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

Status: Recruiting
Location: See all (7) locations...
Intervention Type: Drug
Study Type: Observational
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
Participation Requirements
Sex: All
Maximum Age: 99
View:

• 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.

Locations
Other Locations
France
CHU de Besançon
RECRUITING
Besançon
Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néphrologie et Rhumatologie Pédiatriques - Hôpital Femme Mère Enfant
RECRUITING
Bron
Hopital Edouard Herriot
RECRUITING
Lyon
AP-HM - Timone Enfants
RECRUITING
Marseille
CHU Paris - Hôpital Necker-Enfants Malades
RECRUITING
Paris
Hôpital Européen G. Pompidou
RECRUITING
Paris
Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres
RECRUITING
Paris
Contact Information
Primary
Mélissa CLOAREC, Clinical Research Associate
melissa.cloarec@chu-lyon.fr
04 27 85 51 54
Backup
Sacha FLAMMIER, Project Manager
sacha.flammier@chu-lyon.fr
04 72 68 13 49
Time Frame
Start Date: 2023-01-01
Estimated Completion Date: 2026-12-31
Participants
Target number of participants: 100
Treatments
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.
Sponsors
Leads: Hospices Civils de Lyon

This content was sourced from clinicaltrials.gov

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