A Clinical Trial to Test the Safety, Tolerability, and How the Body Processes CPV-104 in Healthy People and Patients With C3-Glomerulopathy

Exclusion Criteria44

• Body weight within 50 kg for male/ 45 kg for female to 110 kg and BMI within the range 18 - 32 kg/m2 (inclusive).
• Childbearing potential (CBP) participants should agree to use a highly effective method of contraception throughout the study and for 90 days after the last dose of the IMP.
• CBP participants should agree not to donate oocytes or freeze for future use for the purposes of assisted reproduction during the study and for a period of 90 days after the last dose of the IMP. Male participants should agree not to donate sperm or freeze sperm for future use for the purposes of assisted reproduction during the study and for a period of 90 days after the last dose of the IMP.
• CBP participants should have a negative pregnancy test at screening.
• Participant provides written informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
• Participant has a history of clinically significant disorders or diseases affecting the endocrine, gastrointestinal, cardiovascular, hematological, liver, immune, kidney, respiratory, reproductive, or neurological systems (such as stroke or epilepsy). Participants with a history of minor medical issues may be considered for the study at the investigator's discretion.
• Participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the study participant's ability to participate in this study.
• Participant has a recent history of febrile illness or other evidence of a clinically significant active infection, within 14 days prior to screening.
• Participant has a history of severe allergies (e.g. to medications, food, or latex) or has experienced an anaphylactic reaction to food or medicine.
• Participant has a known hypersensitivity to any components of the IMP as stated in this protocol.
• Alanine transaminase (ALT) or Aspartate aminotransferase (AST) \>1.5 x upper limit of normal (ULN).
• Total Bilirubin \>1 x ULN, \> 1.5 x ULN if Gilbert's syndrome.
• Current or chronic history of liver disease or known hepatic or biliary abnormalities (except for Gilbert's syndrome or asymptomatic gallstones).
• Participant has received any complement modifying treatment within 6 months prior to the first dosing day.
• Exposure to more than four new chemical entities within 12 months prior to the first dosing day.
• Participation in any other clinical study with a medical device or investigational medicinal product concurrently or within 5 half-life times or 3 months before the first dosing day (whichever is longer).
• Presence of a QTc interval \>450 ms for males or \>460 ms for females, a history of risk factors for Torsades de Pointes (such as heart failure, cardiomyopathy, or a family history of long QT syndrome), uncorrected hypokalemia or hypomagnesemia, or concurrent use of medications known to prolong the QT/QTc interval.
• Participant is an employee of the sponsor or an employee or relative of the investigator.
• Participant is unable to comply with the protocol (e.g. clinically relevant medical condition making implementation of the protocol difficult, unstable social situation, or otherwise unlikely to complete the study) or is, in the opinion of the investigator, otherwise unsuited for the study.
• Participant has made a blood donation or blood products within 90 days prior to Baseline or plans to donate blood during the study.
• Participant has an alcohol consumption of more than 21 units (males) or 14 units (females) of alcohol per week. One unit is equivalent to 8 g of alcohol: a half pint (240 mL) of beer, 1 glass (125 mL) of wine, or 1 (25 mL) measure of spirits).
• Study participant has a high consumption of caffeine- or other xanthine-containing products (≥300 mg of caffeine- or xanthine-equivalent per day) (1 cup of coffee ≈ 100 mg of caffeine; 1 cup of tea ≈ 30 mg of caffeine; 1 glass of cola ≈ 20 mg of caffeine).
• Patient has a history of clinically significant disorders or diseases affecting the endocrine, gastrointestinal, cardiovascular (including thromboembolic events like deep vein thrombosis, pulmonary embolism, known coagulopathy), hematological, liver, immune, kidney, respiratory, reproductive, or neurological systems (such as stroke or epilepsy). Participants with a history of minor medical issues may be considered for the study at the investigator's discretion.
• Patient has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the study participant's ability to participate in this study.
• Patient had a recent history of febrile illness or other evidence of a clinically significant active infection, within 14 days prior to screening.
• Patient has a history of severe allergies (e.g. to medications, food, or latex) or has experienced an anaphylactic reaction to food or medicine.
• Patient has a known hypersensitivity to any components of the IMP as stated in this protocol.
• Patient had evidence of monoclonal gammopathy of unclear significance, infections, malignancy, autoimmune diseases, or other conditions to which C3G is secondary.
• Patient with other renal diseases that would interfere with interpretation of the study.
• Patient is receiving renal replacement therapy.
• Patient is receiving or planned for receiving plasmapheresis.
• Patient had a major organ transplant (e.g. heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant.
• Patient had a history or presence of any clinically relevant co-morbidities (e.g. advanced cardiac disease (NYHA class 4), severe pulmonary arterial hypertension (WHO class 4).
• Alanine transaminase (ALT) or Aspartate aminotransferase (AST) \>2 x upper limit of normal (ULN).
• Total Bilirubin \>1 x ULN, \> 1.5 x ULN if Gilbert's syndrome.
• Current or chronic history of liver disease, or known hepatic or biliary abnormalities (except for Gilbert's syndrome or asymptomatic gallstones).
• Patient has received any complement modifying treatment within 6 months prior to the first dosing day.
• Patient in any other clinical study with a medical device or investigational medicinal product concurrently or within 5 half-life times or 3 months before study start (whichever is longer).
• Presence of a QTc interval \>450 ms for males or \>460 ms for females, a history of risk factors for Torsades de Pointes (such as heart failure, cardiomyopathy, or a family history of long QT syndrome), uncorrected hypokalemia or hypomagnesemia, or concurrent use of medications known to prolong the QT/QTc interval.
• Participant is an employee of the sponsor or an employee or relative of the investigator.
• Participant is unable to comply with the protocol (e.g. clinically relevant medical condition making implementation of the protocol difficult, unstable social situation, or otherwise unlikely to complete the study) or is, in the opinion of the investigator, otherwise unsuited for the study.
• Participant has made a blood donation or blood products within 90 days prior to Baseline or plans to donate blood during the study.
• Participant has an alcohol consumption of more than 21 units (males) or 14 units (females) of alcohol per week. One unit is equivalent to 8 g of alcohol: a half pint (\~240 mL) of beer, 1 glass (125 mL) of wine, or 1 (25 mL) measure of spirits).
• Study participant has a high consumption of caffeine- or other xanthine-containing products (≥300 mg of caffeine- or xanthine-equivalent per day) (1 cup of coffee ≈ 100 mg of caffeine; 1 cup of tea ≈ 30 mg of caffeine; 1 glass of cola ≈ 20 mg of caffeine).