NCT04817618 · Novartis Pharmaceuticals
Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.
(APPEAR-C3G)
What this study is about
The Primary Completion Date and Study Completion Date have been updated to reflect completion of the adolescent group of participants, which has been added to the protocol.
View original scientific description
The Primary Completion Date and Study Completion Date have been updated to reflect completion of the adolescent cohort, which has been added to the protocol. 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.
Interventions
DRUG
Placebo
Placebo to iptacopan 200mg b.i.d. (Adults 200mg b.i.d; Adolescents 2x 100mg b.i.d)
DRUG
iptacopan
iptacopan 200 mg b.i.d. (Adults 200mg b.i.d; Adolescents 2x 100mg b.i.d)
Primary outcome measures
Adult cohort: Log-transformed ratio to baseline in UPCR (sampled from a 24-hour urine collection)
Time frame: 6 months (double-blind)
To demonstrate the superiority of iptacopan compared to placebo in reducing proteinuria at 6 months of treatment.
Adolescent cohort: Log-transformed ratio to baseline in UPCR (sampled from a 24-hour urine collection)
Time frame: 6 months (double-blind)
To evaluate the effect of iptacopan on proteinuria at 6 months.
Change from baseline in log-transformed UPCR at the 12-month visit (both study treatment arms).
Time frame: 12 months (double-blind and open-label)
To evaluate the effect of iptacopan on proteinuria at 12 months.
Change in log-transformed UPCR from the 6-month visit to the 12-month visit in the placebo arm
Time frame: From month 6 to month 12 (open-label)
To evaluate the effect of iptacopan on proteinuria at 12 months.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Male and female participants age ≥ 12 and ≤ 60 years at screening.
- Diagnosis of C3G as confirmed by renal biopsy within 12 months prior to enrollment in adults and within 3 years in adolescents.
- Prior to randomization, all participants must have been on a maximally recommended or tolerated dose of an angiotensin converting enzyme inhibitor (ACEI) or angiotensin receptor blocker (ARB) for at least 90 days. The doses of other antiproteinuric medications including mycophenolic acid, corticosteroids and mineralocorticoid receptor antagonists should be stable for at least 90 days prior to randomization.
- Reduced serum C3 (defined as less than 0.85 x lower limit of the central laboratory normal range) at Screening.
- UPCR ≥ 1.0 g/g sampled from the first morning void urine sample at Day -75 and Day -15.
- Estimated GFR (using the CKD-EPI formula for ages ≥ 18 years and modified Schwartz formula for ages 12 to 17 years) or measured GFR ≥ 30 ml/min/1.73m2 at screeni
Where
- Aurora, Colorado
- Miami, Florida
- Lawrenceville, Georgia
- Indianapolis, Indiana
- Iowa City, Iowa
- Baltimore, Maryland
- Boston, Massachusetts
- Hackensack, New Jersey
- Albany, New York
- New York, New York
- Temple, Texas
- Madison, Wisconsin
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 13, 2026 · Source of record for eligibility and locations