Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France.
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Oxaluria evolution..
- Who it may be relevant to
- Registry conditions: Patients With PH1 Treated With Lumasiran in France. Basic parameters: 0 years — 99 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France - DAILY-LUMA
Overview
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.
Interventions
- Drug Oxaluria evolution.
To collect real data from the specific French experience by collecting data from patients treated throughout the country and to monitor in particular the evolution of oxaluria before and after treatment.
Primary outcome measures
- Evolution of oxaluria. [Time frame: 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]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Study design
- Observational model
- Cohort
Study locations
France · 7 centers
- CHU de Besançon — Besançon
- Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néph — Bron
- Hopital Edouard Herriot — Lyon
- AP-HM - Timone Enfants — Marseille
- Hôpital Européen G. Pompidou — Paris
- CHU Paris - Hôpital Necker-Enfants Malades — Paris
- Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres — Paris
Identifiers
NCT: NCT06225882 · 69HCL22_0535