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

A Phase 1 First-in-Human Clinical Trial in Healthy Participants and Patients With C3-Glomerulopathy to Assess Safety, Tolerability, and Pharmacokinetics of CPV-104

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.

Study Overview

• Status: Recruiting
• Conditions: Healthy Adult Participants; C3 Glomerulopathy (C3G)
• Intervention / Treatment: Drug: CPV-104/Placebo; Drug: CPV-104

Detailed Description

CPV-104-101 is a phase 1, first-in-human, dose-escalation, prospective trial, which will be conducted in two parts: Part 1 (Single Ascending Dose with healthy volunteers - SAD-HV) and Part 2 (Multiple Ascending Dose with C3G patients - MAD-C3G). Part 1 is double-blind, randomized, and placebo-controlled, while Part 2 is open-label and single-arm (CPV-104 only).

Following a screening period, 21 healthy volunteers who meet the eligibility criteria will be assigned to one of four cohorts in SAD-HV Part 1. Three healthy volunteers will be assigned to Cohort 1 within SAD-HV Part 1 to receive a single dose of CPV-104. After completion of Cohort 1, 18 healthy volunteers will be randomly allocated within SAD-HV Cohorts 2, 3, and 4 to receive a single dose of either CPV-104 or placebo.

After completion of SAD-HV Part 1, 18 C3G patients will be allocated within MAD-C3G Cohorts 5, 6, and 7 to receive four doses of CPV-104.

All treatments will be administered intravenously by a healthcare professional (HCP). Before dosing in Cohorts 2, 3, 4, 5, 6, and 7 can begin, safety data will be reviewed by an SRC. Safety data from Cohorts 2, 3 and 4 will be blinded for the principal investigators and the medical monitor in the SRC. Safety data will be unblinded for the three independent members of the SRC.

• Study Type: Interventional
• Enrollment (Estimated): 39
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Julia Flugel; Phone Number: +49 761 470 990; Email: jflugel@elevabiologics.com
• Study Contact Backup: Name: Daniela Wittmann; Phone Number: +49 761 470 990; Email: dwittmann@elevabiologics.com

Study Locations

• Austria
  • Vienna, Austria
    • Recruiting
    • Medizinische Universität Wien
• Belgium
  • Brussels, Belgium
    • Recruiting
    • Cliniques Universitaires Saint-luc
• Czechia
  • Prague, Czechia
    • Recruiting
    • Fakultni Thomayerova nemocnice
• France
  • Paris, France
    • Not yet recruiting
    • Hôpital Européen Georges-Pompidou HEGP
  • Toulouse, France
    • Recruiting
    • Centre Hospitalier Universitaire de Toulouse
• Greece
  • Athens, Greece
    • Recruiting
    • Laiko General Hospital of Athens
• Latvia
  • Riga, Latvia
    • Recruiting
    • Pauls Stradins Clinical University Hospital
• Lithuania
  • Vilnius, Lithuania
    • Recruiting
    • Vilnius University Hospital Santaros Klinikos
• Netherlands
  • Amsterdam, Netherlands
    • Recruiting
    • Amsterdam UMC Stichting
• Portugal
  • Lisbon, Portugal
    • Recruiting
    • Hospital Curry Cabral - Centro Hospitalar de Lisboa Central - ULS Sao José
• Spain
  • Barcelona, Spain
    • Not yet recruiting
    • Fundacio Puigvert
  • Madrid, Spain
    • Recruiting
    • Hospital Universitario 12 de Octubre
  • Pamplona, Spain
    • Recruiting
    • Clinica Universidad de Navarra
  • Seville, Spain
    • Recruiting
    • Hospital Universitario Virgen de la Macarena
  • Seville, Spain
    • Recruiting
    • University Hospital Virgen Del Rocio S.L.
• Sweden
  • Huddinge, Sweden
    • Recruiting
    • Karolinska University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria Healthy Volunteers (Part 1 - SAD-HV) :

• 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 exclusion criteria that is outside the reference range for the population being studied may be included only if the investigator, in consultation with the Medical Monitor, agree and document that the finding is unlikely to introduce additional risk factors and will not interfere with the study procedures or results.
• 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.

Exclusion Criteria Healthy Volunteers (Part 1 - SAD-HV) :

• 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).

Inclusion Criteria C3G Patient (Part 2 - MAD-C3G):

• Patient must be at least 18 years old, at the time of consent, and must be able to sign and date the informed consent form (ICF) themselves.
• Patient must have a diagnosis of C3G confirmed by historical renal biopsy.
• Patient must have proteinuria at screening.
• Patient must have stable or worsening renal disease, be on stable and optimized symptomatic treatment, in the opinion of the PI, for at least 30 days prior to screening (treatments may include, but are not limited to, immunosuppressive agents, anti-hypertensives, steroids).
• 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.

Exclusion Criteria C3G Patients (Part 2 - MAD-C3G) :

• 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).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part 1 - SAD-HV: Single ascending dose cohorts in healthy subjects; Healthy volunteer subject cohorts randomized 4:2 receiving a single dose of CPV-104 or placebo. The first cohort consisting of three volunteers will receive CPV-104 only. Dose escalation will occur if CPV-104 or placebo is tolerated.	Drug: CPV-104/Placebo; CPV-104 or Placebo
Experimental: Part 2 - MAD-C3G: Multiple ascending dose cohorts in C3G patients; Patients to receive multiple ascending doses of CPV-104, dosed weekly over 28 days. Dose escalation will occur if CPV-104 is tolerated.	Drug: CPV-104; CPV-104

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of severe drug reactions (severe ADRs) and serious adverse drug reactions (SADRs)	Up to Day 29 for Part 1 - SAD-HV and up to Day 50 for Part 2 - MAD-C3G

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse drug reactions (ADRs)		Up to Day 29 for Part 1 - SAD-HV and up to Day 50 for Part 2 - MAD-C3G
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)	Up to Day 50
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).	Up to Day 50
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)	Up to Day 29 for Part 1 - SAD-HV and up to Day 50 for Part 2 - MAD-C3G
Cmax of CPV-104 after single dose (SAD-HV)	Maximum observed plasma concentration of CPV-104 after administration of a single IV dose.	Up to Day 29
Tmax of CPV-104 after single dose (SAD-HV)	Time to reach maximum observed plasma concentration of CPV-104 after a single IV dose.	Up to Day 29
AUC0-∞ of CPV-104 after single dose (SAD-HV)	Area under the plasma concentration-time curve from zero to infinity after a single IV dose of CPV-104.	Up to Day 29
Terminal half-life (t½) of CPV-104 after single dose (SAD-HV)	Terminal elimination half-life of CPV-104 after a single IV dose.	Up to Day 29
Clearance (CL) of CPV-104 after single dose (SAD-HV)	Total body clearance of CPV-104 from plasma after a single IV dose.	Up to Day 29
Volume of distribution (Vz) of CPV-104 after single dose (SAD-HV)	Volume of distribution of CPV-104 during the terminal phase after a single IV dose.	Up to Day 29
Cmax of CPV-104 after first dose (MAD-C3G)	Maximum observed plasma concentration of CPV-104 after the first weekly dose in the multiple-dose phase.	Up to Day 50
Tmax of CPV-104 after first dose (MAD-C3G)	Time to reach maximum observed plasma concentration of CPV-104 after the first weekly dose in the multiple-dose phase.	Up to Day 50
AUCτ of CPV-104 after first dose (MAD-C3G)	Area under the plasma concentration-time curve over the dosing interval after the first weekly dose of CPV-104.	Up to Day 50
Cmax of CPV-104 after fourth dose (MAD-C3G)	Maximum observed plasma concentration of CPV-104 after the fourth weekly dose in the multiple-dose phase.	Up to Day 50
Tmax of CPV-104 after fourth dose (MAD-C3G)	Time to reach maximum observed plasma concentration of CPV-104 after the fourth weekly dose in the multiple-dose phase.	Up to Day 50
AUCτ of CPV-104 after fourth dose (MAD-C3G)	Area under the plasma concentration-time curve over the dosing interval after the fourth weekly dose of CPV-104.	Up to Day 50
AUC0-∞ of CPV-104 after fourth dose (MAD-C3G)	Area under the plasma concentration-time curve extrapolated to infinity after the fourth weekly dose of CPV-104.	Up to Day 50
Terminal half-life (t½) of CPV-104 after fourth dose (MAD-C3G)	Terminal elimination half-life of CPV-104 after the fourth weekly dose.	Up to Day 50
Clearance (CL) of CPV-104 after fourth dose (MAD-C3G)	Total body clearance of CPV-104 from plasma after the fourth weekly dose.	Up to Day 50
Volume of distribution (Vz) of CPV-104 after fourth dose (MAD-C3G)	Volume of distribution of CPV-104 during the terminal phase after the fourth weekly dose.	Up to Day 50
Cmax of endogenous Factor H after single dose (SAD-HV)	Maximum observed plasma concentration of endogenous Factor H measured at the same pharmacokinetic timepoints as CPV-104 following a single IV dose.	Up to Day 29
Tmax of endogenous Factor H after single dose (SAD)	Time to reach maximum observed plasma concentration of endogenous Factor H following a single IV dose.	Up to Day 29
AUC0-∞ of endogenous Factor H after single dose (SAD)	Area under the plasma concentration-time curve from zero to infinity for endogenous Factor H following a single IV dose.	Up to Day 29
Cmax of endogenous Factor H after first dose (MAD-C3G)	Maximum observed plasma concentration of endogenous Factor H measured at the same pharmacokinetic timepoints as CPV-104 after the first weekly dose in the multiple-dose phase.	Up to Day 8
Tmax of endogenous Factor H after first dose (MAD-C3G)	Time to reach maximum observed plasma concentration of endogenous Factor H after the first weekly dose in the multiple-dose phase.	Up to Day 8
AUCτ of endogenous Factor H after first dose (MAD-C3G)	Area under the plasma concentration-time curve over the dosing interval for endogenous Factor H after the first weekly dose.	Up to Day 8
Cmax of endogenous Factor H after fourth dose (MAD-C3G)	Maximum observed plasma concentration of endogenous Factor H after the fourth weekly dose in the multiple-dose phase.	Up to Day 50
Tmax of endogenous Factor H after fourth dose (MAD-C3G)	Time to reach maximum observed plasma concentration of endogenous Factor H after the fourth weekly dose.	Up to Day 50
AUCτ of endogenous Factor H after fourth dose (MAD-C3G)	Area under the plasma concentration-time curve over the dosing interval for endogenous Factor H after the fourth weekly dose.	Up to Day 50
AUC0-∞ of endogenous Factor H after fourth dose (MAD-C3G)	Area under the plasma concentration-time curve from zero to infinity for endogenous Factor H after the fourth weekly dose.	Up to Day 50
Incidence and titers of anti-drug antibodies (ADA)		Up to Day 29 for Part 1 - SAD-HV and up to Day 50 for Part 2 - MAD-C3G

Other Outcome Measures

Outcome Measure	Time Frame
Change from baseline in estimated glomerular filtration rate (eGFR)	Up to Day 29 for Part 1 - SAD-HV and up to Day 50 for Part 2 - MAD-C3G
Change from baseline in urine protein-to-creatinine ratio (UPCR)	Up to Day 29 for Part 1 - SAD-HV and up to Day 50 for Part 2 - MAD-C3G

Collaborators and Investigators

• Sponsor: eleva GmbH
• Investigators: Principal Investigator: Bernd Jilma, Prof., Medical University of Vienna

Study record dates

Study Major Dates

• Study Start (Actual): June 26, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026

Study Registration Dates

• First Submitted: March 5, 2026
• First Submitted That Met QC Criteria: March 16, 2026
• First Posted (Actual): March 19, 2026

Study Record Updates

• Last Update Posted (Actual): April 21, 2026
• Last Update Submitted That Met QC Criteria: April 16, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: kidney disease; kidney; C3G; CPV-104; Complement System; Factor H
• Additional Relevant MeSH Terms: Urogenital Diseases; Male Urogenital Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Kidney Diseases
• Other Study ID Numbers: CPV-104-101; 2024-517992-19-00 (Ctis)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No