Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France. (DAILY-LUMA)

January 17, 2024 updated by: Hospices Civils de Lyon

Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France - DAILY-LUMA

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • France
      • Besançon, France, 25030
        • Recruiting
        • CHu de Besançon
        • Contact:
        • Principal Investigator:
          • Francois NOBILI, MD,PhD
      • Bron, France, 69500
        • Recruiting
        • Centre de Référence des Maladies Rénales Rares - Hospices Civils de Lyon - Service de Néphrologie et Rhumatologie Pédiatriques - Hôpital Femme Mère Enfant
        • Principal Investigator:
          • Justine BACCHETTA, MD
        • Contact:
      • Lyon, France, 69003
        • Recruiting
        • Hôpital Edouard Herriot
        • Contact:
        • Principal Investigator:
          • Sandrine LEMOINE, PU-PH
      • Marseille, France, 13385
        • Recruiting
        • AP-HM - Timone Enfants
        • Contact:
        • Principal Investigator:
          • Caroline ROUSSET-ROUVIERE, MD PHD
      • Paris, France, 75015
        • Recruiting
        • Hopital Europeen G. Pompidou
        • Contact:
        • Principal Investigator:
          • Alexandre KARRAS, Pr
      • Paris, France, 75743
        • Recruiting
        • CHU Paris - Hôpital Necker-Enfants Malades
        • Contact:
        • Contact:
        • Principal Investigator:
          • Aude SERVAIS, PU PH
        • Sub-Investigator:
          • Olivia BOYER, MD PHD
    • Ile De France
      • Paris, Ile De France, France, 75015
        • Recruiting
        • Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Sampling Method

Non-Probability Sample

Study Population

Patient with primary hyperoxaluria type 1 who has been treated with Lumasiran in France.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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.
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evolution of oxaluria.
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
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Hospices Civils de Lyon

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2023

Primary Completion (Actual)

January 1, 2024

Study Completion (Estimated)

December 31, 2026

Study Registration Dates

First Submitted

January 17, 2024

First Submitted That Met QC Criteria

January 17, 2024

First Posted (Estimated)

January 26, 2024

Study Record Updates

Last Update Posted (Estimated)

January 26, 2024

Last Update Submitted That Met QC Criteria

January 17, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 69HCL22_0535

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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