Ravulizumab for IgA Nephropathy

This trial tests a medicine called ravulizumab for children and teens with IgA nephropathy, a kidney disease that can cause protein to leak into the urine. The goal is to understand how the medicine works in young people compared to adults. Participants receive the medicine through an IV (a tube into a vein) on specific days, with dosing frequency based on their weight. Children and teens with this kidney issue who meet certain health requirements might be suitable for this study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new treatment.

The trial requires that participants stay on a stable dose of RASI (ACEI and/or ARB) for at least 3 months before and during the study. If you are on these medications, you will need to continue taking them without changes.

Research has shown that ravulizumab is generally safe for people with IgA nephropathy. In past studies, participants taking ravulizumab experienced side effects similar to those taking a placebo, a harmless pill with no active ingredients. This indicates that the treatment did not cause more side effects than the placebo, suggesting its safety. Ravulizumab has been used for years to treat other conditions, maintaining a strong safety record. While some side effects can occur, they are usually mild and comparable to those of other treatments.¹²³⁴⁵

Ravulizumab is unique because it offers a new approach for treating IgA nephropathy by targeting and inhibiting the complement system, which plays a key role in the disease. Unlike standard treatments that often involve immunosuppressive drugs, Ravulizumab specifically blocks the C5 protein in the complement pathway, potentially reducing inflammation and kidney damage more effectively. Researchers are excited about this treatment because it only requires infusions every 4 to 8 weeks, which could be more convenient for patients compared to the more frequent dosing schedules of existing therapies.

Research has shown that ravulizumab, the investigational treatment in this trial, may effectively treat IgA nephropathy, a kidney condition. One study found it reduced protein in the urine by 41.9% compared to a placebo over 26 weeks. Lowering protein in the urine helps protect the kidneys. Another study reported a 30.1% reduction in protein levels after about six months. Patients taking ravulizumab also maintained stable kidney function, as measured by the eGFR test, and the treatment was generally well-tolerated. These findings suggest that ravulizumab could be a helpful option for managing IgA nephropathy.¹²³⁵⁶