A Long-term Efficacy and Safety of NTQ5082 Capsules

NCT07177872 | Not Yet Recruiting Phase 3

• 1 other identifier: NTQ5082-PNH-302
• Study Type: interventional
• Target: 78
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of adverse events

  96 Weeks

Secondary Outcomes (2)

• the proportion of subjects with hemoglobin (HB) levels ≥ 120 g/L without red blood cell (RBC) transfusion

  96 weeks

• the change in hemoglobin level compared to before NTQ5082 capsule treatment without red blood cell transfusion

  96 weeks

Study Arms (1)

NTQ5082

EXPERIMENTAL

NTQ5082

Drug: NTQ5082 capsules 200 mg

Interventions

NTQ5082 capsules 200 mg DRUG

NTQ5082 capsules 200 mg

NTQ5082

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \) 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.

You may not qualify if:

• \) 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.

Sponsors & Collaborators

• Nanjing Chia-tai Tianqing Pharmaceutical: lead

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences ,

Tianjin, Tianjin Municipality, 300020, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Central Study Contacts

yumeng zhou

CONTACT

86-025-85109999; yumeng_zhou@163.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2025
• First Posted: September 17, 2025
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): June 1, 2028
• Last Updated: September 17, 2025

Record last verified: 2025-09

Locations

CN (1)