RECRUITING

Clinical Study of Regulatory T Cells (Tregs) in the Treatment of Neurodegenerative Diseases

Conditions

Amyotrophic Lateral Sclerosis (ALS)Alzheimer's Disease(AD)Multiple System Atrophy, MSA Treg regulatory T cells neurodegenerative diseases Alzheimer's Disease Multiple System Atrophy Amyotrophic Lateral Sclerosis

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

An open, multi- center phase Ⅰ clinical study evaluating the safety and efficacy of autologous human polyclonal regulatory T cell injection (NP001 cell injection) in patients with Neurodegenerative diseases (ALS, MSA, AD).

Official Title

An Open, Single-Arm, Multi-Center Clinical Study to Evaluate the Safety and Tolerability of Regulatory T Cells (Tregs) in the Treatment of Neurodegenerative Diseases

Quick Facts

Study Start:2024-11

Study Completion:2027-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06671236

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.

Ages Eligible for Study:18 Years to 70 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Male or female patients aged 18 to 70 years; 2. According to current international diagnostic criteria: 1. ALS: defined by the Gold Coast Diagnostic Criteria (Shefner, 2020) as having a diagnosis of sporadic or familial amyotrophic lateral sclerosis (ALS), diagnosed as a probable, probable, or definite patient with laboratory support according to the World Federation of Neurology El Escorial criteria; 2. MSA: UMSARS I (Question 11 omitted) score of 5 -17 and able to walk independently (i.e., able to walk for at least 40 meters without the use of crutches or walkers and without other support (e.g., holding the arm or touching)). Expected survival of at least 3 years in the opinion of the investigator. Normal cognition as assessed by the Montreal Cognitive Assessment (MOCA), i.e., ≥26. 3. AD: age 65 or above; Clinical diagnosis of AD (MoCA 10-20 and Clinical Dementia Rating Scale/CDR = 1) on stable doses of medication for at least three months, no clinically significant findings on blood tests, able to voluntarily provide written informed consent. 3. Must have an autologous T cell source sufficient to make NP001 T cells, defined as a peripheral CD3+ T cell count of \> 800 cells/pl. or assay of available autologous Tregs with CD4, CD25, and FoxP3 expression greater than or equal to 50% by flow cytometry. 4. If there is a stable dose for more than one month prior to study entry. For example, patients with ALS can continue treatment with riluzole (Rilutek®) and/or edaravone (Radicava®); 5. Patients must have \> two weeks after the end of major surgery and after the completion of participation in other research trials; 6. Patients must have recovered from clinical toxicity (CTCAE \[5th Edition\] toxicity values have resolved to \< 2); 7. Serum creatinine less than or equal to 2.0 mg/dL; 8. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 3 x upper limit of normal; 9. Bilirubin \< 1.5 (except Gilbert's disease); 10. Lung slow vital capacity (SVC) \> 50% of predicted normal; 11. No history of abnormal bleeding tendency; 12. Informed consent must be obtained prior to performing any study-related procedures that are not part of standard medical care, with the understanding that the participant may withdraw from the study without influence for the future medical care.	1. uncontrolled infection; 2. \< 3 drugs do not adequately control hypertension; 3. Documented history of pulmonary embolism within 6 months of enrollment; 4. Clinically significant cardiology, defined as: myocardial infarction, NYHA-graded class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmia, or ECG evidence of acute ischemia or abnormal conduction system within 6 months prior to enrollment; 5. Patients with a history of coronary artery bypass grafting or angioplasty will be evaluated by cardiology and considered on a case-by-case basis; 6. Seropositive for HIV, hepatitis B or hepatitis C; 7. Pregnant or lactating patients; 8. Patients of childbearing potential or males with partners of childbearing potential who are unwilling to use contraception; 9. Participation in any other interventional study; 10. Treatment with another investigational drug, biologic, or device within 30 days or 5 half-lives (whichever is longer) of the screening period. Patient participation in observational/non-interventional clinical studies will be discussed with the Medical Monitor; 11. Prior treatment with ALS, MSA or AD gene or cell therapy; 12. History of clinically significant tumor, liver or kidney disease, or other uncontrolled disease; 13. presence of a feeding tube; 14. Current use of antipsychotics, antiepileptic drugs (except benzodiazepines, gabapentin, pre-Bahrain) or class 1 (e.g., flecainide) or class 3 (e.g., amiodarone) antiarrhythmic drugs; 15. Subjects who, in the opinion of the investigator, are at significant risk of suicide; 16. Other conditions that the investigator considers unsuitable for enrollment.

Inclusion Criteria

Exclusion Criteria

1. Male or female patients aged 18 to 70 years; 2. According to current international diagnostic criteria:
1. ALS: defined by the Gold Coast Diagnostic Criteria (Shefner, 2020) as having a diagnosis of sporadic or familial amyotrophic lateral sclerosis (ALS), diagnosed as a probable, probable, or definite patient with laboratory support according to the World Federation of Neurology El Escorial criteria;
2. MSA: UMSARS I (Question 11 omitted) score of 5 -17 and able to walk independently (i.e., able to walk for at least 40 meters without the use of crutches or walkers and without other support (e.g., holding the arm or touching)). Expected survival of at least 3 years in the opinion of the investigator. Normal cognition as assessed by the Montreal Cognitive Assessment (MOCA), i.e., ≥26.
3. AD: age 65 or above; Clinical diagnosis of AD (MoCA 10-20 and Clinical Dementia Rating Scale/CDR = 1) on stable doses of medication for at least three months, no clinically significant findings on blood tests, able to voluntarily provide written informed consent.
3. Must have an autologous T cell source sufficient to make NP001 T cells, defined as a peripheral CD3+ T cell count of \> 800 cells/pl. or assay of available autologous Tregs with CD4, CD25, and FoxP3 expression greater than or equal to 50% by flow cytometry.
4. If there is a stable dose for more than one month prior to study entry. For example, patients with ALS can continue treatment with riluzole (Rilutek®) and/or edaravone (Radicava®); 5. Patients must have \> two weeks after the end of major surgery and after the completion of participation in other research trials; 6. Patients must have recovered from clinical toxicity (CTCAE \[5th Edition\] toxicity values have resolved to \< 2); 7. Serum creatinine less than or equal to 2.0 mg/dL; 8. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 3 x upper limit of normal; 9. Bilirubin \< 1.5 (except Gilbert's disease); 10. Lung slow vital capacity (SVC) \> 50% of predicted normal; 11. No history of abnormal bleeding tendency; 12. Informed consent must be obtained prior to performing any study-related procedures that are not part of standard medical care, with the understanding that the participant may withdraw from the study without influence for the future medical care.

1. uncontrolled infection;
2. \< 3 drugs do not adequately control hypertension;
3. Documented history of pulmonary embolism within 6 months of enrollment;
4. Clinically significant cardiology, defined as: myocardial infarction, NYHA-graded class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmia, or ECG evidence of acute ischemia or abnormal conduction system within 6 months prior to enrollment;
5. Patients with a history of coronary artery bypass grafting or angioplasty will be evaluated by cardiology and considered on a case-by-case basis;
6. Seropositive for HIV, hepatitis B or hepatitis C;
7. Pregnant or lactating patients;
8. Patients of childbearing potential or males with partners of childbearing potential who are unwilling to use contraception;
9. Participation in any other interventional study;
10. Treatment with another investigational drug, biologic, or device within 30 days or 5 half-lives (whichever is longer) of the screening period. Patient participation in observational/non-interventional clinical studies will be discussed with the Medical Monitor;
11. Prior treatment with ALS, MSA or AD gene or cell therapy;
12. History of clinically significant tumor, liver or kidney disease, or other uncontrolled disease;
13. presence of a feeding tube;
14. Current use of antipsychotics, antiepileptic drugs (except benzodiazepines, gabapentin, pre-Bahrain) or class 1 (e.g., flecainide) or class 3 (e.g., amiodarone) antiarrhythmic drugs;
15. Subjects who, in the opinion of the investigator, are at significant risk of suicide;
16. Other conditions that the investigator considers unsuitable for enrollment.

Contacts and Locations

Study Contact

Mingqi Lu, MD., PhD

CONTACT

609-716-7786; +86 15800843507

lumingqi@novabiotx.com

Study Locations (Sites)

Ascension Via Christi Clinic

Wichita, Kansas, 67230

United States

Collaborators and Investigators

Sponsor: Novabio Therapeutics

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 Registration Dates

Study Start Date2024-11

Study Completion Date2027-12

Study Record Updates

Study Start Date2024-11

Study Completion Date2027-12

Terms related to this study

Keywords Provided by Researchers

Treg
regulatory T cells
neurodegenerative diseases
Alzheimer's Disease
Multiple System Atrophy
Amyotrophic Lateral Sclerosis

Additional Relevant MeSH Terms

Amyotrophic Lateral Sclerosis (ALS)
Alzheimer's Disease(AD)
Multiple System Atrophy, MSA