Interventional Study
a clinical trial in which a new drug or device is used to measure a specific outcome.
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 6915Site Name
Johns Hopkins Hospital
25 Hackett Blvd Dept. Of Medicine MC69 Albany, NY 12208
Interventional Study
a clinical trial in which a new drug or device is used to measure a specific outcome.
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.