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

Recruiting

• Conditions: Patients With PH1 Treated With Lumasiran in France
• Interventions: Drug: Oxaluria evolution.

• Registration Number: NCT06225882
• Lead Sponsor: Hospices Civils de Lyon

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-01-01
• Status Verified: 2024-01
• Primary Completion: 2024-01-01
• Study First Submitted: 2024-01-17
• Study First Submitted QC: 2024-01-17
• Last Update Submitted: 2024-01-17
• Study First Posted: 2024-01-26
• Last Update Posted: 2024-01-26
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 100

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Lumasiran	Oxaluria evolution.	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.

Primary Outcome Measures

Name	Time	Method
Evolution of oxaluria.	At baseline, At 1 month from the baseline, At 2 months from baseline, At 3 months from baseline, At 6 months from baseline, At 9 months from baseline, At 12 months from baseline, At 18 months from baseline, And 2 times a year until 5 year	The evolution of oxaluria is followed by urinary biological analysis.

Trial Locations

Locations (7)

Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres; 🇫🇷 Paris, Ile De France, France

CHU Paris - Hôpital Necker-Enfants Malades; 🇫🇷 Paris, France

Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néphrologie et Rhumatologie Pédiatriques - Hôpital Femme Mère Enfant; 🇫🇷 Bron, France

AP-HM - Timone Enfants; 🇫🇷 Marseille, France

Hôpital Européen G. Pompidou; 🇫🇷 Paris, France

CHU de Besançon; 🇫🇷 Besançon, France

Hopital Edouard Herriot; 🇫🇷 Lyon, France