Study of SGB-9768 in Patients With Paroxysmal Nocturnal Hemoglobinuria

A Multicenter, Randomized, Open-Label Phase II Study to Evaluate the Efficacy and Safety of Multiple Doses of SGB-9768 in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

This is a multicenter, randomized, open-Label Phase II study to evaluate the efficacy and safety of multiple doses of SGB-9768 in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

Inclusion Criteria:

1. Male and female participants ≥ 18 years of age;
2. Diagnosis of PNH confirmed by flow cytometry, with a PNH clone size >10% in granulocytes and/or monocytes;
3. Mean hemoglobin level <100 g/L at screening.
4. Lactate Dehydrogenase (LDH) > 1.5 x Upper Limit of Normal (ULN) at screening.
5. Complement inhibitor-naïve, or previous complement inhibitor therapy discontinued for more than 5 drug half-lives or 3 months before randomization.
6. Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment. If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given.

Exclusion Criteria:

1. Patients with reticulocytes <100x10⁹/L; platelets <30x10⁹/L; neutrophils <0.5x10⁹/L.
2. History of congenital asplenia or splenectomy.
3. Known or suspected hereditary or acquired complement deficiencies/abnormalities.
4. Active or recurrent invasive infections caused by encapsulated bacteria (e.g., Neisseria meningitidis, Streptococcus pneumoniae, or Haemophilus influenzae).
5. Active systemic bacterial, viral, or fungal infection within 14 days before randomization.
6. Evidence or history of tuberculosis infection (except adequately treated inactive tuberculosis with negative screening results).
7. Recurrent chronic infections within 1 year before screening.
8. Positive virology tests indicating active Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), Human Immunodeficiency Virus (HIV), or syphilis infection.