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

Last updated: October 25, 2024
Sponsor: Kira Pharmacenticals (US), LLC.
Overall Status: Active - Not Recruiting

Phase

2

Condition

Proteinuria

Red Blood Cell Disorders

Aplastic Anemia

Treatment

KP104

Clinical Study ID

NCT05476887
KP104-201
  • Ages > 18
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

Initial Treatment Period:

  • Documented diagnosis of PNH confirmed by flow cytometry evaluation of white bloodcells 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 ofScreening.

  • LDH >= 2.0 × upper limit of normal (ULN) at screening.

  • Hemoglobin <= 10.0 g/dL at screening.

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

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

Extension Treatment Period (OLE):

  • Complete the 12-week (weekly dosing) or 13-week (biweekly dosing) Initial TreatmentPeriod per the protocol.

  • Benefited from KP104 treatment and will continue benefiting from KP104 treatment perthe investigator's judgement.

  • Willing to participate in Extension Treatment Period, able to comply with thisprotocol and be available for the entire duration of the study.

Exclusion

Exclusion Criteria:

Initial Treatment Period:

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

  • Treatment of any infection with IV (within 30 days of Screening) or oral (within 14days 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 humanimmunodeficiency 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.

Extension Treatment Period (OLE):

  • Women who are pregnant.

  • Women of childbearing potential and men with sexual partners of childbearingpotential who are not using adequate contraception and who are not willing to useadequate contraception.

  • Any medical condition that, in the opinion of the investigator, may pose a safetyrisk to a participant in this study, or may interfere with study participation.

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design

Total Participants: 35
Treatment Group(s): 1
Primary Treatment: KP104
Phase: 2
Study Start date:
November 25, 2022
Estimated Completion Date:
February 28, 2025

Connect with a study center

  • Peking Union Medical College Hospital

    Beijing,
    China

    Site Not Available

  • Jiangsu Province Hospital

    Nanjing,
    China

    Site Not Available

  • Chinese Academy of Medical Sciences Peking Union Medical College - Institute of Hematology Blood Diseases Hospital

    Tianjin,
    China

    Site Not Available

  • Henan Cancer Hospital

    Zhengzhou,
    China

    Site Not Available

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