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The purpose of this study is to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in complement inhibitor-naïve participants with PNH. The study will be conducted in 2 parts. Part 1 is a dose-selection study to assess escalating doses and varying dose intervals of KP104. Part 2 is a proof-of-concept (POC) study assessing the efficacy of the optimal intravenous (IV) loading dose followed by the optimal maintenance dose and regimen of KP104. Participants who complete the Initial Treatment Period and demonstrate benefit from KP104 will be eligible for a 9-month open-label extension (OLE) treatment period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part 1: Dose escalation Cohort 1 | Experimental | Participants will receive escalating and varying dose intervals of KP104 every week. |
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| Part 1: Dose escalation Cohort 2 | Experimental | Participants will receive escalating and varying dose intervals of KP104 weekly or biweekly. |
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| Part 1: Dose escalation Cohort 3 | Experimental | Participants will receive escalating and varying dose intervals of KP104 weekly or biweekly. |
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| Part 2: Proof-of-concept Cohort 1 | Experimental | Participants will receive escalating and varying dose intervals of KP104 weekly or biweekly. |
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| Open-label extension (OLE) | Experimental | Participants will receive KP104, who benefit from KP104 treatment in Part 1 and 2 |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| KP104 | Drug | KP104 intravenously (IV loading + subcutaneous [SC] maintenance every week [QW] or every 2 weeks [Q2W]) will be administered. |
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| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Number of participants with Dose-limiting toxicities (DLT) | A DLT is defined as any adverse event considered by the investigator to be KP104-related with a severity greater than or equal to (>=) National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 Grade 3 which also represents a shift from baseline clinical status of > 1 NCI CTCAE grade. A hypersensitivity/administration reaction occurring with a severity of Grade 2 despite the use of pre-medications will also be designated as a DLT. | Up to Week 4 |
| Part 2: Percentage of participants with >= 2 grams per deciliter (g/dL) increase in hemoglobin level from Baseline in the absence of transfusion for weekly dosing | Blood samples will be collected for the analysis of increase in hemoglobin levels in the absence of transfusion. | Baseline and at Week 12 |
| Part 2: Percentage of participants with >= 2 g/dL increase in hemoglobin level from Baseline in the absence of transfusion for biweekly dosing | Blood samples will be collected for the analysis of increase in hemoglobin levels in the absence of transfusion. | Baseline and at Week 13 |
| Open-label Extension (OLE): Number of participants reporting Treatment Emergent Adverse Events (TEAEs), treatment-emergent serious adverse events (TESAEs) and AEs of special interest (AESIs) | Up to 9 months |
| Measure | Description | Time Frame |
|---|---|---|
| Part 2: Change from Baseline in serum lactate dehydrogenase (LDH) levels for weekly dosing | Blood samples will be collected for the analysis of serum LDH. | Baseline and at Week 12 |
| Part 2: Change from Baseline in serum LDH levels for biweekly dosing |
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Inclusion Criteria:
Initial Treatment Period:
Extension Treatment Period (OLE):
Exclusion Criteria:
Initial Treatment Period:
Extension Treatment Period (OLE):
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | Beijing | China | ||||
| Jiangsu Province Hospital |
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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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Blood samples will be collected for the analysis of serum LDH.
| Baseline and at Week 13 |
| Part 2: Change from Baseline in hemoglobin level for weekly dosing | Blood samples will be collected for the analysis of hemoglobin level | Baseline and at Week 12 |
| Part 2: Change from Baseline in the hemoglobin level for biweekly dosing | Blood samples will be collected for the analysis of hemoglobin level. | Baseline and at Week 13 |
| Part 2: Change from Baseline in red blood cell (RBC) transfusion dependence for weekly dosing | The RBC transfusion dependence is the difference in the volume of RBC transfusions per month for the 3 months prior to initiation of investigational product versus the volume of RBC transfusions per month for each month on study. | Baseline and at Week 12 |
| Part 2: Change in RBC transfusion dependence for biweekly dosing | The RBC transfusion difference is the difference in the volume of RBC transfusions per month for the 3 months prior to initiation of investigational product versus the volume of RBC transfusions per month for each month on study. | Baseline and at Week 13 |
| Part 1 and 2: Number of participants reporting TEAEs, TESAEs and AESIs | Up to Week 13 |
| Part 1 and 2: Concentration within one hour of end of infusion (CEOI) of KP104 | Up to Week 13 |
| Part 1 and 2: Trough concentration (Ctrough) of KP104 | Up to Week 13 |
| Nanjing |
| China |
| Chinese Academy of Medical Sciences Peking Union Medical College - Institute of Hematology Blood Diseases Hospital | Tianjin | China |
| Henan Cancer Hospital | Zhengzhou | China |
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |