Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy

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

Conditions

• Thrombotic Microangiopathy
• Hematopoietic Stem Cell Transplantation (HSCT)

Interventions

DRUG

iptacopan

Iptacopan will be administered under the supervision of hospital staff during inpatient stays or self-managed by patients in an outpatient setting. The induction phase lasts 4 weeks at a dosage of 200 mg twice daily (BID). Starting from Day 29, patients will enter the maintenance phase at a dosage of 200 mg once daily (QD), continuing until treatment completion at Week 12.

Sponsors & Collaborators

• Ruijin Hospital

  collaborator OTHER

• The First Affiliated Hospital of Zhengzhou University

  collaborator OTHER

• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

  collaborator OTHER

• Tongji Hospital

  collaborator OTHER

• The Children's Hospital of Zhejiang University School of Medicine

  collaborator OTHER

• First Affiliated Hospital of Ningbo University

  collaborator NETWORK

• Nanfang Hospital, Southern Medical University

  collaborator OTHER

• Peking University People's Hospital

  collaborator OTHER

• Fujian Medical University Union Hospital

  collaborator OTHER

• Hebei Yanda Ludaopei Hospital

  collaborator OTHER

• First Affiliated Hospital of Zhejiang University

  lead OTHER

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

12 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2026-01-01

Primary Completion

2027-12-31

Completion

2029-12-31

Countries

• China

Study Locations