Informations générales (source: ClinicalTrials.gov)
Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France - DAILY-LUMA (DAILY-LUMA)
Observational
Hospices Civils de Lyon (Voir sur ClinicalTrials)
janvier 2023
décembre 2026
29 juin 2024
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.
Etablissements
| Les établissements d'Île-de-France dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| AP-HP - Hôpital Europeen Georges Pompidou | Alexandre KARRAS, Pr | Contact (sur clinicalTrials) | |||
| AP-HP - Hôpital Necker-Enfants Malades | Aude SERVAIS, PU PH | Contact (sur clinicalTrials) | |||
| Les établissements sans correspondance certaine dans le répertoire FINESS dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| AP-HM - Timone Enfants - 13385 - Marseille - France | Caroline ROUSSET-ROUVIERE, MD PHD | Contact (sur clinicalTrials) | |||
| Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néphrologie et Rhumatologie Pédiatriques - Hôpital Femme Mère Enfant - 69500 - Bron - France | Justine BACCHETTA, MD | Contact (sur clinicalTrials) | |||
| CHU de Besançon - 25030 - Besançon - France | Francois NOBILI, MD,PhD | Contact (sur clinicalTrials) | |||
| Hopital Edouard Herriot - 69003 - Lyon - France | Sandrine LEMOINE, PU-PH | Contact (sur clinicalTrials) | |||
Critères
Tous
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.
- 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.
- Opposition of the patient or his legal representatives for minors.
- Not covered by social security.