Paroxysmal Nocturnal HaemoglobinuriaPNH-RECORDNCT06633536
Real-life Treatment Outcomes of Ravulizumab in PNH
An observational study of Paroxysmal Nocturnal Haemoglobinuria, run by AstraZeneca at 11 sites in Poland.
Trial summary
The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Haemoglobinuria
- What is being tested?
- Ravulizumab
- How many people
- 64 participants
- Where
- 11 sitesPoland
- Study dates
- 6 Feb 2025to 1 Aug 2028
- 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: Adult (aged ≥18) patients with PNH
Who may be able to join
- Adult (aged ≥18) patients with PNH receiving ravulizumab treatment in the frames of NDP in Poland.
- Patients willing to participate in the study and signed ICF.
Who cannot join
- Those who participated in ravulizumab clinical trial in the past, and/or those who participated/plans to participate in clinical trial on/after the date of first ravulizumab infusion through NDP.
- Cognitive incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
Poland11 sites
Research Site
Bialystok
Research Site
Brzozów
Research Site
Bydgoszcz
Research Site
Gdansk
Research Site
Krakow
Research Site
Lodz
Research Site
Lublin
Research Site
Opole
Research Site
Szczecin
Research Site
Wałbrzych
Research Site
Warsaw
About this study
Official title: Real-life Treatment Outcomes of Ravulizumab in Polish Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH). Prospective and Retrospective, Multicenter, Non-interventional Study.
The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.
Read the detailed description
PNH-RECORD study aims to aims to collect data on the patients' characteristics and clinical outcomes of ravulizumab administered in the scope of routine clinical practice in PNH.
- Conditions
- Paroxysmal Nocturnal Haemoglobinuria
- Being tested
- Ravulizumab
- Keywords
- Paroxysmal nocturnal haemoglobinuria, PNH, ravulizumab
- 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
open-label arm with ravulizumab administrated within the scope of routine clinical practice
| Study group | Role | What they receive |
|---|---|---|
| Ravulizumab group | Open-label Receives: Drug: Ravulizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in LDH levels from index date (first dose of ravulizumab) at every 6 month
Measured: up to 36 months
Proportion of patients achieving or remaining at LDH<1.5 ULN at every 6 month
Measured: up to 36 months
Secondary outcomes (10)
Proportion of transfusion-free patients from index date at every 6 month
Measured: up to 36 months
Number of blood units used per patient-year
Measured: up to 36 months
To present patients characteristics
Mean age (years) at PNH diagnosis
Measured: Baseline
To present patients characteristics
Mean age (years) at index date
Measured: Baseline
To present patients characteristics
Proportion (%) of men and women
Measured: Baseline
To present patients characteristics
Mean body mass index (kg/m²)
Measured: Baseline
To present clinical characteristics
Mean and median (years) duration of PNH
Measured: Baseline
To present clinical characteristics
Mean and median (months) time from diagnosis to complement inhibitors treatment initiation
Measured: Baseline
To present clinical characteristics
Proportion (%) of patients with complication of haemolysis, thrombosis, serious vascular event andaplastic anemia
Measured: Baseline
To present clinical characteristics
Proportion (%) of patients naïve to complement inhibitors
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2024
- Study start6 Feb 2025
- Primary completion1 Aug 2028 (estimated)
- Study completion1 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HaemoglobinuriaStudies of the same condition, open and under way.
- More trials run by AstraZenecaThe 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
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06633536
- Sponsor's protocol ID
- D9289R00001
- Acronym
- PNH-RECORD
- Status last verified
- Sep 2026
- First submitted
- 9 Aug 2024
- Last updated
- 23 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real-life Treatment Outcomes of Ravulizumab in Polish Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH). Prospective and Retrospective, Multicenter, Non-interventional Study.
- ClinicalTrials.gov ID
- NCT06633536
- Acronym / sponsor ID
- PNH-RECORD / D9289R00001
- Conditions
- Paroxysmal Nocturnal Haemoglobinuria
- Keywords
- Paroxysmal nocturnal haemoglobinuria, PNH, ravulizumab
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 64 participants (actual)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- 6 Feb 2025 (actual)
- Primary completion
- 1 Aug 2028 (estimated)
- Study completion
- 1 Aug 2028 (estimated)
- First posted
- 9 Oct 2024 (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.
The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.
Detailed description
PNH-RECORD study aims to aims to collect data on the patients' characteristics and clinical outcomes of ravulizumab administered in the scope of routine clinical practice in PNH.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ravulizumab group | – | Drug: RavulizumabOpen-label |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult (aged ≥18) patients with PNH
Key inclusion criteria (2 listed)
- Adult (aged ≥18) patients with PNH receiving ravulizumab treatment in the frames of NDP in Poland.
- Patients willing to participate in the study and signed ICF.
Key exclusion criteria (2 listed)
- Those who participated in ravulizumab clinical trial in the past, and/or those who participated/plans to participate in clinical trial on/after the date of first ravulizumab infusion through NDP.
- Cognitive incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
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 |
|---|---|---|
| Ravulizumabopen-label arm with ravulizumab administrated within the scope of routine clinical practice | Drug | Ravulizumab group |
Primary outcome
Change in LDH levels from index date (first dose of ravulizumab) at every 6 month
Time frame: up to 36 months
Proportion of patients achieving or remaining at LDH<1.5 ULN at every 6 month
Time frame: up to 36 months
10 secondary outcomes
Proportion of transfusion-free patients from index date at every 6 month
Time frame: up to 36 months
Number of blood units used per patient-year
Time frame: up to 36 months
To present patients characteristics
Time frame: Baseline
To present patients characteristics
Time frame: Baseline
To present patients characteristics
Time frame: Baseline
To present patients characteristics
Time frame: Baseline
To present clinical characteristics
Time frame: Baseline
To present clinical characteristics
Time frame: Baseline
To present clinical characteristics
Time frame: Baseline
To present clinical characteristics
Time frame: Baseline
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 23 Sep 2026.
- Individual participant dataSharing planned: Yes. Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
Related trials for Paroxysmal Nocturnal Haemoglobinuria
Enrolling by invitationObservational study
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
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
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.
11 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 Haemoglobinuria, run by AstraZeneca at 11 sites in Poland.
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 2 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: under way, not looking for participants, as of the sponsor's last update on 23 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Poland. 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?
Change in LDH levels from index date (first dose of ravulizumab) at every 6 month (time frame: up to 36 months). The study lists 2 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 23 Sep 2026.