An observational study in Patients With PH1 Treated With Lumasiran in France, sponsored by Hospices Civils de Lyon. Recruiting at 7 sites in France. Open to participants aged 0 Years to 99 Years. Per ClinicalTrials.gov, last updated 2024-01-26.
Sponsored by Hospices Civils de Lyon · Observational
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.
22 studies on the registry are indexed under Primary hyperoxaluria type 1; 8 are open to participants now.
Browse Primary hyperoxaluria type 1 studies →Hospices Civils de Lyon is the lead sponsor of 1,866 studies on the registry; 448 are open to participants now.
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Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran in France.
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Exclusion 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.
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.
Evolution of oxaluria.
The evolution of oxaluria is followed by urinary biological analysis.
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
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Primary hyperoxaluria type 1 →