To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104

• Status: Not yet recruiting
• Trial end date: 2024-09-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in complement inhibitor-naïve subjects 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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Kira Pharmacenticals (US), LLC.

Criteria

Inclusion Criteria:

• Documented diagnosis of PNH confirmed by flow cytometry evaluation of white blood cells and red blood cells, with granulocyte or monocyte clone size of >= 10 percent (%) within 6 months of the Screening visit.

• Presence of 1 or more PNH-related signs or symptoms within 3 months of initiation of Screening.

• LDH >= 2.0 × ULN at screening.

• Hemoglobin <= 10.0 g/dL at screening.

• Females of childbearing potential and males must practice effective contraception from Screening until 28 days after the end of study (EOS) visit.

• Females of childbearing potential must have a negative pregnancy test at Screening and within 24 hours prior to dosing of study drug.

Exclusion Criteria:

• Any clinically significant poorly controlled underlying illness other than PNH per discretion of investigators.

• Treatment of any infection with IV (within 30 days of Screening) or oral (within 14 days of Screening) antibiotics, antivirals, or antifungals.

• History of meningococcal infection.

• History of untreated tuberculosis.

• History of splenectomy

• Positive serology for Hepatitis C Virus (HCV) ribonucleic acid (RNA) or human immunodeficiency virus (HIV) at Screening

• History of bone marrow or stem cell transplantation

• Absolute neutrophil count (ANC) <500 cells per microliter (cells/μL)

• Reticulocyte count< 100 x 10^3 cells/μL

• Platelet count< 30,000 cells/μL

• History of systemic autoimmune disease

• Estimated glomerular filtration rate (eGFR) <30 milliliters/minute/1.73 meter square (mL/min/1.73 m^2) calculated by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation NOTE: Other protocol defined Inclusion/Exclusion criteria may apply