Evaluate DF-003 in ex Vivo Assays Using Peripheral Blood Mononuclear Cell From Subjects With ROSAH Syndrome

Launched by HOSPICES CIVILS DE LYON · Mar 31, 2022

Keywords

ClinConnect Summary

This clinical trial is studying a potential treatment for a condition called ROSAH Syndrome, which is linked to a genetic change known as the T237M mutation in a gene called ALPK1. Researchers believe that this mutation causes increased activity in the ALPK1 gene, leading to inflammation and other symptoms in patients with ROSAH Syndrome. The trial will test a new medication, called DF-003, which is designed to block the effects of the ALPK1 mutation. By examining blood samples from patients, the researchers hope to see if DF-003 can reduce the activity of genes related to inflammation.

To participate in this trial, individuals must be at least 18 years old and have a confirmed diagnosis of ROSAH Syndrome with the T237M mutation. Unfortunately, people with certain medical conditions or those unable to provide consent cannot take part. If eligible, participants will receive either the DF-003 treatment or a placebo (a substance with no active ingredients) and will be monitored to see how their blood cells react. This study is currently recruiting participants, and the hope is that it will lead to a better understanding and potential treatment for ROSAH Syndrome.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Male or female aged over 18
• • Patient with ROSAH syndrome with the confirm T237M mutation
• Exclusion Criteria:
• • person under legal protection or under protectives measures
• • person unable to express consent
• • person in emergency situation (vital or not)
• • person infected by Human Immunodeficiency Virus and/or Hepatitis B Virus and/or Hepatitis C Virus