Phase
Condition
Proteinuria
Red Blood Cell Disorders
Aplastic Anemia
Treatment
KP104
Clinical Study ID
Ages > 18 All Genders
Study Summary
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
Connect with a study center
Peking Union Medical College Hospital
Beijing,
ChinaSite Not Available
Jiangsu Province Hospital
Nanjing,
ChinaSite Not Available
Chinese Academy of Medical Sciences Peking Union Medical College - Institute of Hematology Blood Diseases Hospital
Tianjin,
ChinaSite Not Available
Henan Cancer Hospital
Zhengzhou,
ChinaSite Not Available
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