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Interventional Study
a clinical trial in which a new drug or device is used to measure a specific outcome.
VALIANT
Phase 3 Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients with C3 Glomerulapathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
Brief Description
The purpose of this study is to assess the efficacy and safety of twice weekly pegcetacoplan compared to placebo in patients with C3G or IC-MPGN, on the basis of a reduction in urinary protein levels.
Site Name
Policlinico Sant' Orsola-Malpighi
Bolognia, Italy
Estimated enrollment
Approximately 90
Estimated end date
March 2025
Interventional Study
a clinical trial in which a new drug or device is used to measure a specific outcome.
VALIANT
Phase 3 Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients with C3 Glomerulapathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
Brief Description
The purpose of this study is to assess the efficacy and safety of twice weekly pegcetacoplan compared to placebo in patients with C3G or IC-MPGN, on the basis of a reduction in urinary protein levels.
Trial is for people with
Confirmed C3G or IC-MPGN, with or without prior renal transplantation. In addition, participants must:
• Be at least 12 years old
• Be diagnosed with C3G or IC-MPGN.
• Have significant proteinuria (protein in the urine) confirmed by urine collections during the screening process
• Have normal to moderately impaired kidney function, confirmed during the screening process
• Weigh 30kg to 100kg (66 – 220 pounds)
• Be willing to use contraception during and for a period after the study
Study Goal
The VALIANT study will assess whether pegcetacoplan, an investigational targeted C3 inhibitor, has an effect on the damage to kidney function that is caused by build-up of the complement protein C3 in the kidney.
What is involved for the Patient?
After completing the screening period to confirm that the patient is able to participate in the study, individuals will be randomly assigned (randomized) to one of two treatment groups.
• Group 1: Will receive pegcetacoplan for the 12-month treatment period.
• Group 2: Will receive placebo for the first 6 months of the treatment period, and then receive pegcetacoplan for the remaining 6 months of the treatment period.
The placebo medication looks like the investigational medication but has no medically active ingredients. There is an equal chance (50%) of being assigned to Group 1 or Group 2 at the start of the study treatment period.
Study participants will not know their study treatment group assignment and they will not have the option of choosing their group.
Study participants who complete the 1-year study treatment period may enter a long-term extension study where they will continue receiving the investigational medication. Participants will enter an 8-week follow-up period after their last dose of the study medication.
Study participants will have approximately 14 in-clinic visits during the 1-year study treatment period and 2 visits during the 8-week follow-up period.
Main study assessments and procedures include, but are not limited to:
• Urine samples, including both samples collected at study visits and samples collected at home
• Adults only: Kidney biopsies (where possible, these will be combined with standard care biopsies)
• Blood samples
• Questionnaires
• Measurements of heart function (ECG)
• Chest X-rays
• Physical examinations
• Completion of an electronic diary to record dosing with study medication and home urine collections
About the drug or intervention
Pegcetacoplan is an investigational, targeted C3 inhibitor. It is administered via a needle, under the skin (subcutaneous), twice weekly at home. Investigational means that pegcetacoplan is not approved by any health authority for the treatment of C3G or IC-MPGN.
