Status:
COMPLETED
Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Paroxysmal Nocturnal Hemoglobinuria
Eligibility:
All Genders
18+ years
Brief Summary
This study will collect clinical response data on participants who were already treated with eculizumab for at least 26 weeks and who started ravulizumab treatment as a specific therapeutic strategy a...
Detailed Description
This study is an Italian multi-center, observational (non-interventional), cohort study composed of both retrospective and prospective observation periods on the same Paroxysmal Nocturnal Hemoglobinur...
Eligibility Criteria
Inclusion
- Body weight of 10 kilogram or above
- Hemolysis with clinical symptom(s) indicative of high disease activity
- Documented diagnoses of PNH confirmed by high-sensitivity flow cytometry evaluation of red blood cells and white blood cells with granulocyte or monocyte clone size of ≥ 5%
- Clinically stable after having been treated with eculizumab for at least the past 6 months
- Participant already assigned to ravulizumab treatment as a specific therapeutic strategy within the current routine clinical practice (this decision has to be made independently and before the enrolment of the participant in the study)
- Vaccinated against Neisseria meningitidis (according to Summary of Product Characteristics) \< 3 years before dosing or at least 2 weeks prior to initiating ravulizumab unless the risk of delaying ravulizumab therapy outweighs the risk of developing a meningococcal infection
- Signed written informed and privacy consent prior to study participation
Exclusion
- History of hematopoietic stem cell transplantation (evaluated at baseline)
- Known pregnant or breastfeeding participant (evaluated at baseline)
- Participant unable to read and write in Italian language and to autonomously fill in questionnaires and scales (evaluated at enrolment)
- Participants enrolled in any clinical study receiving experimental treatments for PNH (evaluated at baseline)
- Hypersensitivity to the active substance or to any of the excipient of the study drug.
- Participants with unresolved N. meningitidis infection at treatment initiation
- Participants who are not currently vaccinated against N. meningitidis unless they receive prophylactic treatment with appropriate antibiotics until 2 weeks after vaccination
Key Trial Info
Start Date :
June 17 2022
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
March 24 2025
Estimated Enrollment :
120 Patients enrolled
Trial Details
Trial ID
NCT05274633
Start Date
June 17 2022
End Date
March 24 2025
Last Update
June 12 2025
Active Locations (6)
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1
Clinical Trial Site
Brescia, Italy
2
Clinical Trial Site
Catania, Italy
3
Clinical Trial Site
Lecce, Italy
4
Clinical Trial Site
Ragusa, Italy