Clinical trial NCT05274633: Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
Official title: Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria a Multicenter Observational Retrospective and Prospective Cohort Study.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: PIGA Gene
Complement-inhibitor Protein
CD55
CD59
Efficacy
Safety
Patient Registry
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2022-03-10
Enrollment: 120
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with documented diagnosis of PNH that were already treated with eculizumab and started treatment with ravulizumab and met the inclusion and exclusion criteria.

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 as per ordinary clinical practice.

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 Hemoglobinuria (PNH) participants. After the First Participant In from different Italian study centers, participants will be consecutively enrolled for 9 months and they will be observed for 52 weeks after the start of ravulizumab.

Eligibility criteria: Inclusion Criteria:

* 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 Criteria:

* 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

Primary outcomes:
measure: Percentage Change In Lactate Dehydrogenase (LDH) From Baseline To End Of Observation
timeFrame: Baseline through up to Week 52
Secondary outcomes:
measure: Percentage Change In LDH From Baseline To End Of Observation On Participants Treated With Ravulizumab With Respect To The Observed Treatment Period With Eculizumab
timeFrame: Baseline through up to Week 52
measure: Number of Transfusions During Treatment Period With Ravulizumab
timeFrame: Baseline through up to Week 52
measure: Total Number Of Transfusion Sessions During The Treatment Period With Ravulizumab
timeFrame: Baseline through up to Week 52
measure: Number Of Participants Undergoing Ravulizumab Without A ≥ 2 gram/deciliter (g/dL) Decrease In Hemoglobin Level In The Absence Of Transfusion
timeFrame: Baseline through up to Week 52
measure: Breakthrough Hemolysis (BTH) In The Presence Of Elevated LDH During Treatment Period With Ravulizumab
timeFrame: Baseline through up to Week 52
measure: Change From Baseline To Each Timepoint Of Assessment Using Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale
timeFrame: Baseline through up to Week 52
measure: Change From Baseline To Each Timepoint Of Assessment Using European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 Scale
timeFrame: Baseline through up to Week 52
measure: PNH-specific Patient Preference Questionnaire (PPQ)
timeFrame: Week 52
measure: Number of Participants Experiencing Adverse Events (AEs)
timeFrame: Baseline through up to Week 52
measure: Costs Related to PNH Sustained By National Health System (NHS) For Ravulizumab Treatment
timeFrame: Baseline, Weeks 18, 34, and 52
measure: Costs Sustained By The Participants Related To The Infusion Visits For Ravulizumab Treatment
timeFrame: Baseline, Weeks 18, 34, and 52
Other outcomes:
Locations:
city: Brescia
country: Italy
facility: Clinical Trial Site
city: Catania
country: Italy
facility: Clinical Trial Site
city: Lecce
country: Italy
facility: Clinical Trial Site
city: Ragusa
country: Italy
facility: Clinical Trial Site
city: Roma
country: Italy
facility: Clinical Trial Site
city: Salerno
country: Italy
facility: Clinical Trial Site
Study officials:
References: