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NCT Number: NCT06225882

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

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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Key information

Conditions

Patients With PH1 Treated With Lumasiran in France Primary hyperoxaluria type 1

Age range

0 year–99 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Hôpital Necker, APHP Paris, Service de néphrologie-dialyse, 149 rue de Sèvres, Paris, Île-de-France Region, France

Loading trial locations.

Who can participate

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

Primary outcomes

  1. 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Mélissa CLOAREC, Clinical Research Associate

CONTACT

[email protected]

04 27 85 51 54

Sacha FLAMMIER, Project Manager

CONTACT

[email protected]

04 72 68 13 49

Sponsors and collaborators

Lead sponsor

Hospices Civils de Lyon

Other

Registry information

Official study title

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

Acronym: DAILY-LUMA

Important dates

Study start
2023
Primary completion
2024
Study completion
2026
First posted
Jan 26, 2024
Registry last updated
Jan 26, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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