Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France.

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

100 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-01-01

Study Completion Date

2026-12-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Patients With PH1 Treated With Lumasiran in France

Study Design

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Observational Model Type

COHORT

Study Time Perspective

CROSS_SECTIONAL

Study Groups

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Lumasiran

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.

Oxaluria evolution.

Intervention Type DRUG

To collect real data from the specific French experience by collecting data from patients treated throughout the country and to monitor in particular the evolution of oxaluria before and after treatment.

Interventions

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Oxaluria evolution.

To collect real data from the specific French experience by collecting data from patients treated throughout the country and to monitor in particular the evolution of oxaluria before and after treatment.

Intervention Type DRUG

Eligibility Criteria

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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.