HemoglobinuriaESR-25-22871NCT07921823
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
An observational study of Hemoglobinuria and Paroxysmal, run by Yonsei University at 1 site in South Korea.
Trial summary
This multicenter, retrospective observational cohort study aims to evaluate the long-term real-world effectiveness and safety of ravulizumab in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in South Korea over a 4-year post-marketing period. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemoglobinuria, Paroxysmal
- How many people
- 78 participants
- Where
- 1 siteSouth Korea
- Study dates
- 22 Jul 2026to 31 Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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: Korean patients with Paroxysmal Nocturnal Hemoglobinuria whose retrospective clinical data are available in the electronic medical records (EMR) of the participating hospitals.
Who may be able to join
- Adult patients (18 years of age or older) with a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) documented by flow cytometry.
- Patients who initiated ravulizumab treatment on or before February 28, 2025, and have at least 6 months of follow-up data available.
- To reflect real-world clinical practice, meningococcal (Neisseria meningitidis) vaccination status will be collected if available, but is not a mandatory requirement for study inclusion.
Who cannot join
- Less than 18 years of age at the time of ravulizumab treatment initiation.
- Known hypersensitivity to ravulizumab or any of its excipients.
- Untreated Neisseria meningitidis infection at the time of ravulizumab treatment initiation.
- Prior or concomitant use of other complement inhibitors for PNH (with the exception of prior eculizumab use).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
This multicenter, retrospective observational cohort study aims to evaluate the long-term real-world effectiveness and safety of ravulizumab in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in South Korea over a 4-year post-marketing period. By analyzing electronic medical records (EMR) from participating institutions, the study investigates intravascular hemolysis control, transfusion avoidance, breakthrough hemolysis (BTH) characteristics, and overall clinical outcomes in an Asian population.
- Conditions
- Hemoglobinuria, Paroxysmal
- Medical subject headings
- Hemoglobinuria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| PNH patients treated with Ravulizumab | Patients diagnosed with PNH and prescribed Ravulizumab at multiple centers in South Korea, whose electronic medical records (EMR) are available for retrospective review. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of patients achieving lactate dehydrogenase (LDH) levels less than or equal to 1.5 times the upper limit of normal (ULN)
Assessment of intravascular hemolysis control, defined as achieving and maintaining serum LDH levels at or below 1.5 times the ULN during the 4-year post-marketing observation period.
Measured: Up to 4 years after ravulizumab initiation
Secondary outcomes (1)
Incidence and clinical characteristics of breakthrough hemolysis (BTH)
BTH is defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia [hemoglobin less than 10 g/dL], major adverse vascular events [MAVE, including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated lactate dehydrogenase (LDH) levels of at least 2 times the upper limit of normal (ULN), after prior reduction of LDH to less than 1.5 times the ULN during therapy. Associated contextual factors, including complement-amplifying conditions, hospitalizations, transfusions, and the use of rescue medication, will also be recorded and characterized at each event.
Measured: hroughout the follow-up period, assessed at each occurrence of a BTH event (up to 4 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2026
- Study start22 Jul 2026
- Primary completion20 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Yonsei UniversityThe 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
- Retrospective
- Enrolment
- 78 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yonsei University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07921823
- Sponsor's protocol ID
- 4-2026-0786
- Acronym
- ESR-25-22871
- Status last verified
- Nov 2026
- First submitted
- 2 Nov 2026
- Last updated
- 6 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
- ClinicalTrials.gov ID
- NCT07921823
- Acronym / sponsor ID
- ESR-25-22871 / 4-2026-0786
- Conditions
- Hemoglobinuria, Paroxysmal
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 78 participants (estimated)
- Lead sponsor
- Yonsei UniversityUniversity, hospital or other
- Study start
- 22 Jul 2026 (actual)
- Primary completion
- 20 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 6 Nov 2026 (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 multicenter, retrospective observational cohort study aims to evaluate the long-term real-world effectiveness and safety of ravulizumab in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in South Korea over a 4-year post-marketing period. By analyzing electronic medical records (EMR) from participating institutions, the study investigates intravascular hemolysis control, transfusion avoidance, breakthrough hemolysis (BTH) characteristics, and overall clinical outcomes in an Asian population.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PNH patients treated with Ravulizumab | – | –Patients diagnosed with PNH and prescribed Ravulizumab at multiple centers in South Korea, whose electronic medical records (EMR) are available for retrospective review. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Korean patients with Paroxysmal Nocturnal Hemoglobinuria whose retrospective clinical data are available in the electronic medical records (EMR) of the participating hospitals.
Key inclusion criteria (3 listed)
- Adult patients (18 years of age or older) with a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) documented by flow cytometry.
- Patients who initiated ravulizumab treatment on or before February 28, 2025, and have at least 6 months of follow-up data available.
- To reflect real-world clinical practice, meningococcal (Neisseria meningitidis) vaccination status will be collected if available, but is not a mandatory requirement for study inclusion.
Key exclusion criteria (4 listed)
- Less than 18 years of age at the time of ravulizumab treatment initiation.
- Known hypersensitivity to ravulizumab or any of its excipients.
- Untreated Neisseria meningitidis infection at the time of ravulizumab treatment initiation.
- Prior or concomitant use of other complement inhibitors for PNH (with the exception of prior eculizumab use).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Proportion of patients achieving lactate dehydrogenase (LDH) levels less than or equal to 1.5 times the upper limit of normal (ULN)
Time frame: Up to 4 years after ravulizumab initiation
Assessment of intravascular hemolysis control, defined as achieving and maintaining serum LDH levels at or below 1.5 times the ULN during the 4-year post-marketing observation period.
1 secondary outcome
Incidence and clinical characteristics of breakthrough hemolysis (BTH)
Time frame: hroughout the follow-up period, assessed at each occurrence of a BTH event (up to 4 years)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2026.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemoglobinuria and Paroxysmal, run by Yonsei University at 1 site in South Korea.
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 3 inclusion and 4 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: enrolling by invitation, as of the sponsor's last update on 6 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. 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?
This study is still under way. Results are not available in the source record yet.
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?
Proportion of patients achieving lactate dehydrogenase (LDH) levels less than or equal to 1.5 times the upper limit of normal (ULN) (time frame: Up to 4 years after ravulizumab initiation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 6 Nov 2026; AzTrials last refreshed its copy on 6 Nov 2026.