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

Launched by NOVABIO THERAPEUTICS · Nov 1, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a treatment using a special type of immune cell called regulatory T cells (or Tregs) to see if it can help patients with neurodegenerative diseases, specifically amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), and multiple system atrophy (MSA). The goal is to assess how safe this treatment is and whether it might improve the health of individuals with these conditions. The trial is currently looking for participants aged 18 to 70 who meet specific health criteria related to their diagnosis, such as having a confirmed diagnosis of ALS, AD, or MSA, and having a sufficient number of their own T cells for the treatment.

Participants in this study can expect to receive injections of their own modified Tregs and will be closely monitored for any effects. It's important to note that there are certain health requirements to be eligible, such as not having serious infections or heart issues, and participants should be stable on their current medications for at least three months before joining. If you or someone you know is interested in participating, it’s a good idea to discuss this with a healthcare provider to understand more about the potential benefits and risks.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • -
• 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:
• • ①ALS: 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;
• • ②MSA: 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.
• • ③AD: 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. 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:
• • -
• 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, 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.