Clinical Research Directory

Inclusion and Exclusion Criteria for Groups 1-4 * Participants voluntarily participate in this clinical study, and voluntarily sign the ICF; * BMI ≥ 18.0 kg/m2; male or female; 18 to 75 years of age; * Confirmed diagnosis of PNH by clinical manifestation and flow cytometry; granulocyte clone size ≥ 10%; * Presence of one or more of PNH-related signs or symptoms within 3 months prior to screening; * Hb \< 100 g/L; * LDH value \> 1.5 × ULN; * One of the following criteria for prior drug therapy for PNH must be met: 1. Having never received any complement inhibitor therapy; 2. Having received C5, C3, or CFB complement inhibitors and having discontinued the complement inhibitor for more than 5 half-lives or 3 months prior to screening; * Participants are willing to receive meningococcal vaccine and pneumococcal vaccine at least 14 days prior to dosing. Inclusion and Exclusion Criteria for Groups 5 * Participants voluntarily participate in this clinical study, and voluntarily sign the ICF; * BMI ≥ 18.0 kg/m2; male or female; 18 to 75 years of age; * Confirmed diagnosis of PNH by clinical manifestation and flow cytometry; granulocyte clone size ≥ 10%; * Participants who have been on a stable dose and interval of a C5 complement inhibitor (approved locally) for at least 3 months prior to the first dose of VSA012; * Within the 3 months prior to screening, have a documented Hb level of \< 105 g/L while on C5 complement inhibitor therapy; * Hb \< 105 g/L; * Participants are willing to receive meningococcal vaccine and pneumococcal vaccine at least 14 days prior to dosing. Exclusion Criteria for Groups 1-4 * History of hypersensitivity to VSA012 or its excipients; * Use of any complement inhibitors within 3 months prior to screening * Use of any targeted small interfering RNA (siRNA) within 18 months prior to screening, or any antisense oligonucleotide molecule within 6 months prior to screening; * Supportive care for PNH does not meet the stable-dose requirements: * Participants have infections; * Laboratory tests meet the following criteria: 1. Reticulocyte count \< 100 × 109/L; 2. Platelet count \< 30 × 109/L; 3. Neutrophil count \< 0.5 × 109/L; 4. Creatinine clearance \< 30 mL/min (calculated by the Cockcroft-Gault formula); Exclusion Criteria for Groups 5 * History of hypersensitivity to VSA012 or its excipients; * Use of any targeted small interfering RNA (siRNA) within 18 months prior to screening, or any antisense oligonucleotide molecule within 6 months prior to screening; * Supportive care for PNH does not meet the stable-dose requirements: * Participants have infections; * History of splenectomy; * Previous suspected/confirmed hereditary complement deficiency; * History of recurrent invasive infections with capsular bacteria, e.g., meningococcus or pneumococcus; * Laboratory tests meet the following criteria: 1. Reticulocyte count \< 100 × 109/L; 2. Platelet count \< 30 × 109/L; 3. Neutrophil count \< 0.5 × 109/L; 4. Creatinine clearance \< 30 mL/min (calculated by the Cockcroft-Gault formula);