PNHNCT05337683
A Retrospective Non-Interventional Study to Evaluate the Impact of Eculizumab in Korean PNH Patients
An observational study of PNH, run by Handok Inc. at 1 site in South Korea.
Trial summary
This study is a retrospective non-interventional study, and the main objective of this study is to identify the impact of eculizumab treatment and the improvement of complications before and after eculizumab treatment, and to evaluate the safety during the treatment and the clinical unmet needs during the treatment in… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- PNH
- How many people
- 80 participants
- Where
- 1 siteSouth Korea
- Study dates
- 11 Dec 2020to 3 Jan 2022
- 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: Patients with PNH aged 18 years or older who started receiving eculizumab treatment accordance with the drug label until January 31, 2020 will be enrolled in this study.
Who may be able to join
- Patients with PNH aged 18 years or older who started eculizumab treatment in accordance with drug label between 2012 and January 31st, 2020. (However, patients who have received eculizumab as part of a clinical trial or an expanded access program/compassionate use program prior to 2012 are also eligible.)
- Patients who are vaccinated against Neisseria meningitidis at least 2 weeks prior to starting eculizumab treatment. Patients who have been treated with appropriate prophylactic antibiotic therapy for 2 weeks after vaccination if they received eculizumab within 2 weeks after the vaccination.
Who cannot join
- Pediatric patients with PNH aged under 18 years
- Patients with hypersensitivity to the active ingredients of eculizumab, murine protein, or other components
- Patients with untreated severe meningococcal (Neisseria meningitidis) infection
- Patients who received other complement inhibitors for PNH before or during eculizumab treatment
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Retrospective Non-Interventional Study to Evaluate the Impact of Eculizumab in Korean Paroxysmal Nocturnal Hemoglobinuria Patients
This study is a retrospective non-interventional study, and the main objective of this study is to identify the impact of eculizumab treatment and the improvement of complications before and after eculizumab treatment, and to evaluate the safety during the treatment and the clinical unmet needs during the treatment in PNH patients in real-world.
- Conditions
- PNH
What is being measured
The primary outcome is the main question the study is designed to answer.
Decrease of hemolysis after eculizumab treatment
The change in LDH levels before and after eculizumab treatment will be compared to determine whether hemolysis decreased after the treatment.
Measured: The results of LDH tests performed before eculizumab treatment, at 6, 12, 24 months after the start of eculizumab treatment, and every 12 months thereafter will be collected.
The Improvement/occurrence of complications and related clinical symptoms before and after eculizumab treatment
The major complications due to PNH include thromboembolism, pulmonary hypertension, renal failure, and smooth muscle spasm, and their occurrence will be determined by the investigator.
In addition, PNH-related symptoms (fatigue, anemia, dysphagia, shortness of breath/dyspnea, chest pain, abdominal pain, erectile dysfunction, hemoglobinuria, others) before and after eculizumab administration will be compared.
Measured: 6 months before eculizumab administration to the end of the administration (It is up to subject's treatment period. Through study completion, an average of 51months)
Severity of anemia
Hb levels will be compared to identify the severity of anemia.
Measured: Before the administration, at 6, 12, 24 months after the start of the treatment and every and 12 months thereafter
The required Unit of pRBC transfusion before and after eculizumab treatment
Measured: From 1 year before the treatment to the end of the administration (It is up to subject's treatment period. Through study completion, an average of 51months)
Effectiveness Evaluation at Last Follow-Up
The investigator will perform the final evaluation of the efficacy for entire observation period based on the collected data. The investigator will comprehensively judge and evaluate the decrease of hemolysis occurrence, the improvement and occurrence of complications, and the severity of anemia, etc.
Measured: At the last follow-up (At the time of data collection retrospectively)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2022
- Study start11 Dec 2020
- Primary completion30 Aug 2021
- Study completion3 Jan 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PNHStudies of the same condition, open and under way.
- 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
- Case only
- Time perspective
- Retrospective
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- Handok Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05337683
- Sponsor's protocol ID
- HD_ECU_OS2020
- Status last verified
- Jan 2025
- First submitted
- 8 Apr 2022
- Last updated
- 28 Jan 2025
- 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 28 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Retrospective Non-Interventional Study to Evaluate the Impact of Eculizumab in Korean Paroxysmal Nocturnal Hemoglobinuria Patients
- ClinicalTrials.gov ID
- NCT05337683
- Acronym / sponsor ID
- HD_ECU_OS2020
- Conditions
- PNH
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 80 participants (actual)
- Lead sponsor
- Handok Inc.Industry (pharma, biotech, devices)
- Study start
- 11 Dec 2020 (actual)
- Primary completion
- 30 Aug 2021 (actual)
- Study completion
- 3 Jan 2022 (actual)
- First posted
- 20 Apr 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 is a retrospective non-interventional study, and the main objective of this study is to identify the impact of eculizumab treatment and the improvement of complications before and after eculizumab treatment, and to evaluate the safety during the treatment and the clinical unmet needs during the treatment in PNH patients in real-world.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with PNH aged 18 years or older who started receiving eculizumab treatment accordance with the drug label until January 31, 2020 will be enrolled in this study.
Key inclusion criteria (2 listed)
- Patients with PNH aged 18 years or older who started eculizumab treatment in accordance with drug label between 2012 and January 31st, 2020. (However, patients who have received eculizumab as part of a clinical trial or an expanded access program/compassionate use program prior to 2012 are also eligible.)
- Patients who are vaccinated against Neisseria meningitidis at least 2 weeks prior to starting eculizumab treatment. Patients who have been treated with appropriate prophylactic antibiotic therapy for 2 weeks after vaccination if they received eculizumab within 2 weeks after the vaccination.
Key exclusion criteria (4 listed)
- Pediatric patients with PNH aged under 18 years
- Patients with hypersensitivity to the active ingredients of eculizumab, murine protein, or other components
- Patients with untreated severe meningococcal (Neisseria meningitidis) infection
- Patients who received other complement inhibitors for PNH before or during eculizumab treatment
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
Decrease of hemolysis after eculizumab treatment
Time frame: The results of LDH tests performed before eculizumab treatment, at 6, 12, 24 months after the start of eculizumab treatment, and every 12 months thereafter will be collected.
The change in LDH levels before and after eculizumab treatment will be compared to determine whether hemolysis decreased after the treatment.
The Improvement/occurrence of complications and related clinical symptoms before and after eculizumab treatment
Time frame: 6 months before eculizumab administration to the end of the administration (It is up to subject's treatment period. Through study completion, an average of 51months)
The major complications due to PNH include thromboembolism, pulmonary hypertension, renal failure, and smooth muscle spasm, and their occurrence will be determined by the investigator. In addition, PNH-related symptoms (fatigue, anemia, dysphagia, shortness of breath/dyspnea, chest pain, abdominal pain, erectile dysfunction, hemoglobinuria, others) before and after eculizumab administration will be compared.
Severity of anemia
Time frame: Before the administration, at 6, 12, 24 months after the start of the treatment and every and 12 months thereafter
Hb levels will be compared to identify the severity of anemia.
The required Unit of pRBC transfusion before and after eculizumab treatment
Time frame: From 1 year before the treatment to the end of the administration (It is up to subject's treatment period. Through study completion, an average of 51months)
Effectiveness Evaluation at Last Follow-Up
Time frame: At the last follow-up (At the time of data collection retrospectively)
The investigator will perform the final evaluation of the efficacy for entire observation period based on the collected data. The investigator will comprehensively judge and evaluate the decrease of hemolysis occurrence, the improvement and occurrence of complications, and the severity of anemia, etc.
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 28 Jan 2025.
- 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. This study is not recruiting at the moment.
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 PNH, run by Handok Inc. 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 2 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: finished, as of the sponsor's last update on 28 Jan 2025. 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?
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?
Decrease of hemolysis after eculizumab treatment (time frame: The results of LDH tests performed before eculizumab treatment, at 6, 12, 24 months after the start of eculizumab treatment, and every 12 months thereafter will be collected.). The study lists 5 primary outcomes in all. 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 28 Jan 2025.