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To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104
Sponsor: Kira Pharmacenticals (US), LLC.
Summary
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.
Official title: An Open-label, Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 in Complement Inhibitor-naïve Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
35
Start Date
2022-11-25
Completion Date
2025-02
Last Updated
2024-10-28
Healthy Volunteers
No
Conditions
Interventions
KP104
KP104 intravenously (IV loading + subcutaneous \[SC\] maintenance every week \[QW\] or every 2 weeks \[Q2W\]) will be administered.
Locations (4)
Peking Union Medical College Hospital
Beijing, China
Jiangsu Province Hospital
Nanjing, China
Chinese Academy of Medical Sciences Peking Union Medical College - Institute of Hematology Blood Diseases Hospital
Tianjin, China
Henan Cancer Hospital
Zhengzhou, China