Actively Recruiting
Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France.
Led by Hospices Civils de Lyon · Updated on 2024-01-26
100
Participants Needed
7
Research Sites
208 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran since the beginning of the temporary authorization for use or in post-marketing
You will not qualify if you...
- Patient or legal representative opposes participation (for minors)
- Not covered by social security
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 7 locations
1
CHU de Besançon
Besançon, France, 25030
Actively Recruiting
2
Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néphrologie et Rhumatologie Pédiatriques - Hôpital Femme Mère Enfant
Bron, France, 69500
Actively Recruiting
3
Hopital Edouard Herriot
Lyon, France, 69003
Actively Recruiting
4
AP-HM - Timone Enfants
Marseille, France, 13385
Actively Recruiting
5
Hôpital Européen G. Pompidou
Paris, France, 75015
Actively Recruiting
6
CHU Paris - Hôpital Necker-Enfants Malades
Paris, France, 75743
Actively Recruiting
7
Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres
Paris, Île-de-France Region, France, 75015
Actively Recruiting
Research Team
M
Mélissa CLOAREC, Clinical Research Associate
CONTACT
S
Sacha FLAMMIER, Project Manager
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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