N-Acetylcysteine as Therapy for Transplantation- Associated Thrombotic Microangiopathy
Condition(s) studied
Investigational drug(s) / intervention(s)
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.
Study 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.
Eligibility
Primary outcome measure(s)
- The efficacy of N-acetylcysteine treatment for TA-TMA — Day 1 to 60 days after the enrollment of N-acetylcysteine
Evaluate the efficacy of N-acetylcysteine in patients with TA-TMA by response defined as: 1. Improvement in TMA laboratory markers of platelet count and lactate dehydrogenase (LDH), and 2. Improvement in clinical status
Trial sites (1)
| Facility | City | Region | Status |
|---|---|---|---|
| The First Affiliated Hospital of Soochow University | Suzhou | China |
More The First Affiliated Hospital of Soochow University trials in China
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07279610 on ClinicalTrials.gov ↗ ← All trials in China