Analysis/Outcomes of Patients who switched from being under eculizumab to ravulizumab treatment in a real-world setting – according to the label but not included in Phase 3 studies

Analysis/Outcomes of Patients who switched from being under eculizumab to ravulizumab treatment in a real-world setting – according to the label but not included in Phase 3 studies - RavuReWo

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

DRKS

Registry ID

DRKS00025479

Enrollment

Registered

2021-10-29

Start date

2023-01-01

Completion date

Unknown

Last updated

2025-04-07

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

Conditions

paroxysmal nocturnal hemoglobinuria

Interventions

Group 1: Ravulizumab (Ultomiris)

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Inclusion criteria

Inclusion criteria: Inclusion criteria: • Patient is = 18 years at time of switch to ravulizumab • Diagnosis of PNH established by flow cytometry • Patient was pretreated with eculizumab and data from treatment with eculizumab are available • Willingness to participate in the study and provide written informed consent • Participant is currently receiving or starting treatment with ravulizumab treatment in accordance with the SmPC • Patient experienced HDA within 6 months of eculizumab treatment prior to switch to ravulizumab, with HDA defined as hemolysis (LDH = 1.5 x ULN) in combination with = 1 of the following PNH symptoms: o Fatigue o Hemoglobinuria o Abdominal pain o Dyspnea o Anemia [< 10 g/dl], o MAVEc [including thrombosis], o Dysphagia o Erectile dysfunction o PNH related transfusion

Exclusion criteria

Exclusion criteria: • Unwillingness / anticipated inability to attend further visits • Off-label treatment with ravulizumab

Design outcomes

Primary

Measure	Time frame
•Hemolysis measured by lactate dehydrogenase (LDH) levels (U/I) (% change and absolute) •Occurrence of BTH •Total number of units of packed red blood cell (pRBCs) transfused •Hemoglobin (Hb) stabilization over time •PNH symptoms (defined as: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia (hemoglobin < 10 g/dL), history of a major adverse vascular event (including thrombosis), dysphagia, or erectile dysfunction; or history of pRBC transfusion due to PNH)	—

Measure

Time frame

•Hemolysis measured by lactate dehydrogenase (LDH) levels (U/I) (% change and absolute) •Occurrence of BTH •Total number of units of packed red blood cell (pRBCs) transfused •Hemoglobin (Hb) stabilization over time •PNH symptoms (defined as: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia (hemoglobin < 10 g/dL), history of a major adverse vascular event (including thrombosis), dysphagia, or erectile dysfunction; or history of pRBC transfusion due to PNH)

—

Secondary

Measure	Time frame
• Time until BTH (time point within 8-week dosing intervals) • Cause of BTH (CAC vs. non-CAC related)	—

Countries

Germany

Contacts

Public ContactNicole Lewke

Alexion Pharma GmbH

nicole.lewke@alexion.com0176-30010167

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: Feb 4, 2026