Clinical trial NCT06225882: Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France.
Official title: Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France - DAILY-LUMA
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Patients With PH1 Treated With Lumasiran in France
Keywords: lumarisan
Primary hyperoxaluria type 1 (PH1)
retrospective and prospective data.
Lead sponsor: Hospices Civils de Lyon
Sponsor class: OTHER
First posted: 2024-01-26
Enrollment: 100
Sex: ALL
Minimum age (years): 0
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran in France.

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.


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.

Primary outcomes:
measure: Evolution of oxaluria.
timeFrame: 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
Secondary outcomes:
Other outcomes:
Locations:
city: Paris
country: France
facility: Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres
state: Ile De France
city: Besançon
country: France
facility: CHU de Besançon
city: Bron
country: France
facility: Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néphrologie et Rhumatologie Pédiatriques - Hôpital Femme Mère Enfant
city: Lyon
country: France
facility: Hopital Edouard Herriot
city: Marseille
country: France
facility: AP-HM - Timone Enfants
city: Paris
country: France
facility: Hôpital Européen G. Pompidou
city: Paris
country: France
facility: CHU Paris - Hôpital Necker-Enfants Malades
Study officials:
References: