Status:
NOT_YET_RECRUITING
A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients
Lead Sponsor:
Nanjing Chia-tai Tianqing Pharmaceutical
Conditions:
Paroxysmal Nocturnal Hemoglobinuria
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
NTQ5082 capsules are a small molecule CFB inhibitor. This study is a multicenter, randomized, open-label, active-controlled Phase III clinical trial designed to evaluate the efficacy and safety of NTQ...
Eligibility Criteria
Inclusion
- \- 1) Age ≥ 18 years, regardless of gender. 2) Subjects diagnosed with PNH according to the PNH diagnostic criteria in the Chinese "Guidelines for the Diagnosis and Treatment of Rare Diseases" (2019 edition), with PNH erythrocyte and/or granulocyte clone levels \> 10% within 6 months prior to screening or during the screening period.
- 3\) PNH patients who have not previously received complement inhibitor therapy. 4) Laboratory test (central laboratory) hemoglobin meets one of the following conditions: (1) The average hemoglobin value during screening period V1 and V2 (interval ≥ 7 days) is \<100g/L; (2) If the subject receives red blood cell transfusion for PNH-related anemia during the screening period, the hemoglobin value at V1 must be \<100g/L; 5) Blood lactate dehydrogenase (LDH) (central laboratory) is \>1.5×upper limit of normal (ULN) during screening period V1 and V2 (interval ≥ 7 days);
Exclusion
- 1\) During the screening period, laboratory (local laboratory) results show bone marrow failure (reticulocyte count \<100×109/L, or platelet count \<30×109/L \[must not have received platelet transfusion within 7 days prior to the laboratory test\], or neutrophil count \<0.5×109/L \[must not have received short-acting granulocyte colony-stimulating factor within 14 days or long-acting granulocyte colony-stimulating factor within 28 days prior to the laboratory test\]).
- 2\) During the screening period, laboratory (local laboratory) results show alanine aminotransferase (ALT), alkaline phosphatase (ALP), or gamma-glutamyltransferase (GGT) \>3×ULN and the investigator determines that the patient is not suitable for study participation.
- 3\) Patients were receiving any of the following medications before screening, and the duration of treatment at a stable dose of the drug: (1) systemic glucocorticoids for less than 4 weeks; or prednisone or equivalent dose of glucocorticoids at a dose of \>15 mg/day; (2) iron, vitamin B12, folic acid, or androgens for less than 4 weeks; (3) vitamin K antagonists (such as warfarin) with a stable international normalized ratio (INR) for less than 4 weeks; (4) low molecular weight heparin, oral anticoagulants (such as aspirin, rivaroxaban, edoxaban, apixaban) for less than 4 weeks; (5) erythropoietin (ESA), hypoxia-inducible factor prolyl hydroxylase inhibitors (HIF-PHI), or immunosuppressants (including but not limited to cyclosporine, tacrolimus, mycophenolate mofetil or mycophenolic acid, cyclophosphamide, methotrexate, etc.) for less than 8 weeks.
- 4\) Previous history of bone marrow/hematopoietic stem cell or solid organ transplantation (such as heart, lung, kidney, liver). 5) Patients with a history of splenectomy or planned surgery during the trial.
- 6\) Patients with a history of recurrent invasive infection with encapsulated bacteria (such as meningococci, Streptococcus pneumoniae, etc.), a history of systemic anti-tuberculosis treatment or current tuberculosis infection, or a history of active systemic bacterial, viral, or fungal infection within 14 days prior to the first administration of the investigational drug (defined as D1) (as determined by the investigator).
Key Trial Info
Start Date :
November 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
November 1 2026
Estimated Enrollment :
78 Patients enrolled
Trial Details
Trial ID
NCT07177859
Start Date
November 1 2025
End Date
November 1 2026
Last Update
September 17 2025
Active Locations (1)
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1
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, China, 300020