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Enrollment Closed
Interventional

APPEAR-C3G

Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy. (APPEAR-C3G)

Brief Description

The study is designed as a multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in complement 3 glomerulopathy.

Trial Physician / Study Coordinator

Richard Lafayette

Email Phone
Site Name

Stanford Univ School of Medicine
300 Pasteur Drive Suite A175 Stanford, California, 94305-5616

Sponsor

Novartis Pharmaceuticals

Study Drug

LNP023/iptacopan

Estimated enrollment

68 patients globally

Estimated end date

August 2023

If there is not a site for a clinical trial nearby, you can ask the study team about the possibility of travel reimbursements (i.e., paying you back for your travel costs). Alternatively, you can ask about the possibility of participating from home.
Enrollment Closed
Interventional

APPEAR-C3G

Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy. (APPEAR-C3G)

Brief Description

The study is designed as a multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in complement 3 glomerulopathy.

Trial is for people with

C3 Glomerulopathy (C3G)

Study Goal

The purpose of this study is to evaluate the efficacy and safety of iptacopan compared to placebo and standard of care in patients with native C3G.

What is involved for the Patient?

Patients will be asked to attend a screening visit to assess eligibly. Medical history will be collected. The study team will collect biosamples (blood, urine, biopsy tissue). Visits may be in conjunction with normally scheduled appointments, but additional study visits may be required. If eligible, a patient will receive study medication or placebo on top of standard of care 6 months followed by active study medication for another 6 months and be routinely assessed for safety and efficacy.

About the drug or intervention

LNP023/iptacopan is an investigational drug that we are studying as a potential targeted treatment for people with C3G and other autoimmune diseases. The term "investigational" means that it is not currently approved for use by the general public. LNP023/iptacopan is thought to block a process in the body known to cause inflammation and kidney damage in people with C3G and other autoimmune diseases. The APPEAR-C3G trial will study the effects of LNP023/iptacopan on reducing inflammation and kidney damage in people with C3G.

Stanford, CA
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.

Clinical studies are any studies that involve people. There are two main types: observational and interventional. In an observational trial, researchers track health outcomes over time in groups of participants to look for patterns that help us better understand a disease. In an interventional trial, participants receive specific interventions, which can include new treatments or behavorial changes (e.g., diet changes). To learn more about clinical research, please visit the NephCure website.