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ACTIVE NOT RECRUITING

NCT07279610

PHASE2/PHASE3

N-Acetylcysteine as Therapy for Transplantation- Associated Thrombotic Microangiopathy

Sponsor: The First Affiliated Hospital of Soochow University

View on ClinicalTrials.gov

Summary

This multicenter, prospective, single-arm clinical trial aims to evaluate the efficacy and safety of N-acetylcysteine (NAC) for treating Transplantation-Associated Thrombotic Microangiopathy (TA-TMA), a severe complication of hematopoietic stem cell transplantation characterized by microangiopathic hemolytic anemia, thrombocytopenia, and organ injury, with an incidence of 4%-30%. Current treatments, including plasma exchange (response rate \<10%) and costly complement inhibitors like Eculizumab (71% response) which are not widely accessible, are inadequate. Inspired by NAC's success in treating the related condition thrombotic thrombocytopenic purpura (TTP) and supported by bioinformatic analyses of patient data revealing enhanced oxidative stress pathways and identifying NAC as a potential targeted therapy, our prior study demonstrated that NAC prophylaxis significantly reduces TA-TMA incidence and improves survival. Building on this promising foundation, this study will enroll patients meeting TA-TMA diagnostic criteria for NAC treatment, assessing its potential as a safe, effective, and affordable therapeutic option.

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

Start Date

2024-05-01

Completion Date

2026-12-31

Last Updated

2025-12-12

Healthy Volunteers

Conditions

Thrombotic Microangiopathy

Interventions

DRUG

N-Acetylcysteine (NAC) Treatment

N-acetylcysteine injection will be administered intravenously to TA-TMA patients at a total daily dose of 16g. The daily dose is divided into two equal doses of 8g each, administered in the morning and evening. Each 8g dose is to be infused over a period of 1 hour. This regimen continues for 14 consecutive days.

Inclusion Criteria: \- 1. Informed Consent: The patient must have the ability to understand and the willingness to participate in the study and must sign a written Informed Consent Form. 2\. Age: ≥ 18 years old, regardless of gender. 3. Diagnosis: Subjects diagnosed with TA-TMA according to the Harmonizing Definitions, defined as meeting one of the following: * TA-TMA confirmed by renal or intestinal biopsy, OR * Fulfilling at least four of the following seven clinical or laboratory criteria within a 14-day period: 1. Anemia, defined as persistent transfusion dependence after myeloid engraftment, OR a decrease in hemoglobin \>10 g/L, OR new-onset transfusion dependence. 2. Thrombocytopenia, defined as failure of platelet engraftment, OR a higher-than-expected platelet transfusion requirement, OR refractory platelet transfusion, OR a \>50% decrease in platelets after initial engraftment. 3. Lactate dehydrogenase (LDH) level above the upper limit of normal (ULN). 4. Presence of schistocytes on peripheral blood smear. 5. Hypertension (blood pressure ≥140/90 mmHg). 6. sC5b-9 level above the ULN. 7. Proteinuria (random urine protein-to-creatinine ratio ≥1 mg/mg). Exclusion Criteria: * 1\. The subject has received complement blockade therapy (e.g., Eculizumab or Narsoplimab) within the past 3 months. 2\. The subject has a history of drug and/or alcohol abuse within the 6 months prior to enrollment. 3\. The subject has a life expectancy of less than 3 months. 4. The subject is considered by the investigator to be unable or unwilling to cooperate with the study procedures. 5\. The subject is a family member or employee of the investigator. 6. The patient is pregnant or lactating. 7. The subject has a history of Human Immunodeficiency Virus (HIV) infection. 8. The subject has a known allergy to any component of the investigational drug (N-acetylcysteine). 9\. The subject has cardiac insufficiency, defined as an ejection fraction (EF) \<30%, or NYHA Class III or higher heart failure, or other cardiac conditions deemed by the investigator as unsuitable for enrollment. 10\. The subject has contraindications to N-acetylcysteine, such as active bronchial asthma or peptic ulcer disease. 11\. The patient refuses to participate in the study.

Locations (1)

The First Affiliated Hospital of Soochow University

Suzhou, China