ClinicalTrials.gov record NCT07177859
A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients
A Multicenter, Randomized, Open-label, Active-controlled Phase III Clinical Trial Evaluating the Efficacy and Safety of NTQ5082 Capsules in the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is a randomized, open-label, active-controlled Phase III trial comparing NTQ5082 capsules with eculizumab in adults with paroxysmal nocturnal hemoglobinuria (PNH). It enrolls adults (≥18 years) with PNH who have not previously received complement inhibitor therapy. The study's main measurements include hemoglobin levels and red blood cell transfusion use between Weeks 18 and 24, and assessment of safety.
What it is studying. The trial evaluates the efficacy and safety of NTQ5082 capsules (200 mg once daily) compared with eculizumab injection in adults with PNH. Key outcomes include achieving specified hemoglobin levels without red blood cell transfusions between Weeks 18 and 24 and the incidence and severity of adverse events.
What the researchers hope to learn. To generate scientific evidence on the efficacy and safety of NTQ5082 capsules compared with eculizumab in treating adults with PNH, including hemoglobin response and adverse event profiles.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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 NTQ5082 capsules in the treatment of patients with paroxysmal nocturnal hemoglobinuria.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- NTQ5082 capsules 200 mg
- Eculizumab Injection
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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.
- 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 criteria
- 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]).
- 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.
- 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.
- 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.
- 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).
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
If eligible, participants are randomized to receive either NTQ5082 capsules (200 mg once daily) or eculizumab injection and will attend regular clinic visits. Visits include blood tests to measure hemoglobin and LDH, recording of any red blood cell transfusions, and monitoring for adverse events. Key outcome measurements occur between Weeks 18 and 24, and safety is assessed through 24 weeks. The trial is open‑label, so treatment assignment is not masked.
How the study is structured. Interventional Phase III study with randomized, parallel assignment and no masking (open-label). Participants are randomly assigned to receive NTQ5082 capsules 200 mg once daily or eculizumab injection. Primary outcomes are assessed between Weeks 18 and 24 and safety is followed through at least 24 weeks.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on paroxysmal nocturnal hemoglobinuria
Important dates
- Study start
- November 2025 (estimated)
- Primary completion
- November 2026 (estimated)
- Study completion
- November 2026 (estimated)
- Target enrollment
- 78 participants (estimated)
- Last updated on registry
- Sep 17, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Nanjing Chia-tai Tianqing Pharmaceutical
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- yumeng zhou 📞 86-025-85109999 ✉ yumeng_zhou@163.com
Participating sites (1)
- Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020 · China
- fengkui zhang
- fengkui zhang
Terms used on this page · explanations by MedicalUpdates.ai
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- active-controlled
- An existing treatment used as the comparison group instead of a placebo, so the study asks whether the new treatment does as well as or better than current care.
- prior to screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- stable dose
- A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
- glucocorticoids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- immunosuppressants
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- investigational drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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