Currently Enrolling
Interventional

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.

Currently Enrolling
Interventional

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.

Barcelona, Spain (Fundació Puigvert)
Frequently Asked Questions

Nephrotic Syndrome is not a disease itself, but rather a group of signs and symptoms that result from damage in the part of the kidney that filters blood (glomeruli).

Common symptoms include:

  • Foamy urine (called proteinuria) caused by protein “spilling” into the urine
  • Severe swelling in parts of the body, most noticeably around the eyes, hands, feet, and abdomen (called edema)
  • Weight gain due to a buildup of extra fluid
  • Fatigue
  • Loss of appetite
  • Low levels of protein in the blood (hypoalbuminemia)
  • Higher than normal fat and cholesterol levels in the blood (hyperlipidemia)

Nephrotic Syndrome can typically be diagnosed with a urine test.

Nephrotic Syndrome can be “primary” or “secondary” in nature.

Diseases that affect only the kidneys are called primary causes of Nephrotic Syndrome. Doctors often call these diseases “idiopathic,” which means that they arise from an unknown cause. Some of these diseases include:

  • Minimal Change Disease (MCD) – most common in children
  • Focal Segmental Glomerulosclerosis (FSGS)
  • Membranous Nephropathy (MN) – most common in adults
  • IgA Nephropathy (IgAN)

Secondary Nephrotic Syndrome is caused by an underlying, systemic condition like diabetes, lupus, HIV, and others.

The Kidney Health Gateway is a website owned and operated by NephCure Kidney International. The purpose of this website is to help patients with rare forms of primary Nephrotic Syndrome get connected to expert care and cutting-edge treatment options. By answering a few questions about you or your loved one’s condition, we can provide you with a list of clinical trials and/or expert doctors in your area.

If you have additional questions, please visit NephCure.org or email Info@NephCure.org.

 

See other frequently asked questions
Did you know that some forms of kidney disease can be genetic?Researchers are continually discovering genetic causes of Nephrotic Syndrome.

Learn more about genetic causes of kidney disease and find out if you may be affected.