Paroxysmal Nocturnal HemoglobinuriaPhase 3NCT07177859
A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients
A Phase 3 study testing NTQ5082 capsules 200 mg and Eculizumab Injection in people with Paroxysmal Nocturnal Hemoglobinuria, run by Nanjing Chia-tai Tianqing Pharmaceutical at 1 site in China.
Trial 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 NTQ5082 capsules in the treatment of patients with paroxysmal nocturnal hemoglobinuria. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- NTQ5082 capsules 200 mg and Eculizumab Injection
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 78 participants
- Where
- 1 siteChina
- Study dates
- Nov 2025to Nov 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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);
Who cannot join
- 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).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020
fengkui zhang
fengkui zhang
About this study
Official title: 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
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.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- NTQ5082 capsules 200 mg, Eculizumab Injection
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NTQ5082 capsules 200 mgDrug
NTQ5082 capsules 200 mg
Eculizumab InjectionDrug
Eculizumab
| Study group | Role | What they receive |
|---|---|---|
| NTQ5082 | Experimental | This group included 52 subjects, all of whom received NTQ5082 capsules 200mg qd administration Receives: Drug: NTQ5082 capsules 200 mg |
| Eculizumab | Active comparator | This group included 26 subjects, all of whom received Eculizumab Receives: Drug: Eculizumab Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects with hemoglobin (HB) levels ≥120 g/L on at least three of the four measurements between Weeks 18 and 24 in the absence of red blood cell (RBC) transfusions (defined as no RBC transfusions after Week 2 (D15) until Week 24 (D169))
Measured: Weeks 18 to 24
Secondary outcomes (2)
Proportion of subjects who had an increase in hemoglobin level ≥20 g/L from baseline on at least three of the four measurements between Weeks 18 and 24 in the absence of red blood cell transfusions (defined as no red blood cell transfusions after Week 2
Measured: Weeks 18 to 24
Incidence and severity of adverse events
Measured: 24 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2025
- Study startNov 2025 (estimated)
- Primary completionNov 2026 (estimated)
- Study completionNov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Nanjing Chia-tai Tianqing PharmaceuticalThe 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 78 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nanjing Chia-tai Tianqing Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07177859
- Sponsor's protocol ID
- NTQ5082-PNH-301
- Status last verified
- Sep 2025
- First submitted
- 10 Sep 2025
- Last updated
- 17 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07177859
- Acronym / sponsor ID
- NTQ5082-PNH-301
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 78 participants (estimated)
- Lead sponsor
- Nanjing Chia-tai Tianqing PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Nov 2025 (estimated)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Nov 2026 (estimated)
- First posted
- 17 Sep 2025 (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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NTQ5082 | Experimental | Drug: NTQ5082 capsules 200 mgThis group included 52 subjects, all of whom received NTQ5082 capsules 200mg qd administration |
| Eculizumab | Active comparator | Drug: Eculizumab InjectionThis group included 26 subjects, all of whom received Eculizumab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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);
Key exclusion criteria (5 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NTQ5082 capsules 200 mgNTQ5082 capsules 200 mg | Drug | NTQ5082 |
| Eculizumab InjectionEculizumab | Drug | Eculizumab |
Primary outcome
Proportion of subjects with hemoglobin (HB) levels ≥120 g/L on at least three of the four measurements between Weeks 18 and 24 in the absence of red blood cell (RBC) transfusions (defined as no RBC transfusions after Week 2 (D15) until Week 24 (D169))
Time frame: Weeks 18 to 24
2 secondary outcomes
Proportion of subjects who had an increase in hemoglobin level ≥20 g/L from baseline on at least three of the four measurements between Weeks 18 and 24 in the absence of red blood cell transfusions (defined as no red blood cell transfusions after Week 2
Time frame: Weeks 18 to 24
Incidence and severity of adverse events
Time frame: 24 Weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2025.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NTQ5082 capsules 200 mg and Eculizumab Injection in people with Paroxysmal Nocturnal Hemoglobinuria, run by Nanjing Chia-tai Tianqing Pharmaceutical at 1 site 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 2 inclusion and 5 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: opening soon, as of the sponsor's last update on 17 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact yumeng zhou (86-025-85109999, yumeng_zhou@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of subjects with hemoglobin (HB) levels ≥120 g/L on at least three of the four measurements between Weeks 18 and 24 in the absence of red blood cell (RBC) transfusions (defined as no RBC transfusions after Week 2 (D15) until Week 24 (D169)) (time frame: Weeks 18 to 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Sep 2025; AzTrials last refreshed its copy on 8 Nov 2026.