The Study of the Phenotype of Hereditary Xerocytosis

Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age10+
SponsorCentre Hospitalier Universitaire, Amiens

About this trial

Hereditary xerocytosis is a dominant red blood cell membrane disorder characterized by an increased leakage of potassium from the interior to the exterior of the red blood cell membrane, leading to water loss, red cell dehydration, and chronic hemolysis. In 90% of cases, it is associated with heterozygous gain-of-function mutations in PIEZO1, a gene that encodes a mechanotransducer responsible for converting mechanical stimuli into biological signals. The remaining 10% of cases are linked to mutations in the GARDOS channel gene.

Eligibility criteria

Qualifiers

Any patient diagnosed with hereditary xerocytosis according to the 2021 PNDS guidelines

Covered by a social security plan

Signature of the consent form for study participation by the patient, or for minors, by the parent(s)/legal representative(s).

Disqualifiers

patients with other hemolysis reason

Trial design

Treatments tested in this trial

  • Blood sample

Treatment groups

No treatment groups listed

Sponsors and collaborators

Centre Hospitalier Universitaire, Amiens

Lead sponsor

Kremlin-Bicetre Hospital, Paris

Collaborator