Enrollment Closed
Interventional

APPEAR-C3G

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

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

Christina Manning

Email Phone 518 262 6915
Site Name

Johns Hopkins Hospital
25 Hackett Blvd Dept. Of Medicine MC69 Albany, NY 12208

Enrollment Closed
Interventional

APPEAR-C3G

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

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.

Eligibility Criteria
Patient Population

C3 Glomerulopathy (C3G)

Age

18 — 26+

History of transplant allowed?

No

History of dialysis allowed?

No

eGFR

30 — > 60

UPCR

1.1 — 3.0 or Above

Permitted medication history

Ace Inhibitors/ARB, Prednisone (Steroids), Abatacept, Acthar, Cellcept (AKA Mycophenolate), Cytoxan (Cyclophosphamide), Prograf (AKA Tacrolimus), Rituximab (AKA Rituxan), Other

Patient should be

Treatment Resistant, Steroid Dependent

About the Drug
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.

Sponsor

Novartis Pharmaceuticals

Estimated End Date

August 2023

About the Trial
Study Drug

LNP023/iptacopan

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.

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.

Albany, NY
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