Actively Recruiting
A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Led by S-INFINITY Pharmaceuticals Co., Ltd · Updated on 2025-05-18
24
Participants Needed
1
Research Sites
78 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a multicenter, randomized, single-blind Phase II trial to evaluate the efficacy and safety of XH-S003 capsules in PNH patients. About 24 PNH patients will be enrolled and randomized to three dose levels and take XH-S003 capsules orally
CONDITIONS
Official Title
A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged 18 years or older
- Weight of at least 40 kg and BMI of at least 18 kg/m2
- Diagnosed with PNH with red blood cell or granulocyte clone levels greater than 10% detected by flow cytometry within 6 months prior to or during screening
- No prior treatment with any complement inhibitor therapy
- Lactate dehydrogenase (LDH) levels greater than 1.5 times the upper limit of normal detected twice during the screening period (interval of 2 to 8 weeks)
- Hemoglobin (Hb) less than 100 g/L at first screening visit with red blood cell transfusion due to PNH-related anemia during screening, or average Hb less than 100 g/L based on two tests during screening (interval of 2 to 8 weeks)
- Vaccinated against Neisseria meningitidis and Streptococcus pneumoniae at least 2 weeks before first administration, or receiving preventive antibiotic treatment if vaccination was less than 2 weeks before first administration
You will not qualify if you...
- Evidence of bone marrow failure during screening (reticulocyte count less than 100 x 10^9/L, platelet count less than 30 x 10^9/L, or neutrophil count less than 0.5 x 10^9/L)
- Receiving other therapies prior to screening without completing required treatment durations: erythropoietin or immunosuppressants (at least 8 weeks), systemic corticosteroids (at least 4 weeks), iron supplements, vitamin B12, or folic acid (at least 4 weeks), anticoagulants including vitamin K antagonists or low molecular weight heparin (at least 4 weeks), hypoxic-inducing factor prolyl hydroxylase inhibitors (HIF-PHI) (at least 8 weeks), or androgens (at least 4 weeks)
- History of bone marrow or hematopoietic stem cell transplantation or solid organ transplantation
- Elevated liver enzymes (ALT, GGT, or ALP greater than 3 times the upper limit of normal) at screening
- Positive HIV antibody, active syphilis, positive hepatitis B surface antigen, active hepatitis C virus infection, or active tuberculosis infection at screening
- Known or suspected immunodeficiency diseases or hereditary complement deficiency
- History of Neisseria meningitidis infection
- Chronic active or recurrent infections within 1 year prior to screening
- Systemic active bacterial, viral (including COVID-19), or fungal infections within 2 weeks prior to first administration
- Body temperature higher than 38°C within 7 days prior to first administration
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
TianJin Medical University General Hospital
Tianjin, China
Actively Recruiting
Research Team
R
Rong Fu
CONTACT
H
Hui Liu
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
SINGLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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