Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic… (NCT07347990) | Clinical Trial Compass
Not Yet RecruitingNot Applicable
Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy
China30 participantsStarted 2026-01-01
Plain-language summary
The goal of this clinical trial is to evaluate the efficacy and safety of Iptacopan as a second-line treatment for high-risk hematopoietic stem cell transplantation-associated thrombotic microangiopathy (TA-TMA). Iptacopan is a selective oral small-molecule complement factor B inhibitor. It acts by inhibiting factor B, blocking the formation of C3 convertase, reducing C3b deposition, thereby suppressing C5 convertase (C3bBbC3b) and ultimately decreasing the formation of the membrane attack complex (MAC), which is expected to mitigate endothelial damage in TA-TMA pathology. The main questions this study aims to answer are:
* Does Iptacopan improve 6-month overall survival in high-risk TA-TMA patients?
* What adverse events do participants experience while taking Iptacopan?
* Does Iptacopan provide hematological response and organ function recovery in TA-TMA patients? In this prospective, multicenter, open-label, single-arm Phase II study, all participants will receive Iptacopan treatment. The primary endpoint of this study is the 6-month overall survival rate from TA-TMA diagnosis. Secondary endpoints include safety evaluation, hematological response, and organ function recovery.
During the study, participants will:
* Receive Iptacopan treatment according to protocol
* Undergo regular assessments for safety and efficacy monitoring
* Be followed for up to 24 months post-treatment initiation
Who can participate
Age range12 Years
SexALL
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
✓. Age ≥12 years at the time of ICF signature.
✓. Previous recipient of autologous or allogeneic HSCT.
✓. Persistent TA-TMA despite initial management of potential triggers (e.g., CNI/mTOR inhibitor reduction, infection or GVHD treatment), with TMA activity sustained for ≥72 hours post-intervention.
✓. TA-TMA diagnosis, confirmed ≤14 days prior to or during screening by either biopsy-proven microthrombi or ≥4 of the following:
✓. LDH ≥2× ULN
✓. Elevated sC5b-9
✓. Proteinuria (rUPCR ≥1 mg/mg)
✓. Multi-organ dysfunction syndrome (MODS)
Exclusion criteria
✕. Known familial or acquired ADAMTS13 deficiency (activity \<5%).
✕. Known Shiga toxin-associated HUS (positive Shiga toxin assay or culture).
What they're measuring
1
Six-month Overall Survival Rate Following TA-TMA Diagnosis
Timeframe: From the date of TA-TMA diagnosis until 6 months post-diagnosis.
Trial details
NCT IDNCT07347990
SponsorFirst Affiliated Hospital of Zhejiang University