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

NCT06671236 | Recruiting Phase 1 DMC

• 1 other identifier: NP001-NDD02
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Keywords

Treg; regulatory T cells; neurodegenerative diseases; Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (2)

• Incidence of Treatment Related adverse events (AEs)

  Incidence of Treatment Related AEs, AEs of special interest and serious adverse events (SAEs)

  6 months

• Identification of Maximum Tolerated Dose (MTD)

  Incidence of dose-limiting toxicities (DLTs)

  28 days

Secondary Outcomes (2)

• Amyotrophic lateral sclerosis functional rating scale- revised (ALSFRS-R)

  1 year

• Rasch Overall ALS Disability Scale (ROADS)

  1 year

Study Arms (1)

Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection)

EXPERIMENTAL

Regulatory T cell therapy, intrathecal injection

Biological: Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection)

Interventions

Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection) BIOLOGICAL

Regulatory T cell therapy, intrathecal injection

Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection)

Eligibility Criteria

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

You may qualify if:

• Patients must meet all of the following criteria to be eligible for enrollment in this study:
• Male or female patients aged 18 to 70 years;
• According to current international diagnostic criteria:
• 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;
• 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®);
• Patients must have \> two weeks after the end of major surgery and after the completion of participation in other research trials;
• Patients must have recovered from clinical toxicity (CTCAE \[5th Edition\] toxicity values have resolved to \< 2);
• Serum creatinine less than or equal to 2.0 mg/dL;
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 3 x upper limit of normal;
• Bilirubin \< 1.5 (except Gilbert's disease);
• Lung slow vital capacity (SVC) \> 70% of predicted normal;
• No history of abnormal bleeding tendency;
• 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.

You may not qualify if:

• Subjects with any of the following cannot be enrolled in this study:
• uncontrolled infection;
• \< 3 drugs do not adequately control hypertension;
• Documented history of pulmonary embolism within 6 months of enrollment;
• 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;
• Patients with a history of coronary artery bypass grafting or angioplasty will be evaluated by cardiology and considered on a case-by-case basis;
• Seropositive for HIV, hepatitis B or hepatitis C;
• Pregnant or lactating patients;
• Patients of childbearing potential or males with partners of childbearing potential who are unwilling to use contraception;
• Participation in any other interventional study;
• 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;
• Prior treatment with ALS gene or cell therapy;
• History of clinically significant tumor, liver or kidney disease, or other uncontrolled disease;
• presence of a feeding tube;
• 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;

Sponsors & Collaborators

• Novabio Therapeutics: lead
• The First Affiliated Hospital of Zhengzhou University: collaborator

Study Sites (1)

The First Affiliated Hospital of Zhengzhou University

Zhengzhou, Henan, 450052, China

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Neurodegenerative Diseases

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Mingqi Lu, MD., PhD

CONTACT

609-716-7786; +86 15800843507; lumingqi@novabiotx.com

Study Design

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

Study Record Dates

• First Submitted: October 29, 2024
• First Posted: November 4, 2024
• Study Start: November 21, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2027
• Last Updated: September 25, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)