A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Ravulizumab in Adult and Adolescent Participants who have Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplant (HSCT)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-510107-22-00

Acronym

ALXN1210-TMA-313

Enrollment

Registered

2024-03-05

Start date

2020-10-27

Completion date

Unknown

Last updated

2025-07-09

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

Brief summary

Event free survival during the 26-week Treatment Period defined as the time from randomization until the first of the two following events: Death and Clinical worsening

Detailed description

Overall survival by Day 100, Week 26, Non-relapse mortality during the 26-week Treatment Period, Number of TMA response criteria met during the 26-week Treatment Period, Hematologic response during the 26-week Treatment Period, Change from baseline in eGFR at Week 26

Interventions

DRUGAnti C5 Complement mAb Placebo

DRUGUltomiris 300 mg/30 mL concentrate for solution for infusion

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Event free survival during the 26-week Treatment Period defined as the time from randomization until the first of the two following events: Death and Clinical worsening	—

Secondary

Measure	Time frame
Overall survival by Day 100, Week 26, Non-relapse mortality during the 26-week Treatment Period, Number of TMA response criteria met during the 26-week Treatment Period, Hematologic response during the 26-week Treatment Period, Change from baseline in eGFR at Week 26	—

Countries

Belgium, France, Germany, Greece, Italy, Netherlands, Poland, Spain, Sweden

Outcome results

None listed

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