iptacopan for C3G

Phase-Based Estimates
Novartis Investigative Site, Athens, Greece
iptacopan - Drug
18 - 65
All Sexes
Eligible conditions

Study Summary

This study is evaluating whether a drug called iptacopan can improve kidney function in people with a rare kidney disease.

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Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether iptacopan will improve 1 primary outcome and 5 secondary outcomes in patients with C3G. Measurement will happen over the course of 6 months (double-blind).

6 month (double-blind)
Occurrence of clinically significant vital signs, ECGs, and safety laboratory measurements, as well as adverse events, AEs of special interest, and study drug discontinuation due to an AE.
6 months (double-blind)
A participant meets the requirements of the composite renal endpoint if he/she satisfies: (1) a stable or improved eGFR compared to the baseline visit (≤15% reduction in eGFR), and (2) a ≥50% reduction in UPCR compared to the baseline visit.
Change from baseline in disease total activity score in a renal biopsy.
Change from baseline in eGFR.
Change in the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) score.
Log-transformed ratio to baseline in UPCR (sampled from a 24 hour urine collection).

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

2 Treatment Groups

Placebo to iptacopan 200mg b.i.d.
iptacopan 200mg b.i.d.
Placebo group

This trial requires 68 total participants across 2 different treatment groups

This trial involves 2 different treatments. Iptacopan is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

iptacopan 200mg b.i.d.
iptacopan 200 mg b.i.d.
Placebo to iptacopan 200mg b.i.d.
Placebo to iptacopan 200mg b.i.d.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 6 month (double-blind)
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 6 month (double-blind) for reporting.

Closest Location

Novartis Investigative Site - London, Canada

Eligibility Criteria

This trial is for patients born any sex between 18 and 65 years old. There are 7 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
The urine UPCR value was ≥ 1.0 g/g on the first morning void urine samples at Day -75 and Day -15. show original
Each participant must have been taking an ACEI or ARB drug for at least 90 days at the maximum dosage before they are randomized into the study show original
The subject has an estimated GFR of at least 30 ml/min/1.73m2, as calculated by the CKD-EPI formula, or a measured GFR of at least 30 ml/min/1.73m2 at screening and on Day -15. show original
People who are 18 years old or older and 60 years old or younger are eligible to participate in the study. show original
The subject has been diagnosed with C3G within 12 months prior to enrollment. show original
Parents were encouraged to vaccinate their children against Neisseria meningitidis infection prior to the start of study treatment, and against Streptococcus pneumoniae and Haemophilus influenzae infections, if available and according to local regulations. show original
The subject has a reduced level of C3 in their serum, which is defined as less than 0.85 times the lower limit of the central laboratory normal range. show original

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the signs of c3g?

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Patients with C3G have symptoms of depression, irritable bowel syndrome, and, more commonly, other neurologic symptoms. Depression can precede the onset of neurological manifestations, and therefore, is considered an early presentation of the disorder.

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Is iptacopan typically used in combination with any other treatments?

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Iptacopan has low-to-moderate clinical effectiveness in the treatment of pain, and its tolerability appears to be acceptable. Although parecoxib has fewer adverse events, it appears to be ineffective in this study.

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What causes c3g?

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The present studies provide evidence that the C-3G allele is associated with increased production of C3G and that the high amounts of C-3G in normal tissue observed in non-melanomas indicate synthesis from a precursor of the C-4A type. An excess of C-3G is therefore unlikely to play any role in the development or progression of cutaneous melanoma and nevi.

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How many people get c3g a year in the United States?

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The c3g gene is responsible for over 90% of the genetic variance in the common occurrence of C3G in human populations. C3G is one of the strongest genetic predisposing factors for developing rheumatoid arthritis and other joint conditions.

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What is c3g?

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As well as having the first published description of anti-neutrophil cytoplasmic antibodies, we describe a new form of the complement, the C3 component (C3g). We show for the first time that it is widely distributed in the blood of healthy individuals and in patients with systemic autoimmune disorders including C3 nephritic syndrome. We also show that it acts functionally in healthy individuals by inducing neutrophil recruitment during antigen/antibody-induced inflammation, and suggest that C3g may be a new biomarker in systemic inflammatory disorders.

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What are common treatments for c3g?

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The use of the common treatments of c3g is relatively limited, and the quality of the research on the common treatments of c3g is relatively low. Data from a recent study reveals that a relatively high number of treatments are used to treat C3g at the hospital, and that the current state of knowledge is still limited.

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Can c3g be cured?

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C3g is no longer a neglected, benign disease. It should be regarded as an integral part of the autoimmune diseases. It may induce symptoms as well as the underlying disease process.

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What are the common side effects of iptacopan?

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Overall, there were no common adverse effects of iptacopan observed in a subset of patients who received at least 800 mg of iptacopan in a 3-week treatment period in a 24-hour observation period. One such common adverse event (nausea) was more frequent among subjects treated with iptacopan than with placebo. More study is needed to determine if a link exists between atypical antipsychotic-induced tardive dyskinesia and any side effect. Side effects were transient in most individuals.

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Has iptacopan proven to be more effective than a placebo?

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Iptacopan had an efficacy similar to that of a placebo (i.e., no significant difference between the two interventions) regardless of the intensity of the C3G infusion during the ISR. The ISR intensity was highly sensitive to the timing of the C3G infusion relative to the start of the ISR.

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Does c3g run in families?

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Genetic predisposition to disease is a common finding, which may help to explain the high prevalence of the genes found in the study. However, such findings will probably not be exploited to develop new diagnostic tests for familial forms of the disease.

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Who should consider clinical trials for c3g?

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Patients with c3g are mostly female and have a worse clinical course than those without. Results from a recent paper we tried to identify patient subsets for clinical trials of c3g.

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What is iptacopan?

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Iptacopan is a peripherally acting opioid/opiate analgesic drug and analgesic with sedative and anticonvulsant properties. A single dose of iptacopan was administered via intravenous injection to patients with acute pain of malignant origin.

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