Patients With PH1 Treated With Lumasiran in France
Conditions
Keywords
lumarisan, Primary hyperoxaluria type 1 (PH1), retrospective and prospective data.
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.
Interventions
DRUGOxaluria 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.
Sponsors
Hospices Civils de Lyon
Study design
Observational model
COHORT
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Age
0 Years to 99 Years
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
France
Contacts
Primary ContactMélissa CLOAREC, Clinical Research Associate
Backup ContactSacha FLAMMIER, Project Manager
Outcome results
None listed