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

Civil Hospices of Lyon

Status: Enrolling

Conditions: Patients With PH1 Treated With Lumasiran in France

Treatments: Drug: Oxaluria evolution.

Study type: Observational

Funder types: Other

Identifiers

NCT06225882

69HCL22_0535

Details and patient eligibility

About

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.

Enrollment: 100 estimated patients

Sex: All

Ages: Under 99 years old

Volunteers: No Healthy Volunteers

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.

Trial design

100 participants in 1 patient group

Lumasiran

Description:

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.

Treatment:

Drug: Oxaluria evolution.