A Real-world, Multi-center, Prospective, Observational Study for PNH in China
ReWoPNH
1 other identifier
observational
724
1 country
40
Brief 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. 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Nov 2023
Typical duration for all trials
40 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 30, 2023
CompletedStudy Start
First participant enrolled
November 10, 2023
CompletedFirst Posted
Study publicly available on registry
December 4, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 3, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
December 3, 2025
CompletedDecember 18, 2025
December 1, 2025
2.1 years
October 30, 2023
December 17, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (5)
Hemolysis：Concentration of LDH changes at each visit from base-line, of all patients
To characterize the progression of PNH
12 months
Number and percentage of patients with thrombosis within follow-up
To characterize the progression of PNH
12 months
average number of units of packed RBCs transfused per month, of all patients
To characterize the progression of PNH
12 months
Number and percentage of patients with renal failure within follow-up
To characterize the progression of PNH
12 months
Number and percentage of patients with pulmonary hypertension within follow-up
To characterize the progression of PNH
12 months
Secondary Outcomes (8)
Number and percentage of patients with each symptom of interest within follow-up
12 months
Demographics at baseline of all patients
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
12 months
All serious adverse events (SAEs)of PNH among eculizumab-treated patients
12 months
Standard descriptive statistics of pregnancy status
12 months
- +3 more secondary outcomes
Other Outcomes (5)
Concentration of LDH changes at each visit from base-line of PNH among eculizumab-treated patients
12 months
Number and percentage of patients with thrombosis within follow up among eculizumab-treated patients
12 months
average number of units of packed RBCs transfused per month within 12 months v
12 months
- +2 more other outcomes
Eligibility Criteria
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.
You may qualify if:
- 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.
You may not qualify if:
- Current or previous treatment with a non-eculizumab complement inhibitor;
- Patients in other PNH clinical trials.
- Unable to give written informed consent.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AstraZenecalead
Study Sites (40)
Research Site
Hefei, Anhui, 230002, China
Research Site
Beijing, Beijing Municipality, 100000, China
Research Site
Beijing, Beijing Municipality, 100044, China
Research Site
Guangzhou, Guangdong, 510080, China
Research Site
Guangzhou, Guangdong, 510180, China
Research Site
Guangzhou, Guangdong, 510515, China
Research Site
Nanning, Guangxi, 530021, China
Research Site
Guiyang, Guizhou, 550004, China
Research Site
Shijiazhuang, Hebei, 050000, China
Research Site
Harbin, Heilongjiang, 150001, China
Research Site
Zhengzhou, Henan, 450003, China
Research Site
Zhengzhou, Henan, 450052, China
Research Site
Zhengzhou, Henan, 463599, China
Research Site
Wuhan, Hubei, 430000, China
Research Site
Wuhan, Hubei, 430030, China
Research Site
Changsha, Hunan, 410005, China
Research Site
Changsha, Hunan, 410008, China
Research Site
Nanjing, Jiangsu, 210029, China
Research Site
Nantong, Jiangsu, 226001, China
Research Site
Xuzhou, Jiangsu, 221002, China
Research Site
Nanchang, Jiangxi, 330006, China
Research Site
Nanchang, Jiangxi, 330008, China
Research Site
Changchun, Jilin, 130021, China
Research Site
Shenyang, Liaoning, 110001, China
Research Site
Jinan, Shandong, 250000, China
Research Site
Qingdao, Shandong, 266000, China
Research Site
Zibo, Shandong, 255090, China
Research Site
Linyi, Shangdong, 276034, China
Research Site
Shanghai, Shanghai Municipality, 200040, China
Research Site
Shanghai, Shanghai Municipality, 200336, China
Research Site
Taiyuan, Shanxi, 030000, China
Research Site
Xi’an, Shanxi, 710061, China
Research Site
Xi’an, Shanxi, 710068, China
Research Site
Chengdu, Sichuan, 610041, China
Research Site
Chengdu, Sichuan, 610072, China
Research Site
Tianjin, Tianjin Municipality, 300020, China
Research Site
Tianjin, Tianjin Municipality, 300052, China
Research Site
Tianjin, Tianjin Municipality, 300190, China
Research Site
Kunming, Yunnan, 650034, China
Research Site
Hangzhou, China
Biospecimen
only collect data from patients' medical history, do not collect patient biospecimen directly.
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Target Duration
- 1 Year
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 30, 2023
First Posted
December 4, 2023
Study Start
November 10, 2023
Primary Completion
December 3, 2025
Study Completion
December 3, 2025
Last Updated
December 18, 2025
Record last verified: 2025-12
Data Sharing
- IPD Sharing
- Will share
- Time Frame
- AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA/PhRMA Data-Sharing Principles. For details of our timelines, please refer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
- Access Criteria
- When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. A Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
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.