A Clinical Trial to Test the Safety, Tolerability, and How the Body Processes CPV-104 in Healthy People and Patients With C3-Glomerulopathy
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
Sponsoreleva GmbH
About this trial
This study is the first time the new medicine CPV-104 is being tested in people. CPV-104 is designed to regulate the complement system, which can be overactive in diseases such as C3 glomerulopathy (C3G), an ultra-rare kidney disorder.
The study includes healthy adults and adult patients with C3G to assess safety, tolerability, how the body processes the medicine, and whether the immune system reacts to it. The study is divided in two part; in Part 1 (SAD), healthy volunteers receive one IV dose of CPV-104 or a placebo while in Part 2 (MAD) patients with C3G receive four weekly IV doses of CPV-104 (no placebo).
Participants will have close monitoring, including side-effect checks, blood and urine tests, ECGs, vital signs, and blood samples to measure drug levels and antibodies. For those with C3G, researchers will also observe kidney function, although the main goal is safety, not testing effectiveness.
A Safety Review Committee will regularly review results to ensure it is safe to continue to the next dose or study group.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
Participants must be at least 18 years old and no more than 50 years old, at the time of consent, and must be able to sign and date the informed consent form (ICF) themselves.
Participants who are overtly healthy as determined by medical evaluation including medical history, physical examination, laboratory tests, and cardiac monitoring. A participant with a clinical abnormality or laboratory parameter(s) not specifically listed in the
Disqualifiers
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.
Trial design
Design model
Sequential
Treatments tested in this trial
CPV-104/Placebo
DrugCPV-104 or Placebo
CPV-104
DrugCPV-104
Treatment groups
39 Participants
are divided into 2 treatment groupsGroup A: Part 1 - SAD-HV: Single ascending dose cohorts in healthy subjectsExperimental treatment 1 intervention
Group B: Part 2 - MAD-C3G: Multiple ascending dose cohorts in C3G patientsExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Incidence of severe drug reactions (severe ADRs) and serious adverse drug reactions (SADRs)
Time frame
Up to Day 29 for Part 1 - SAD-HV and up to Day 50 for Part 2 - MAD-C3GSecondary outcomes
1
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse drug reactions (ADRs)
Time frame
Up to Day 29 for Part 1 - SAD-HV and up to Day 50 for Part 2 - MAD-C3G2
Change from baseline in patient reported parameters (patients with C3G only)
Change in scores from baseline from the following patient reported outcome instruments: * EuroQol 5 Dimension 5 Level (EQ 5D 5L) * PROMIS® 29+2 Profile * Kidney Disease Quality of Life - Short Form (KDQOL SF) * Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT Fatigue)
Time frame
Up to Day 503
Change from baseline in physician global assessment (patients with C3G only)
Change from baseline in physician assessed global disease severity using a 0-100 scale (0 = no signs of disease, 100 = worst imaginable severity).
Time frame
Up to Day 504
Change from baseline in safety laboratory parameters, physical examinations, vital signs, and 12-lead ECG parameters
Change from baseline in predefined safety assessments including: * Clinical laboratory tests (chemistry, hematology, coagulation, urinalysis) * Physical examination findings (abnormal findings) * Vital signs (blood pressure, pulse, temperature, respiratory rate) * ECG parameters (PR/PQ, QRS, QT/QTc, heart rate)
Time frame
Up to Day 29 for Part 1 - SAD-HV and up to Day 50 for Part 2 - MAD-C3GOther outcomes
1
Change from baseline in estimated glomerular filtration rate (eGFR)
Time frame
Up to Day 29 for Part 1 - SAD-HV and up to Day 50 for Part 2 - MAD-C3G2
Change from baseline in urine protein-to-creatinine ratio (UPCR)
Time frame
Up to Day 29 for Part 1 - SAD-HV and up to Day 50 for Part 2 - MAD-C3G Source ClinicalTrials.gov