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

Recruiting now

Conditions studied: Patients With PH1 Treated With Lumasiran in France

In brief

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.

Key facts

Study ID

NCT06225882

Run by

Hospices Civils de Lyon

People needed

100

Starts

2023-01-01

Expected to finish

2026-12-31

Last updated by the study team

2024-01-26

Who can join

Age: any, up to 99. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Opposition of the patient or his legal representatives for minors.
• Not covered by social security.

Where it is running

• CHU de Besançon — Besançon, France (enrolling)
• Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néphrologie et Rhumatologie Pédiatriques - Hôpital Femme Mère Enfant — Bron, France (enrolling)
• Hopital Edouard Herriot — Lyon, France (enrolling)
• AP-HM - Timone Enfants — Marseille, France (enrolling)
• Hôpital Européen G. Pompidou — Paris, France (enrolling)
• CHU Paris - Hôpital Necker-Enfants Malades — Paris, France (enrolling)
• Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres — Paris, Île-de-France Region, France (enrolling)

Full record on ClinicalTrials.gov

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