Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria

Study Identifier:
HEMRAV601IT
ClinicalTrials.gov Identifier:
NCT05274633
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Paroxysmal Nocturnal Hemoglobinuria
Study Drug
  • Drug: Ravulizumab
Date
Jun 2022 - Jun 2024
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years
Requirements Information
Inclusion and Exclusion 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
Sex
Female & Male
Age
18+ years

Study Details

Medical Condition
  • Paroxysmal Nocturnal Hemoglobinuria
Study Drug
  • Drug: Ravulizumab
Date
Jun 2022 - Jun 2024
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years years
Requirements Information
Inclusion and Exclusion 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

Protocol 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.

Trial Locations

Location
Status
Location
Clinical Trial Site
Brescia, Italy
Status
N/A
Location
Clinical Trial Site
Catania, Italy
Status
N/A
Location
Clinical Trial Site
Lecce, Italy
Status
N/A
Location
Clinical Trial Site
Ragusa, Italy
Status
N/A
Location
Clinical Trial Site
Roma, Italy
Status
N/A
Location
Clinical Trial Site
Salerno, Italy
Status
N/A