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NCT07279610

N-Acetylcysteine as Therapy for Transplantation- Associated Thrombotic Microangiopathy

Active, enrolled Phase 2, PHASE3 Last updated 12 December 2025
What this trial tests

Phase 2, PHASE3 trial testing N-Acetylcysteine (NAC) Treatment in Thrombotic Microangiopathy in 44 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline
1 May 2024
Primary endpoint
31 December 2026
31 December 2026

Quick facts

Lead sponsorThe First Affiliated Hospital of Soochow University
PhasePhase 2, PHASE3
StatusActive, enrolled
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment44
Start date1 May 2024
Primary completion31 December 2026
Estimated completion31 December 2026
Sites1 location across China

Drugs / interventions tested

Conditions studied

Sponsor

The First Affiliated Hospital of Soochow University

Who can join

18 and older, any sex, with Thrombotic Microangiopathy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.

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Data sources for this page

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