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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Lumasiran | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Oxaluria evolution. | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Evolution of oxaluria. | The evolution of oxaluria is followed by urinary biological analysis. | 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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Inclusion Criteria:
Exclusion Criteria:
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Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran in France.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Mélissa CLOAREC, Clinical Research Associate | Contact | 04 27 85 51 54 | melissa.cloarec@chu-lyon.fr | |
| Sacha FLAMMIER, Project Manager | Contact | 04 72 68 13 49 | sacha.flammier@chu-lyon.fr |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CHU de Besançon | Recruiting | Besançon | 25030 | France |
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| Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néphrologie et Rhumatologie Pédiatriques - Hôpital Femme Mère Enfant | Recruiting | Bron | 69500 | France |
|
| Hopital Edouard Herriot | Recruiting | Lyon | 69003 | France |
|
| AP-HM - Timone Enfants | Recruiting | Marseille | 13385 | France |
|
| Hôpital Européen G. Pompidou | Recruiting | Paris | 75015 | France |
|
| CHU Paris - Hôpital Necker-Enfants Malades | Recruiting | Paris | 75743 | France |
|
| Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres | Recruiting | Paris | Île-de-France Region | 75015 | France |
|
| ID | Term |
|---|---|
| C536414 | Primary hyperoxaluria type 1 |
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