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
- Conditions
- paroxysmal nocturnal hemoglobinuria
- Registration Number
- DRKS00025479
- Lead Sponsor
- Alexion Pharma Deutschland GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting withdrawn before recruiting started
- Sex
- All
- Target Recruitment
- 30
•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:
oFatigue
oHemoglobinuria
oAbdominal pain
oDyspnea
oAnemia [< 10 g/dl],
oMAVEc [including thrombosis],
oDysphagia
oErectile dysfunction
oPNH related transfusion
•Unwillingness / anticipated inability to attend further visits
•Off-label treatment with ravulizumab
Study & Design
- Study Type
- observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method