Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France.
Trial Parameters
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Brief Summary
Primary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by mutation in the AGXT gene encoding the hepatic peroxisomal enzyme AGT. Reduced AGT activity results in increased glyoxylate and oxalate production, causing the formation of kidney stones, nephrocalcinosis and renal failure. Clinical trials of Lumasiran have provided information on the efficacy and safety of Lumasiran in the treatment of primary hyperoxaluria type 1. However, they do not provide data on long-term efficacy, safety and patient management. As part of the post-marketing follow-up of Lumasiran, in agreement with the authorities, this study proposes a retrospective and prospective follow-up over 5 years of pediatrics and adults patients treated in France with a standardized clinical, biological and radiological follow-up. The main objective is to monitor the evolution of PH1 parameters and particularly oxaluria before and after treatment.
Eligibility Criteria
Inclusion Criteria: * Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran, since the beginning of the ATU (temporary authorization for use) and in post-marketing. Exclusion Criteria: * Opposition of the patient or his legal representatives for minors. * Not covered by social security.