Paroxysmal Nocturnal HemoglobinuriaNCT05274633
Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc. at 6 sites in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- Ravulizumab
- How many people
- 120 participants
- Where
- 6 sitesItaly
- Study dates
- 17 Jun 2022to 24 Mar 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Italy6 sites
Clinical Trial Site
Brescia
Clinical Trial Site
Catania
Clinical Trial Site
Lecce
Clinical Trial Site
Ragusa
Clinical Trial Site
Roma
Clinical Trial Site
Salerno
About this study
Official title: Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria a Multicenter Observational Retrospective and Prospective Cohort Study.
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.
Read the 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.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- Ravulizumab
- Keywords
- PIGA Gene, Complement-inhibitor Protein, CD55, CD59, Efficacy, Safety, Patient Registry
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RavulizumabDrug
Also called Ultomiris
Participants will be observed for 52 weeks after the start of ravulizumab.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Ravulizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
The difference of percentage change in LDH from baseline to end of observation will be calculated on participants treated with ravulizumab (over the 52 weeks after baseline) and on the same participants treated with eculizumab (during up to 52 weeks before baseline).
Measured: Baseline through up to Week 52
Number of Transfusions During Treatment Period With Ravulizumab
The number of participants who needed transfusions, with relative frequency and percentage, and descriptive statistics on the total number of transfusions (number of packed red blood cell units transfused) will be calculated for both the treatment period with ravulizumab and the treatment period with eculizumab.
Measured: Baseline through up to Week 52
Total Number Of Transfusion Sessions During The Treatment Period With Ravulizumab
The number of participants who needed transfusions, with relative frequency and percentage, and descriptive statistics on the number of transfusion sessions (number of days) will be calculated for both the treatment period with ravulizumab and the treatment period with eculizumab.
Measured: 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
The proportion of participants without a ≥ 2 g/dL decrease in hemoglobin level in the absence of transfusion will be calculated for both the treatment period with ravulizumab and the treatment period with eculizumab.
Measured: Baseline through up to Week 52
Breakthrough Hemolysis (BTH) In The Presence Of Elevated LDH During Treatment Period With Ravulizumab
BTH is defined as at least 1 new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia [hemoglobin < 10 g/dL], major adverse vascular event including thrombosis, dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥ 2 * upper limit of normal (ULN) after prior LDH reduction to < 1.5 * ULN while on therapy.
Measured: Baseline through up to Week 52
Change From Baseline To Each Timepoint Of Assessment Using Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale
The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants will score each item on a 5-point scale. Total scores range from 0 to 52, with a higher score indicating better Quality of Life (QoL).
Measured: Baseline through up to Week 52
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
EORTC is a questionnaire developed to assess the QoL of cancer participants over the preceding 7 days. The questionnaire has 30 questions related to QoL, with the first 28 questions scored on a 4-point scale and the final 2 questions that probe the participant's overall health and QoL scored on a scale of 1 (very poor) to 7 (excellent). Each subscale has a range of 0 to 100%. Thus, a high score for a functional scale represents a high/healthy level of functioning, a high score for the global health status/QoL represents a high QoL, but a high score for a symptom scale/item represents a high level of symptomatology/problem.
Measured: Baseline through up to Week 52
PNH-specific Patient Preference Questionnaire (PPQ)
PNH-PPQ is a participant-centered approach for evaluating preferences for the treatment of PNH. It contains 11 questions assessing overall treatment preference, evaluating treatment preference according to 9 treatment characteristics, assessing the most important treatment characteristic for participant overall medication preference, and evaluating those same aspects of treatment with ravulizumab.
Measured: Week 52
Number of Participants Experiencing Adverse Events (AEs)
An AE is any untoward medical occurrence in a participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. AEs that will occur during the ravulizumab treatment will be collected and will be reported to the Investigator or qualified designee by the participant.
Measured: Baseline through up to Week 52
Costs Related to PNH Sustained By National Health System (NHS) For Ravulizumab Treatment
The following medical costs related to PNH sustained by NHS will be collected: specialist visits, pharmacological and non-pharmacological treatments, hospital and emergency rooms admissions, and examinations (by examination type). This will be reported through descriptive statistics and will be appraised considering the quantity of resource consumption by medical resources and its unit costs.
Measured: Baseline, Weeks 18, 34, and 52
Costs Sustained By The Participants Related To The Infusion Visits For Ravulizumab Treatment
The following costs sustained by the participants related to the infusion visits will be collected: average cost to reach the structure, overall time required for the infusion (time to reach the structure + time of infusion), retirement status of the participant, loss of working days, and need of a caregiver. This will be calculated in terms of the total cost of transport and overall time required for infusion.
Measured: Baseline, Weeks 18, 34, and 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2022
- Study start17 Jun 2022
- Primary completion30 Jun 2024
- Study completion24 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 120 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05274633
- Sponsor's protocol ID
- HEMRAV601IT
- Status last verified
- Feb 2026
- First submitted
- 2 Mar 2022
- Last updated
- 7 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Publications
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.
PubMed 41566054
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria a Multicenter Observational Retrospective and Prospective Cohort Study.
- ClinicalTrials.gov ID
- NCT05274633
- Acronym / sponsor ID
- HEMRAV601IT
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PIGA Gene, Complement-inhibitor Protein, CD55, CD59, Efficacy, Safety, Patient Registry
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 120 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 17 Jun 2022 (actual)
- Primary completion
- 30 Jun 2024 (actual)
- Study completion
- 24 Mar 2025 (actual)
- First posted
- 10 Mar 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants With PNH | – | Drug: RavulizumabData 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (7 listed)
- 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)
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RavulizumabAlso: UltomirisParticipants will be observed for 52 weeks after the start of ravulizumab. | Drug | Participants With PNH |
Primary outcome
Percentage Change In Lactate Dehydrogenase (LDH) From Baseline To End Of Observation
Time frame: Baseline through up to Week 52
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.
11 secondary outcomes
Percentage Change In LDH From Baseline To End Of Observation On Participants Treated With Ravulizumab With Respect To The Observed Treatment Period With Eculizumab
Time frame: Baseline through up to Week 52
Number of Transfusions During Treatment Period With Ravulizumab
Time frame: Baseline through up to Week 52
Total Number Of Transfusion Sessions During The Treatment Period With Ravulizumab
Time frame: 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
Time frame: Baseline through up to Week 52
Breakthrough Hemolysis (BTH) In The Presence Of Elevated LDH During Treatment Period With Ravulizumab
Time frame: Baseline through up to Week 52
Change From Baseline To Each Timepoint Of Assessment Using Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale
Time frame: Baseline through up to Week 52
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
Time frame: Baseline through up to Week 52
PNH-specific Patient Preference Questionnaire (PPQ)
Time frame: Week 52
Number of Participants Experiencing Adverse Events (AEs)
Time frame: Baseline through up to Week 52
Costs Related to PNH Sustained By National Health System (NHS) For Ravulizumab Treatment
Time frame: Baseline, Weeks 18, 34, and 52
Costs Sustained By The Participants Related To The Infusion Visits For Ravulizumab Treatment
Time frame: Baseline, Weeks 18, 34, and 52
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2026.
- Derived from this study: PubMed 41566054Iori 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.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc. at 6 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage Change In Lactate Dehydrogenase (LDH) From Baseline To End Of Observation (time frame: Baseline through up to Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05274633, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 May 2026.