A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH.

NCT07747090 | Not Yet Recruiting Phase 3

• 1 other identifier: FXS6837-PNH-301
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 1

Brief Summary

An open label study to evaluate the efficacy and safety of FXS6837 capsules compared to eculizumab in naive PNH patients. About 90 PNH patients who are naive to complement inhibitor therapies will be enrolled to take FXS6837 capsules or eculizumab for 24 weeks according to protocol.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcomes (1)

• Proportion of participants with hemoglobin (Hb) level ≥ 120 g/L in at least 3 of 4 measurements between W18 and W24 in the absence of RBC transfusion (defined as no RBC transfusion after W2 through W24)

  24 weeks from Baseline

Secondary Outcomes (10)

• Proportion of participants with an increase from baseline in Hb level of ≥ 20 g/L in at least 3 of 4 measurements between W18 and W24 in the absence of RBC transfusion (defined as no RBC transfusion after Week 2 through Week 24)

  24 weeks from Baseline

• Proportion of subjects without RBC transfusion during the treatment period from Week 2 to Week 24

  24 weeks from Baseline

• Change from baseline in Hb during the treatment period from Week 18 to Week 24

  24 weeks from Baseline

• Change from baseline in reticulocyte count during the treatment period from Week 18 to Week 24

  24 weeks from Baseline

• Percentage change from baseline in lactate dehydrogenase (LDH) during the treatment period from Week 18 to Week 24

  24 weeks from Baseline

Study Arms (2)

FXS6837 Capsules

EXPERIMENTAL

FXS6837 capsule orally taken for 24 weeks

Drug: FXS6837 Capsule

Eculizumab infusion

ACTIVE COMPARATOR

Eculizumab infusion treated for 24 weeks

Biological: Eculizumab infusion

Interventions

FXS6837 Capsule DRUG

FXS6837 capsule orally taken for 24 weeks

FXS6837 Capsules

Eculizumab infusion BIOLOGICAL

Eculizumab infusion treated for 24 weeks

Eculizumab infusion

Eligibility Criteria

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

You may qualify if:

• Male or female subjects aged ≥ 18 years at screening;
• Body weight ≥ 40 kg and body mass index (BMI) ≥ 18 kg/m2 at screening;
• Diagnosis of PNH by the investigator according to the PNH diagnostic criteria, with red blood cell and white blood cell (monocytes or neutrophil) clone levels \> 10% detected by high sensitivity flow cytometry within 6 months before screening or during the screening period;
• PNH patients with no prior treatment involving any complement inhibitors;
• At least two measurements during the screening period (2 to 6 weeks apart) showing LDH \> 1.5 × ULN (multiple measurements are allowed during the screening period);
• Hb meeting one of the following conditions: (1) Hb concentration \< 100 g/L at the first screening visit, and received RBC transfusion therapy due to PNH-related anemia during the screening period; (2) Mean Hb concentration from two measurements during the screening period \< 100 g/L (these two measurements should be 2 to 6 weeks apart; multiple Hb measurements are allowed during the screening period);

You may not qualify if:

• With laboratory evidence of bone marrow failure during screening (reticulocyte count \< 100 × 109/L, or platelet count \< 30 × 109/L, or neutrophil count \< 0.5 × 109/L); Received acute treatment (such as platelet transfusion, granulocyte colony-stimulating factor) for thrombocytopenia or neutropenia within 30 days before screening;
• Participants receiving other therapies before screening that have not reached the following stable treatment durations:
• Erythropoietin for at least 8 weeks
• Immunosuppressants for at least 8 weeks, systemic corticosteroids (≤ 15 mg/day) for at least 4 weeks
• Iron, vitamin B12, or folic acid supplementation for at least 4 weeks
• Anticoagulants: Vitamin K antagonists (such as warfarin) used for at least 4 weeks with a stable international normalized ratio (INR) (as determined by the investigator), low molecular weight heparin, oral anticoagulants such as aspirin, rivaroxaban, apixaban, etc., for at least 4 weeks;
• Hypoxia-inducible factor prolyl hydroxylase inhibitors (HIF-PHI) for at least 8 weeks;
• Androgens for at least 4 weeks;
• History of malignant tumors of any organ or system within 5 years before screening (except local basal cell carcinoma of the skin or carcinoma in situ of the cervix), regardless of whether treatment was received and whether there is evidence of local recurrence or metastasis;
• History of bone marrow/hematopoietic stem cell or solid organ transplantation (such as heart, lung, kidney, liver);
• History of splenectomy or planned surgery during the study period;
• Subjects with significantly abnormal liver function at screening: any parameter of alanine aminotransferase (ALT), γ-glutamyl transpeptidase (GGT), or alkaline phosphatase (ALP) \> 3 × ULN;
• Human immunodeficiency virus (HIV) infection (HIV antibody positive), active syphilis infection, hepatitis B virus infection (hepatitis B surface antigen positive), active hepatitis C virus infection, or active tuberculosis infection at screening;
• Subjects with concurrent systemic major diseases, including but not limited to: advanced heart disease (such as New York Heart Association \[NYHA\] class IV), severe lung disease (such as severe pulmonary hypertension \[WHO class IV\]), active hepatitis, severe kidney disease (estimated glomerular filtration rate eGFR \< 30 mL/min/1.73 m2 or chronic kidney disease \[CKD\] stage 4 or dialysis patients), unstable thrombosis, active gastrointestinal bleeding, other hematologic diseases (such as chronic anemia unrelated to PNH), and deemed unsuitable for study participation by the investigator;
• Known or suspected by investigators to have immunodeficiency diseases or hereditary complement deficiency;

Sponsors & Collaborators

• Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.: lead

Study Sites (1)

TianJin Medical University General Hospital

Tianjin, Tianjin Municipality, China

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

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

Study Officials

• Rong Fu

  Tianjin Medical University General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Rong Fu

CONTACT

+86 13920350233; furong8369@tmu.edu.cn

Hui Liu

CONTACT

+86 13821113189; liuhui8003@qq.com

Study Design

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

Study Record Dates

• First Submitted: July 22, 2026
• First Posted: August 5, 2026
• Study Start (Estimated): December 10, 2026
• Primary Completion (Estimated): May 5, 2028
• Study Completion (Estimated): July 31, 2028
• Last Updated: August 5, 2026

Record last verified: 2026-07

Locations

CN (1)