International Multicenter Study on Lipin-1 Deficiency

Last updated: February 28, 2024
Sponsor: Imagine Institute
Overall Status: Active - Recruiting

Phase

N/A

Condition

N/A

Treatment

N/A

Clinical Study ID

NCT05564520
HJ-23-LIPIN1
  • Ages > 12
  • All Genders

Study Summary

LIPIN1 is an international registry that allows the collection of data on the follow-up, management and treatment of patients with Lipin-1 deficiency.

Eligibility Criteria

Inclusion

Inclusion Criteria: Patient at least 12 months with Lipin1 deficiency confirmed by molecular biology (twocausal mutations in the LPIN1 gene)

Exclusion

Exclusion Criteria: Opposition of the patient or his parents to participate to the study

Study Design

Total Participants: 150
Study Start date:
November 24, 2023
Estimated Completion Date:
December 31, 2026

Study Description

This study evaluates survival, cardiac function, quality of life, fatigability, treatment tolerance and comparison of the efficacy of different current treatments in patients with Lipin-1 deficiency.

Connect with a study center

  • Necker - Enfants Malades Hospital

    Paris, 75015
    France

    Active - Recruiting

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