Paroxysmal Nocturnal HemoglobinuriaReWoPNHNCT06154512
A Real-world, Multi-center, Prospective, Observational Study for PNH in China
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by AstraZeneca at 40 sites in China.
Trial summary
As a rare disease listed in the First Catalogue of Rare Diseases in China (National Health Commission of the People's Republic of China, 2019), PNH is poorly studied in China subse-quently leading to the inadequate elucidation of disease characteristics and clinical outcomes. Eculizumab was recently approved by NMPA. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- How many people
- 724 participants
- Where
- 40 sitesChina
- Study dates
- 10 Nov 2023to 3 Dec 2025
- How long will I take part?
- 1 Year
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Study Population: Number of patients (estimate): 1000; Inclusion Criteria 1. Patients of any age; 2. Diagnosed PNH with detected proportion of PNH clone cells of at least 1%; 3. Patient or patient's family must be willing and able to give written informed consent. Exclusion Criteria 1. Current or previous treatment with a non-eculizumab complement inhibitor; 2. Patients in other PNH clinical trials. 3. Unable to give written informed consent.
Who may be able to join
- Patients of any age;
- Diagnosed PNH with detected proportion of PNH clone cells of at least 1%;
- Patient or patient's family must be willing and able to give written informed consent.
Who cannot join
- Current or previous treatment with a non-eculizumab complement inhibitor;
- Patients in other PNH clinical trials.
- Unable to give written informed consent.
Trial locations and contacts
40 sites in 1 country.
40 sites in 1 country
China40 sites
Research Site
Beijing, Beijing Municipality, 100000
Research Site
Beijing, Beijing Municipality, 100044
Research Site
Changchun, Jilin, 130021
Research Site
Changsha, Hunan, 410005
Research Site
Changsha, Hunan, 410008
Research Site
Chengdu, Sichuan, 610041
Research Site
Chengdu, Sichuan, 610072
Research Site
Guangzhou, Guangdong, 510080
Research Site
Guangzhou, Guangdong, 510180
Research Site
Guangzhou, Guangdong, 510515
Research Site
Guiyang, Guizhou, 550004
Research Site
Hangzhou
Research Site
Harbin, Heilongjiang, 150001
Research Site
Hefei, Anhui, 230002
Research Site
Jinan, Shandong, 250000
Research Site
Kunming, Yunnan, 650034
Research Site
Linyi, Shangdong, 276034
Research Site
Nanchang, Jiangxi, 330006
Research Site
Nanchang, Jiangxi, 330008
Research Site
Nanjing, Jiangsu, 210029
Research Site
Nanning, Guangxi, 530021
Research Site
Nantong, Jiangsu, 226001
Research Site
Qingdao, Shandong, 266000
Research Site
Shanghai, Shanghai Municipality, 200040
Research Site
Shanghai, Shanghai Municipality, 200336
Research Site
Shenyang, Liaoning, 110001
Research Site
Shijiazhuang, Hebei, 050000
Research Site
Taiyuan, Shanxi, 030000
Research Site
Tianjin, Tianjin Municipality, 300020
Research Site
Tianjin, Tianjin Municipality, 300052
Research Site
Tianjin, Tianjin Municipality, 300190
Research Site
Wuhan, Hubei, 430000
Research Site
Wuhan, Hubei, 430030
Research Site
Xi’an, Shanxi, 710061
Research Site
Xi’an, Shanxi, 710068
Research Site
Xuzhou, Jiangsu, 221002
Research Site
Zhengzhou, Henan, 450003
Research Site
Zhengzhou, Henan, 450052
Research Site
Zhengzhou, Henan, 463599
Research Site
Zibo, Shandong, 255090
About this study
Official title: A Real-world, Multi-center, Prospective, Observational Study for Paroxysmal Nocturnal Haemoglobinuria (PNH) in China
As a rare disease listed in the First Catalogue of Rare Diseases in China (National Health Commission of the People's Republic of China, 2019), PNH is poorly studied in China subse-quently leading to the inadequate elucidation of disease characteristics and clinical outcomes. Eculizumab was recently approved by NMPA. The availability of Eculizumab in China pro-vides people living with PNH with a new treatment option that can reduce disease symptoms and prevent the dysregulated complement system from causing further damage. A Phase Ⅳ study is necessary to understand the natural history of disease and the clinical outcomes with different medical interventions.
Read the detailed description
Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare and life-threatening acquired disorder of the pluripotent hematopoietic stem cell and therefore can affect erythrocytes, leukocytes, thrombocytes and probably some endothelial cells. These hematopoietic stem cells have acquired a somatic mutation in the phosphatidylinositol glycan class A (PIG-A) This gene is required for the synthesis of the glycosyl phosphatidyl-inositol (GPI) anchor, which is necessary to attach some proteins to the blood cell membrane. Therefore, a lack of two important complement regulatory proteins is observed on the cell surface: 'decay-accelerating factor' (DAF), also called 'CD55' and 'membrane inhibitor of reactive lysis' (MIRL), also called 'CD59' Thus, red blood cells are more vulnerable to the attack by the complement activation product MAC (complement membrane attack complex). This leads to a complement-mediated intravascular hemolysis. The predisposition of venous thrombosis, hemolytic anemia, complete thrombocytopenia, and thrombosis are the three main characteristics of the disease. Its incidence is not really known but estimated at 0.1~0.6/100 000 per-sons/yr, and prevalence is estimated at 1~4 cases/100,000 persons/yr. PNH was listed in the First Catalogue of Rare Diseases in China, and its incidence was previously reported to be 1/100,000 persons/year, peak onset age 20~40 years.
PNH can be classified into 3 different forms: classical PNH, PNH associated with aplastic anemia (PNH-AA), and subclinical PNH, based on clinical features, bone marrow characteristics, and the size of the mutant clone. The traditional treatment of PNH is still aimed at "protecting" the PNH clone, reducing complement attack and destruction, and alleviating hemolysis with symptomatic supportive therapy. In acute hemolytic episodes, could be administered adrenal glucocorticoids, complemented by cell membrane stabilizers, folic acid, and alkaline drugs. In case of PNH-AA syndrome, treatment with androgens and immunosuppressants may be used; anticoagulation and heparin therapy should be given for the occurrence of thrombosis; other symptomatic supportive treatments include transfusion of red blood cells and platelets if necessary as well as antibacterial drugs in case of infection. Bone marrow transplantation is the only curative therapy for PNH presupposes, but patient need to achieve complete remission with chemotherapy first and a suitable donor is needed.
Besides supportive care, global guidance/consensus also recommend C5 complement inhibitor Eculizumab as a treatment method, and its use could significantly improve 5-year survival rate to 95.5%. Eculizumabis a humanized, first-in-class, anti-C5 antibody that binds with high affinity to C5 and blocks the terminal complement-C5a and C5b-9 formation, reducing the chronic uncontrolled complement activation and its consequences. Eculizumab has been approved for PNH by National Medical Products Administration in August, 2022.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What is being measured
The primary outcome is the main question the study is designed to answer.
Hemolysis：Concentration of LDH changes at each visit from base-line, of all patients
To characterize the progression of PNH
Measured: 12 months
Number and percentage of patients with thrombosis within follow-up
To characterize the progression of PNH
Measured: 12 months
average number of units of packed RBCs transfused per month, of all patients
To characterize the progression of PNH
Measured: 12 months
Number and percentage of patients with renal failure within follow-up
To characterize the progression of PNH
Measured: 12 months
Number and percentage of patients with pulmonary hypertension within follow-up
To characterize the progression of PNH
Measured: 12 months
Secondary outcomes (8)
Number and percentage of patients with each symptom of interest within follow-up
To describe clinical characteristics of patients with PNH in China
Measured: 12 months
Demographics at baseline of all patients
To describe clinical characteristics of patients with PNH in China
Measured: baseline
Number and percentage of patients receiving each type of treatment method at baseline and each visit within follow up, including glucocorticoid, red blood cell transfusion, other supportive treatment, bone marrow transplant, eculizumab, of all patients
To describe the treatment pattern of PNH in China
Measured: 12 months
All serious adverse events (SAEs)of PNH among eculizumab-treated patients
To describe the safety of Eculizumab treatment via Serious Adverse Events
Measured: 12 months
Standard descriptive statistics of pregnancy status
To describe clinical characteristics of patients with PNH in China
Measured: 12 months
Standard descriptive statistics of lactation status
To describe clinical characteristics of patients with PNH in China
Measured: 12 months
Standard descriptive statistics of PNH classification
To describe clinical characteristics of patients with PNH in China
Measured: 12 months
Standard descriptive statistics of flow cytometry results
To describe clinical characteristics of patients with PNH in China
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2023
- Study start10 Nov 2023
- Primary completion3 Dec 2025
- Study completion3 Dec 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 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
- Prospective
- Enrolment
- 724 participants (actual)
- Target duration
- 1 Year
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06154512
- Sponsor's protocol ID
- D7414R00001
- Acronym
- ReWoPNH
- Status last verified
- Sep 2026
- First submitted
- 30 Oct 2023
- Last updated
- 24 Sep 2026
- Results posted
- 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 approved.
Publications
Han B, Liu H, He G, Jia J, Hong M, Weng J, Wang S, Zhang F, Niu T, Ma T, Xu J, Fu R. A real-world, multi-center, prospective, observational study for paroxysmal nocturnal haemoglobinuria (PNH) in China: Baseline characteristics, disease burden and treatment patterns. Ther Adv Hematol. 2026 Sep 4;17:20406207261481224. doi: 10.1177/20406207261481224. eCollection 2026.
PubMed 42707331
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Real-world, Multi-center, Prospective, Observational Study for Paroxysmal Nocturnal Haemoglobinuria (PNH) in China
- ClinicalTrials.gov ID
- NCT06154512
- Acronym / sponsor ID
- ReWoPNH / D7414R00001
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 724 participants (actual)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- 10 Nov 2023 (actual)
- Primary completion
- 3 Dec 2025 (actual)
- Study completion
- 3 Dec 2025 (actual)
- First posted
- 4 Dec 2023 (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.
As a rare disease listed in the First Catalogue of Rare Diseases in China (National Health Commission of the People's Republic of China, 2019), PNH is poorly studied in China subse-quently leading to the inadequate elucidation of disease characteristics and clinical outcomes. Eculizumab was recently approved by NMPA. The availability of Eculizumab in China pro-vides people living with PNH with a new treatment option that can reduce disease symptoms and prevent the dysregulated complement system from causing further damage. A Phase Ⅳ study is necessary to understand the natural history of disease and the clinical outcomes with different medical interventions.
Detailed description
Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare and life-threatening acquired disorder of the pluripotent hematopoietic stem cell and therefore can affect erythrocytes, leukocytes, thrombocytes and probably some endothelial cells. These hematopoietic stem cells have acquired a somatic mutation in the phosphatidylinositol glycan class A (PIG-A) This gene is required for the synthesis of the glycosyl phosphatidyl-inositol (GPI) anchor, which is necessary to attach some proteins to the blood cell membrane. Therefore, a lack of two important complement regulatory proteins is observed on the cell surface: 'decay-accelerating factor' (DAF), also called 'CD55' and 'membrane inhibitor of reactive lysis' (MIRL), also called 'CD59' Thus, red blood cells are more vulnerable to the attack by the complement activation product MAC (complement membrane attack complex). This leads to a complement-mediated intravascular hemolysis. The predisposition of venous thrombosis, hemolytic anemia, complete thrombocytopenia, and thrombosis are the three main characteristics of the disease. Its incidence is not really known but estimated at 0.1~0.6/100 000 per-sons/yr, and prevalence is estimated at 1~4 cases/100,000 persons/yr. PNH was listed in the First Catalogue of Rare Diseases in China, and its incidence was previously reported to be 1/100,000 persons/year, peak onset age 20~40 years.
PNH can be classified into 3 different forms: classical PNH, PNH associated with aplastic anemia (PNH-AA), and subclinical PNH, based on clinical features, bone marrow characteristics, and the size of the mutant clone. The traditional treatment of PNH is still aimed at "protecting" the PNH clone, reducing complement attack and destruction, and alleviating hemolysis with symptomatic supportive therapy. In acute hemolytic episodes, could be administered adrenal glucocorticoids, complemented by cell membrane stabilizers, folic acid, and alkaline drugs. In case of PNH-AA syndrome, treatment with androgens and immunosuppressants may be used; anticoagulation and heparin therapy should be given for the occurrence of thrombosis; other symptomatic supportive treatments include transfusion of red blood cells and platelets if necessary as well as antibacterial drugs in case of infection. Bone marrow transplantation is the only curative therapy for PNH presupposes, but patient need to achieve complete remission with chemotherapy first and a suitable donor is needed.
Besides supportive care, global guidance/consensus also recommend C5 complement inhibitor Eculizumab as a treatment method, and its use could significantly improve 5-year survival rate to 95.5%. Eculizumabis a humanized, first-in-class, anti-C5 antibody that binds with high affinity to C5 and blocks the terminal complement-C5a and C5b-9 formation, reducing the chronic uncontrolled complement activation and its consequences. Eculizumab has been approved for PNH by National Medical Products Administration in August, 2022.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 1 Year
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Study Population: Number of patients (estimate): 1000; Inclusion Criteria 1. Patients of any age; 2. Diagnosed PNH with detected proportion of PNH clone cells of at least 1%; 3. Patient or patient's family must be willing and able to give written informed consent. Exclusion Criteria 1. Current or previous treatment with a non-eculizumab complement inhibitor; 2. Patients in other PNH clinical trials. 3. Unable to give written informed consent.
Key inclusion criteria (3 listed)
- Patients of any age;
- Diagnosed PNH with detected proportion of PNH clone cells of at least 1%;
- Patient or patient's family must be willing and able to give written informed consent.
Key exclusion criteria (3 listed)
- Current or previous treatment with a non-eculizumab complement inhibitor;
- Patients in other PNH clinical trials.
- Unable to give written informed consent.
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
Hemolysis：Concentration of LDH changes at each visit from base-line, of all patients
Time frame: 12 months
To characterize the progression of PNH
Number and percentage of patients with thrombosis within follow-up
Time frame: 12 months
To characterize the progression of PNH
average number of units of packed RBCs transfused per month, of all patients
Time frame: 12 months
To characterize the progression of PNH
Number and percentage of patients with renal failure within follow-up
Time frame: 12 months
To characterize the progression of PNH
Number and percentage of patients with pulmonary hypertension within follow-up
Time frame: 12 months
To characterize the progression of PNH
8 secondary outcomes
Number and percentage of patients with each symptom of interest within follow-up
Time frame: 12 months
Demographics at baseline of all patients
Time frame: baseline
Number and percentage of patients receiving each type of treatment method at baseline and each visit within follow up, including glucocorticoid, red blood cell transfusion, other supportive treatment, bone marrow transplant, eculizumab, of all patients
Time frame: 12 months
All serious adverse events (SAEs)of PNH among eculizumab-treated patients
Time frame: 12 months
Standard descriptive statistics of pregnancy status
Time frame: 12 months
Standard descriptive statistics of lactation status
Time frame: 12 months
Standard descriptive statistics of PNH classification
Time frame: 12 months
Standard descriptive statistics of flow cytometry results
Time frame: 12 months
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 24 Sep 2026.
- Derived from this study: PubMed 42707331Han B, Liu H, He G, Jia J, Hong M, Weng J, Wang S, Zhang F, Niu T, Ma T, Xu J, Fu R. A real-world, multi-center, prospective, observational study for paroxysmal nocturnal haemoglobinuria (PNH) in China: Baseline characteristics, disease burden and treatment patterns. Ther Adv Hematol. 2026 Sep 4;17:20406207261481224. doi: 10.1177/20406207261481224. eCollection 2026.
- CSR Synopsis\_redactedLink provided by the sponsor.
- 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 approved.
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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.
40 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 AstraZeneca at 40 sites in China.
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 3 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 24 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 40 sites in China. 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?
Hemolysis：Concentration of LDH changes at each visit from base-line, of all patients (time frame: 12 months). 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?
On the ClinicalTrials.gov record for NCT06154512, 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 24 Sep 2026.