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Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT06764303

A Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients

A Phase 2 study testing NTQ5082 100mg qd and NTQ5082 200mg qd in people with Paroxysmal Nocturnal Hemoglobinuria, run by Nanjing Chia-tai Tianqing Pharmaceutical at 2 sites in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

NTQ5082 capsule is a small molecule CFB factor inhibitor. This study is a multicenter, randomized, open label phase II clinical trial aimed at evaluating the efficacy, safety, and PK/PD characteristics 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 100mg qd and NTQ5082 200mg qd
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
2 sitesChina
Study dates
23 Jan 2025to 8 Jul 2027
Phase
Phase 2
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

  • Age ≥ 18 years old (including lower limit), gender not limited.
  • According to the diagnostic criteria for PNH in the Chinese "Guidelines for the Diagnosis and Treatment of Rare Diseases" (2019 edition), subjects diagnosed with PNH have a red blood cell and/or granulocyte clone level of>10% within the 6 months prior to screening.
  • I have not received complement inhibitor treatment in the past.
  • Laboratory testing (local laboratory) shows that hemoglobin meets one of the following conditions: (1) screening period V1 and V2 (interval ≥ 7 days) hemoglobin<100g/L; (2) If the subject receives red blood cell transfusion treatment for PNH related anemia during the screening period, they must meet the condition of hemoglobin<100g/L at V1;
  • Screening periods V1 and V2 (with an interval of ≥ 7 days) were used to detect blood lactate dehydrogenase (LDH) levels (local laboratory)>1.5 times the upper limit of normal (ULN);
  • Vaccination with ACYW135 meningococcal and pneumococcal vaccines (if previously administered, effective titers should be provided) at least 14 days prior to the initial administration of the study drug (defined as D1); If vaccinated within 14 days before D1, prophylactic antibiotic treatment should be given until at least 14 days after vaccination;
  • Agree to use at least one effective contraceptive measure during sexual intercourse with their partner from the time of signing the informed consent form until 4 weeks after the last administration, and not to participate in sperm or egg donation.
  • Agree to sign the informed consent form and promise to comply with all regulations in the study.

Who cannot join

  • During the screening period, there was laboratory (local laboratory) evidence of bone marrow failure (reticulocyte count<100 × 109/L, platelet count<30 × 109/L, or neutrophil count<0.5 × 109/L).
  • During the screening period, the laboratory (local laboratory) results showed ALT or ALP>3 × ULN and the researcher deemed it unsuitable to participate in the study.
  • Prior to screening, the subject was receiving treatment with the following drugs, and the duration of treatment at the stable dose of the drug did not meet the following conditions: systemic use of corticosteroids for at least 4 weeks (≤ 15mg/day of prednisone or equivalent dose of corticosteroids); Iron supplements, vitamin B12, folic acid or androgens for at least 4 weeks; Vitamin K antagonists (such as warfarin) should be used for at least 4 weeks and the international normalized ratio (INR) should be stable; Low molecular weight heparin and oral anticoagulants (such as aspirin, rivaroxaban, idoxaban, apixaban) for at least 4 weeks; Erythropoietin (ESA), hypoxia inducible factor prolyl hydroxylase inhibitor (HIF-PHI), or immunosuppressant for at least 8 weeks.
  • Previous history of bone marrow/hematopoietic stem cell or solid organ transplantation (such as heart, lung, kidney, liver).
  • Individuals with a history of splenectomy or planning to undergo surgery during the trial period.
  • Previous history of meningococcal infection or active systemic bacterial, viral, or fungal infection within 14 days prior to the first administration of the study drug (defined as D1) (at the discretion of the investigator).
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  • Patients with a history of malignant tumors within the past 5 years before screening, but those who have already been cured of local basal cell carcinoma of the skin, squamous cell carcinoma of the skin, papillary thyroid carcinoma, and cervical carcinoma in situ, are excluded.
  • Known or suspected genetic complement deficiency or primary or severe secondary immunodeficiency.
  • There is a history of clinically significant kidney, heart, liver, lung, and other conditions that the researchers have determined are not suitable for participation in this study, including but not limited to the following: severe uncontrolled hypertension, severe kidney disease (such as eGFR<30 mL/min/1.73m2), advanced heart disease (NYHA IV grade), severe lung disease (such as severe pulmonary arterial hypertension (WHO IV grade), unstable thrombotic events, etc.
  • Suffering from active hepatitis B (HBsAg positive and HBV-DNA>local laboratory detection limit), or hepatitis C (HCV antibody positive and HCV-RNA>local laboratory detection limit); Or tested positive for HIV; Or positive for Treponema pallidum antibody.
  • Have received any type of attenuated live vaccine within the previous 4 weeks of screening, or plan to receive any attenuated live vaccine during the research process.
  • Participated in any other interventional clinical trials (including drug and device clinical trials) within the previous 4 weeks prior to screening.
  • Suspect individuals who are allergic to the investigational drug or any of its components.
  • Pregnant and lactating female subjects.
  • Any medical condition that the researcher determines may affect the patient's participation in the study, may increase significant risks to the safety of the subjects, or other situations that the researcher determines are not suitable for participation in the study.

Trial locations and contacts

2 sites in 1 country.

China2 sites

  • The First Affiliated Hospital, College of Medicine, Zhejiang University

    Hangzhou, Zhejiang, 310012

  • Hematology Hospital of the Chinese Academy of Medical Sciences

    Tianjin, Tianjin Municipality, 250033

Other trials in China

About this study

Official title: A Multicenter, Randomized, Open Label Phase II Clinical Trial Evaluating the Efficacy and Safety of NTQ5082 Capsules in the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria

NTQ5082 capsule is a small molecule CFB factor inhibitor. This study is a multicenter, randomized, open label phase II clinical trial aimed at evaluating the efficacy, safety, and PK/PD characteristics of NTQ5082 capsules in the treatment of patients with paroxysmal nocturnal hemoglobinuria.

Being tested
NTQ5082 100mg qd, NTQ5082 200mg qd
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 100mg qdDrug

    NTQ5082 100mg qd

  • NTQ5082 200mg qdDrug

    NTQ5082 200mg qd

Study groups in NCT06764303
Study groupRoleWhat they receive
100mg qdExperimentalThis group included 12 subjects, all of whom received NTQ5082 capsules 100mg qd administration
Receives: Drug: NTQ5082 100mg qd
200mg qdExperimentalThis group included 12 subjects, all of whom received NTQ5082 capsules 200mg qd administration
Receives: Drug: NTQ5082 200mg qd

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The proportion of subjects whose hemoglobin (Hb) levels continue to increase by ≥ 20 g/L compared to baseline

    Measured: 12 Weeks

Secondary outcomes (18)

  1. The proportion of subjects with Hb levels consistently ≥ 120 g/L

    Measured: 12 Weeks

  2. The proportion of subjects who did not receive blood transfusion

    Measured: 12 Weeks

  3. The number of red blood cell units transfused to the subjects receiving blood transfusion

    Measured: 12 Weeks

  4. Changes in Hb compared to baseline

    Measured: 12 Weeks

  5. Changes in reticulocyte count compared to baseline

    Measured: 12 Weeks

  6. Changes in lactate dehydrogenase (LDH) compared to baseline

    Measured: 12 Weeks

  7. Changes in total/indirect bilirubin compared to baseline

    Measured: 12 Weeks

  8. Changes in conjugated hemoglobin compared to baseline

    Measured: 12 Weeks

  9. Changes in free hemoglobin compared to baseline

    Measured: 12 Weeks

  10. The proportion of subjects with LDH<1.5 × ULN

    Measured: 12 Weeks

  11. Changes in Chronic Disease Treatment Functional Evaluation Fatigue (FACIT-F) Scale Score Compared to Baseline

    Measured: 12 Weeks

  12. Changes in the size of PNH red blood cell clones

    Measured: 12 Weeks

  13. Changes in the deposition of C3 complement protein fragments on PNH red blood cells

    Measured: 12 Weeks

  14. NTQ5082 capsule PD related biomarker levels: complement pathway activity, plasma fragment Bb, and human soluble terminal complement complex (sC5b-9) levels.

    Measured: 12 Weeks

  15. The Plasma concentration of NTQ5082 and metabolite NTQ5082-M in plasma under steady-state conditions (ss)

    Measured: 12 Weeks

  16. The incidence of breakthrough hemolysis in clinical practice.

    Measured: 12 Weeks

  17. The incidence of thromboembolic events.

    Measured: 12 Weeks

  18. Incidence and severity of adverse events

    Measured: 12 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Jan 2025
  2. Study start23 Jan 2025
  3. Primary completion8 Jul 2025
  4. Study completion8 Jul 2027 (estimated)

Resources

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
24 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06764303
Sponsor's protocol ID
NTQ5082-201
Status last verified
Dec 2024
First submitted
24 Dec 2024
Last updated
23 Sep 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing NTQ5082 100mg qd and NTQ5082 200mg qd in people with Paroxysmal Nocturnal Hemoglobinuria, run by Nanjing Chia-tai Tianqing Pharmaceutical at 2 sites 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 8 inclusion and 15 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: under way, not looking for participants, as of the sponsor's last update on 23 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 2 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The proportion of subjects whose hemoglobin (Hb) levels continue to increase by ≥ 20 g/L compared to baseline (time frame: 12 Weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06764303.

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 23 Sep 2025.

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