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

NCT05274633 | Completed FDA Drug

• 1 other identifier: HEMRAV601IT
• Study Type: observational
• Target: 120
• Locations: 1 country
• Sites: 6

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

PIGA Gene; Complement-inhibitor Protein; CD55; CD59; Efficacy; Safety; Patient Registry

Outcome Measures

Primary Outcomes (1)

• Percentage Change In Lactate Dehydrogenase (LDH) From Baseline To End Of Observation

  The analysis using descriptive statistics will be performed on the Full Analysis Set, including the participants having the LDH evaluation at both the baseline and at the end of observation.

  Baseline through up to Week 52

Secondary Outcomes (11)

• Percentage Change In LDH From Baseline To End Of Observation On Participants Treated With Ravulizumab With Respect To The Observed Treatment Period With Eculizumab

  Baseline through up to Week 52

• Number of Transfusions During Treatment Period With Ravulizumab

  Baseline through up to Week 52

• Total Number Of Transfusion Sessions During The Treatment Period With Ravulizumab

  Baseline through up to Week 52

• Number Of Participants Undergoing Ravulizumab Without A ≥ 2 gram/deciliter (g/dL) Decrease In Hemoglobin Level In The Absence Of Transfusion

  Baseline through up to Week 52

• Breakthrough Hemolysis (BTH) In The Presence Of Elevated LDH During Treatment Period With Ravulizumab

  Baseline through up to Week 52

Study Arms (1)

Participants With PNH

Data will be collected on participants who were already treated with eculizumab for at least 26 weeks and who started ravulizumab treatment as per clinical practice.

Drug: Ravulizumab

Interventions

Ravulizumab DRUG

Participants will be observed for 52 weeks after the start of ravulizumab.

Also known as: Ultomiris

Participants With PNH

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (6)

Clinical Trial Site

Brescia, Italy

Location

Clinical Trial Site

Catania, Italy

Location

Clinical Trial Site

Lecce, Italy

Location

Clinical Trial Site

Ragusa, Italy

Location

Clinical Trial Site

Roma, Italy

Location

Clinical Trial Site

Salerno, Italy

Location

Related Publications (1)

• Iori AP, De Vivo A, Di Bona E, Caocci G, Fioritoni F, Ciceri F, Beggiato E, Rapezzi D, Amendola A, Figuera A, Selleri C, Longu F, Fattizzo B, Tucci A, Cignetti A, Amico V, Sica S, Metafuni E, Raso S, Urbano TA, Marano L, Di Renzo N, Spedini P, Rambaldi A, Lanza F, Clissa C, Danesin C, Greve MB, Cabibbo S, Ori A, Cassanelli F, Sottana F, Campolo B, Gasparri G, Carini F, Barcellini W. Real life use of ravulizumab in Italian patients with paroxysmal nocturnal hemoglobinuria: evidence from the REACTION observational study. Ann Hematol. 2026 Jan 22;105(2):50. doi: 10.1007/s00277-026-06792-w.

  PMID: 41566054 DERIVED

Related Links

• Related Info

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

ravulizumab

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 2, 2022
• First Posted: March 10, 2022
• Study Start: June 17, 2022
• Primary Completion: June 30, 2024
• Study Completion: March 24, 2025
• Last Updated: May 7, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, CSR

Locations

IT (6)