Using regulatory T cells to treat neurodegenerative diseases

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

This study is testing if injections of specially made immune cells can help people with neurodegenerative diseases like ALS, Alzheimer's, and Multiple System Atrophy feel better.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Novabio Therapeutics (Industry-sponsored)
• Locations: 1 site (Zhengzhou, Henan)
• Trial ID: NCT06671236 on ClinicalTrials.gov

What this trial studies

This clinical study evaluates the safety and efficacy of autologous human polyclonal regulatory T cell injections (NP001) in patients with neurodegenerative diseases such as ALS, Alzheimer's Disease, and Multiple System Atrophy. Participants will undergo apheresis to collect cells for the manufacturing of NP001, which will be administered intrathecally at specified doses over a 12-month period. The study is open-label and non-randomized, with follow-up assessments to monitor safety and efficacy outcomes.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 to 70 with a confirmed diagnosis of ALS, Alzheimer's Disease, or Multiple System Atrophy.

Not a fit: Patients with advanced stages of neurodegenerative diseases or those with significant cognitive impairment may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a novel therapeutic option for patients suffering from debilitating neurodegenerative diseases.

How similar studies have performed: While the use of regulatory T cells in treating neurodegenerative diseases is a novel approach, similar studies have shown promise in other conditions, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

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Patients must meet all of the following criteria to be eligible for enrollment in this study:

1. Male or female patients aged 18 to 70 years;
2. 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;
3. 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®);
4. Patients must have \> two weeks after the end of major surgery and after the completion of participation in other research trials;
5. Patients must have recovered from clinical toxicity (CTCAE \[5th Edition\] toxicity values have resolved to \< 2);
6. Serum creatinine less than or equal to 2.0 mg/dL;
7. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 3 x upper limit of normal;
8. Bilirubin \< 1.5 (except Gilbert's disease);
9. Lung slow vital capacity (SVC) \> 70% of predicted normal;
10. No history of abnormal bleeding tendency;
11. 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.

Exclusion Criteria:

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Subjects with any of the following cannot be enrolled in this study:

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 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.

Where this trial is running

Zhengzhou, Henan

• The First Affiliated Hospital of Zhengzhou University — Zhengzhou, Henan, China (Recruiting)

Study contacts

• Study coordinator: Mingqi Lu, MD., PhD

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