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This is a multicenter, randomized, open-label, Phase II clinical study to evaluate the efficacy, safety, and pharmacokinetics of LP-005 injection in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LP-005 900 mg | Experimental | Patients will receive intravenous infusion of LP-005 Injection at a dose of 900 mg once every 4 weeks. |
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| LP-005 1200 mg | Experimental | Patients will receive intravenous infusion of LP-005 Injection at a dose of 1200 mg once every 4 weeks. |
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| LP-005 1500 mg | Experimental | Patients will receive intravenous infusion of LP-005 Injection at a dose of 1500 mg once every 4 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LP-005 Injection | Biological | IV infusion, Q4W |
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| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in serum lactate dehydrogenase (LDH) levels. | Baseline and at Week 12. | |
| Proportion of participants with ≥2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion. | Baseline and at Week 24. |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of participants with ≥2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion. | Baseline and at Week 12 and 48 | |
| Proportion of participants who are transfusion-free. | Baseline to Week 12, 24 and 48 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Guangsheng He | The First Affiliated Hospital with Nanjing Medical University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The First Affiliated Hospital of Nanjing Medical University | Nanjing | China |
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| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| Change from Baseline in the number of red blood cell (RBC) transfusions. | Baseline to Week 24 and 48. |
| Change from Baseline in hemoglobin levels. | Baseline, Week 12, 24 and 48 |
| Proportion of participants achieving hemoglobin levels ≥100 g/L in the absence of transfusion. | Baseline, Week 12, 24 and 48 |
| Proportion of participants with LDH levels normalized. | Baseline, Week 12, 24 and 48 |
| Changes in serum complement hemolytic activity. | Observation from Predose on Day 1 through 337 days post-administration. |
| Change From Baseline in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) | FACIT-F is a 40-item measure that assesses self-reported fatigue and its impact upon daily activities and function. The higher the score, the better the QOL. | Baseline and at Week 12, 36, and 48. |
| Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs). | Baseline up to approximately 13 months. |
| Serum concentrations of LP-005. | Observation from Predose on Day 1 through 337 days post-administration. |
| Number of Participants with Treatment-emergent Anti-Drug Antibodies (ADA) and neutralizing antibodies (Nab) Response to LP-005. | Observation from Predose on Day 1 through 337 days post-administration. |
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |