N-Acetylcysteine as Therapy for Transplantation- Associated Thrombotic Microangiopathy (NCT07279610) | Clinical Trial Compass
Active — Not RecruitingPhase 2/3
N-Acetylcysteine as Therapy for Transplantation- Associated Thrombotic Microangiopathy
China44 participantsStarted 2024-05-01
Plain-language 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Anemia, defined as persistent transfusion dependence after myeloid engraftment, OR a decrease in hemoglobin \>10 g/L, OR new-onset transfusion dependence.
. 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.
. Lactate dehydrogenase (LDH) level above the upper limit of normal (ULN).
. Presence of schistocytes on peripheral blood smear.
. Hypertension (blood pressure ≥140/90 mmHg).
. sC5b-9 level above the ULN.
. Proteinuria (random urine protein-to-creatinine ratio ≥1 mg/mg).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The efficacy of N-acetylcysteine treatment for TA-TMA
Timeframe: Day 1 to 60 days after the enrollment of N-acetylcysteine
Trial details
NCT IDNCT07279610
SponsorThe First Affiliated Hospital of Soochow University