Ignite Creation Date:
2024-05-06 @ 7:51 PM
Last Modification Date:
2024-10-26 @ 3:15 PM
Study NCT ID:
NCT06154512
Status:
RECRUITING
Last Update Posted:
2024-05-29
First Post:
2023-10-30
Brief Title:
A Real-world Multi-center Prospective Observational Study for PNH in China
Sponsor:
AstraZeneca
Organization:
AstraZeneca
Study Overview
Official Title:
A Real-world Multi-center Prospective Observational Study for Paroxysmal Nocturnal Haemoglobinuria PNH in China
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ReWoPNH
Brief Summary:
As a rare disease listed in the First Catalogue of Rare Diseases in China National Health Commission of the Peoples 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 0106100 000 per-sonsyr and prevalence is estimated at 14 cases100000 personsyr PNH was listed in the First Catalogue of Rare Diseases in China and its incidence was previously reported to be 1100000 personsyear peak onset age 2040 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 guidanceconsensus also recommend C5 complement inhibitor Eculizumab as a treatment method and its use could significantly improve 5-year survival rate to 955 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
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 guidanceconsensus also recommend C5 complement inhibitor Eculizumab as a treatment method and its use could significantly improve 5-year survival rate to 955 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None