A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants (from 1 month to <18 years of age) with Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSCT)

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-507850-33-00

Acronym

ALXN1210-TMA-314

Enrollment

Registered

2023-11-17

Start date

2021-02-05

Completion date

2025-05-27

Last updated

2025-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hematopoietic stem cell transplant-associated thrombotic microangiopathy(HSCT- TMA)

Brief summary

TMA response

Detailed description

Time to TMA response, Change from baseline in TMA-associated organ dysfunction in renal system, cardiovascular system, pulmonary system, CNS, and GI system at 6 months and 1 year, TMA relapse during the follow-up period, Overall survival, Non-relapse mortality, Platelet response, Hematologic response

Interventions

DRUGUltomiris 300 mg/30 mL concentrate for solution for infusion

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
TMA response	—

Secondary

Measure	Time frame
Time to TMA response, Change from baseline in TMA-associated organ dysfunction in renal system, cardiovascular system, pulmonary system, CNS, and GI system at 6 months and 1 year, TMA relapse during the follow-up period, Overall survival, Non-relapse mortality, Platelet response, Hematologic response	—

Countries

France, Germany, Italy, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026