Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

Launched by NOVARTIS PHARMACEUTICALS · Jun 29, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called iptacopan for patients with a rare condition known as atypical hemolytic uremic syndrome (aHUS). The goal is to see if switching from their current medication, which is an anti-C5 antibody, to iptacopan is both safe and effective. The study is currently looking for participants aged 18 and older who have been on the anti-C5 treatment for at least three months and have shown a positive response to it, such as stable blood platelet levels and kidney function.

If you or someone you know is considering participating, it’s important to know that certain health criteria must be met. Eligible participants should not have had a relapse of aHUS while on the anti-C5 treatment, and they must be up to date on specific vaccinations to prevent infections. The study will involve regular check-ups to monitor health and response to the new treatment. This trial could provide valuable information about a new option for managing aHUS, potentially improving the lives of those affected by this condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Male and female adult participants ≥ 18 years of age with diagnosis of aHUS for whom etiologies of other types of TMA and non-aHUS kidney disease have been excluded.
• • Currently on the recommended weight-based dosage regimen of anti-C5 antibody treatment for at least 3 months prior to the screening visit.
• * Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) for at least 3 months prior to entering the screening period as defined by:
• • 1. Hematological normalization in platelet count ≥150 x 109/L and LDH below upper limit of normal \[ULN\], and
• • 2. Stable or improving kidney function as defined by ≤15% increase in serum creatinine.
• • Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of treatment with iptacopan.
• • If not received previously or if a booster is required, vaccination against Haemophilus influenzae infection, should be given, if available and according to local regulations.
• Exclusion Criteria:
• • History of aHUS disease relapse while on anti-C5 antibody treatment.
• • eGFR \< 30 ml/min/1.73m\^2
• • Active infection or history of recurrent invasive infections caused by encapsulated bacteria, i.e., meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) or H. influenzae.
• • Participants with sepsis or active systemic bacterial, viral (including COVID-19) or fungal infection within 14 days prior to study treatment administration.
• • Kidney, bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT), heart, lung, small bowel, pancreas, liver transplantation or any other cell or solid organ transplantation
• • Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study
• • Any medical condition deemed likely to interfere with the patient's participation in the study