Who may be eligible for the A Long-term Efficacy and Safety of NTQ5082 Capsules trial?

Inclusion Criteria: * 1\) Age ≥ 18 years, regardless of gender. 2) Subjects who have previously received and completed NTQ5082 study treatment and, as determined by the investigator, have benefited from treatment and are likely to benefit from continued NTQ5082 treatment, or subjects in the control group receiving eculizumab. 3\) Subjects who have received ACYW135 group meningococcal and pneumococcal vaccines as required by the previous protocol, and whose vaccine protection period, as determined by the investigator, falls within the treatment duration of this study. 4\) Subjects who agree to use at least one effective contraceptive method during sexual intercourse with their partner from the time they sign the informed consent form until 4 weeks after the last dose, refrain from cryopreservation of sperm or eggs, and refrain from sperm or egg donation. 5\) Subjects who agree to sign the informed consent form and commit to abide by all study regulations. Exclusion Criteria: * 1\) Previous history of bone marrow/hematopoietic stem cell or solid organ transplant (e.g., heart, lung, kidney, liver). 2\) Previous history of splenectomy. 3) Previous history of recurrent invasive infection with encapsulated bacteria (e.g., meningococci, Streptococcus pneumoniae), previous history of systemic anti-tuberculosis treatment, or current tuberculosis infection. 4\) History of malignancy within 5 years prior to screening, excluding cured localized basal cell carcinoma of the skin, squamous cell carcinoma of the skin, papillary thyroid carcinoma, or carcinoma in situ of the cervix. 5\) Known or suspected hereditary complement deficiency or primary or severe secondary immunodeficiency. 6) Patients with a history of clinically significant kidney, heart, liver, or lung disease who are deemed unsuitable for participation by the investigator, including but not limited to severe kidney disease (e.g., eGFR \<30 mL/min/1.73 m²), advanced heart disease (e.g., NYHA class IV), or severe lung disease (e.g., severe pulmonary hypertension (WHO class IV). 7\) Patients suspected of being allergic to the study drug or any component of the study drug. 8\) Pregnant or lactating female subjects. 9) Other conditions deemed unsuitable for participation by the investigator.

ClinicalTrials.gov record NCT07177872

A Long-term Efficacy and Safety of NTQ5082 Capsules

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

A Multicenter, Open-label Study Evaluating the Long-term Efficacy and Safety of NTQ5082 Capsules in the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.

Age18 years old and older

SexAll

LocationChina

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 multicenter, open-label Phase 3 study evaluates long-term safety and efficacy of NTQ5082 capsules (200 mg) in adults with paroxysmal nocturnal hemoglobinuria (PNH). It enrolls adults (age ≥18) who previously completed NTQ5082 treatment or who were in the eculizumab control group; enrolled participants receive NTQ5082. Key measures include incidence and severity of adverse events and hemoglobin outcomes assessed through 96 weeks.

What it is studying. The study is testing the long-term efficacy and safety of NTQ5082 capsules in treating PNH. It measures incidence and severity of adverse events over 96 weeks and tracks hemoglobin outcomes, including the proportion of people with hemoglobin ≥120 g/L without red blood cell transfusion and the change in hemoglobin compared with before NTQ5082 treatment.

What the researchers hope to learn. Scientific goals: to evaluate long-term safety by measuring the incidence and severity of adverse events; to assess the proportion of subjects with hemoglobin (HB) ≥120 g/L without red blood cell (RBC) transfusion at 96 weeks; and to measure the change in hemoglobin level compared to before NTQ5082 treatment.

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, open-label study evaluating the long-term 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

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

Age ≥ 18 years, regardless of gender. 2) Subjects who have previously received and completed NTQ5082 study treatment and, as determined by the investigator, have benefited from treatment and are likely to benefit from continued NTQ5082 treatment, or subjects in the control group receiving eculizumab.
Subjects who have received ACYW135 group meningococcal and pneumococcal vaccines as required by the previous protocol, and whose vaccine protection period, as determined by the investigator, falls within the treatment duration of this study.
Subjects who agree to use at least one effective contraceptive method during sexual intercourse with their partner from the time they sign the informed consent form until 4 weeks after the last dose, refrain from cryopreservation of sperm or eggs, and refrain from sperm or egg donation.
Subjects who agree to sign the informed consent form and commit to abide by all study regulations.

Exclusion criteria

Previous history of bone marrow/hematopoietic stem cell or solid organ transplant (e.g., heart, lung, kidney, liver).
Previous history of splenectomy. 3) Previous history of recurrent invasive infection with encapsulated bacteria (e.g., meningococci, Streptococcus pneumoniae), previous history of systemic anti-tuberculosis treatment, or current tuberculosis infection.
History of malignancy within 5 years prior to screening, excluding cured localized basal cell carcinoma of the skin, squamous cell carcinoma of the skin, papillary thyroid carcinoma, or carcinoma in situ of the cervix.
Known or suspected hereditary complement deficiency or primary or severe secondary immunodeficiency. 6) Patients with a history of clinically significant kidney, heart, liver, or lung disease who are deemed unsuitable for participation by the investigator, including but not limited to severe kidney disease (e.g., eGFR <30 mL/min/1.73 m²), advanced heart disease (e.g., NYHA class IV), or severe lung disease (e.g., severe pulmonary hypertension (WHO class IV).
Patients suspected of being allergic to the study drug or any component of the study drug.
Pregnant or lactating female subjects. 9) Other conditions deemed unsuitable for participation 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.

Participants will take NTQ5082 capsules (200 mg) as the study treatment and attend regular clinic visits for safety monitoring and blood tests. The study tracks adverse events and measures hemoglobin and transfusion status over a follow-up period described through 96 weeks. Participants must sign informed consent, follow vaccination and contraception requirements, and comply with other study rules determined by the investigator.

How the study is structured. Interventional Phase 3, open-label, single-group study. All participants receive NTQ5082 capsules 200 mg and know the treatment (no masking). Safety and efficacy outcomes are followed and measured over 96 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

Clinical trials44 trials listed for paroxysmal nocturnal hemoglobinuriaEvery trial the registry lists for paroxysmal nocturnal hemoglobinuria, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in paroxysmal nocturnal hemoglobinuria researchLatest monthly update: September 2026. Includes: Treatment access. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: June 2026 (estimated)
Primary completion: June 2028 (estimated)
Study completion: June 2028 (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

open-label: No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
control group: The group the study treatment is compared with. It may receive a placebo, an existing treatment (active comparator), a sham procedure, or no intervention.
informed consent form: The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
prior to screening: The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
eGFR: A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
study 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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