2 years after administration of investigational drug
  • Patients who have received electric shock therapy within 30 days prior to surgery
  • Patients who are participating in other clinical trials, or have been enrolled in other clinical studies and received intervention therapy within 3 months prior to the surgery
  • Patients with poor compliance based on clinical evaluation of the investigator
  • Patients who are being treated with drugs such as apomorphine, or levodopa/carbidopa infusion therapy
  • Patients with severe dyskinesia in both on- and off-drug states

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: NouvNeu001
Biological: Human Dopaminergic Progenitor Cells
Single injection of Human Dopaminergic Progenitor Cells into the putamen/striatum region of brain.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Motor Function
Time Frame: 24 weeks post-transplant
Changes in Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part Ⅲ score from baseline to 24 weeks post-transplant. MDS-UPDRS Part III assesses the motor signs of PD, a higher score indicates more severe symptoms of PD.
24 weeks post-transplant
Safety and Tolerability
Time Frame: 24 weeks and 48 weeks post-transplant
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs).
24 weeks and 48 weeks post-transplant

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Continued Safety and Tolerability
Time Frame: 96 weeks and 5 years post-transplant
Incidence and severity of AEs and SAEs within 96 weeks and 5 years post-transplant.
96 weeks and 5 years post-transplant
The Motor Function and Non-motor Function
Time Frame: 48 weeks and 96 weeks post-transplant
Changes in MDS-UPDRS Part I-IV Score from baseline to 48 weeks and 96 weeks post-transplant. A higher score indicates more severe symptoms or worse health.
48 weeks and 96 weeks post-transplant

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

iRegene Therapeutics Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 17, 2024

Primary Completion (Estimated)

December 1, 2025

Study Completion (Estimated)

July 1, 2029

Study Registration Dates

First Submitted

November 24, 2023

First Submitted That Met QC Criteria

December 4, 2023

First Posted (Actual)

December 12, 2023

Study Record Updates

Last Update Posted (Estimated)

November 1, 2024

Last Update Submitted That Met QC Criteria

October 30, 2024

Last Verified

July 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NouvNeu001-02

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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