Follow-up of patients with primary hyperoxaluria type 1 treated with Lumasiran in France
Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France - DAILY-LUMA
This study is looking at how well Lumasiran works and how safe it is for people in France with primary hyperoxaluria type 1 over five years.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 100 (estimated) |
| Ages | 0 Years to 99 Years |
| Sex | All |
| Sponsor | Hospices Civils de Lyon Academic / other |
| Locations | 7 sites (Besançon and 6 other locations) |
| Trial ID | NCT06225882 on ClinicalTrials.gov |
What this trial studies
This study involves both retrospective and prospective follow-up of patients diagnosed with primary hyperoxaluria type 1 (PH1) who have been treated with Lumasiran in France. The aim is to monitor the long-term efficacy and safety of Lumasiran by evaluating the evolution of oxaluria and other clinical parameters over a period of five years. The study will include standardized clinical, biological, and radiological assessments to gather comprehensive data on patient management and outcomes. This approach seeks to fill the gap in knowledge regarding the long-term effects of Lumasiran treatment.
Who should consider this trial
Good fit: Ideal candidates for this study are patients diagnosed with primary hyperoxaluria type 1 who have received treatment with Lumasiran.
Not a fit: Patients who have not been treated with Lumasiran or those who oppose participation will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term management and outcomes of patients with primary hyperoxaluria type 1 treated with Lumasiran.
How similar studies have performed: While there have been clinical trials on Lumasiran, this study aims to provide novel long-term data on its effects in a real-world setting.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Besançon and 6 other locations
- CHU de Besançon — Besançon, France (Recruiting)
- Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néphrologie et Rhumatologie Pédiatriques - Hôpital Femme Mère Enfant — Bron, France (Recruiting)
- Hopital Edouard Herriot — Lyon, France (Recruiting)
- AP-HM - Timone Enfants — Marseille, France (Recruiting)
- Hôpital Européen G. Pompidou — Paris, France (Recruiting)
- CHU Paris - Hôpital Necker-Enfants Malades — Paris, France (Recruiting)
- Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres — Paris, Île-de-France Region, France (Recruiting)
Study contacts
- Study coordinator: Mélissa CLOAREC, Clinical Research Associate
- Email: melissa.cloarec@chu-lyon.fr
- Phone: 04 27 85 51 54
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.