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Recruiting NCT05319132

NCT05319132 Evaluate DF-003 in ex Vivo Assays Using Peripheral Blood Mononuclear Cell From Subjects With ROSAH Syndrome

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Clinical Trial Summary
NCT ID NCT05319132
Status Recruiting
Phase
Sponsor Hospices Civils de Lyon
Condition Unrecognized Condition
Study Type OBSERVATIONAL
Enrollment 4 participants
Start Date 2022-09-06
Primary Completion 2028-05-06

Eligibility & Interventions

Sex All sexes
Min Age 18 Years
Max Age N/A
Study Type OBSERVATIONAL
Interventions
Adult subjects with ROSAH syndrome

Eligibility Fast-Check

Enter your details for a quick preliminary check. This does not replace medical advice.

What to Expect as a Participant

This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.

This trial targets 4 participants in total. It began in 2022-09-06 with a primary completion date of 2028-05-06.

⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.

Brief Summary

Alpha-1 kinase (ALPK1) has been reported as a potential causative gene for ROSAH Syndrome. Genetic variants including T237M have been found in ROSAH Syndrome patients. Our in-house study has found that T237M mutation leads to hyperactivity of ALPK1, which may be the cause of the inflammatory syndromes found in ROSAH Syndrome patients. We hypothesize that T237M mutation ALPK1 cause ROSAH Syndrome and an ALPK1 inhibitor can be a potential therapy for treating this disease. To test our hypothesis, we designed an experiment in which ex vivo peripheral blood mononuclear cells (PBMCs) from ROSAH Syndrome patients will be exposed to a potent ALPK1 inhibitor (DF-003) or placebo. We expect to see downregulation of activated inflammatory genes, chemokine/cytokines and acute phase proteins in the ROSAH Syndrome patient samples that are exposed DF-003.

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

Contact & Investigator

Central Contact

YVAN JAMILLOUX, MD

✉ yvan.jamilloux@chu-lyon.fr

📞 04 26 73 26 36

Principal Investigator

YVAN JAMILLOUX

PRINCIPAL INVESTIGATOR

Service de medecine interne - Hôpital de la Croix Rousse

Frequently Asked Questions

Who can join the NCT05319132 clinical trial?

This trial is open to participants of all sexes, aged 18 Years or older, studying Unrecognized Condition. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.

Is NCT05319132 currently recruiting?

Yes, NCT05319132 is actively recruiting participants. Contact the research team at yvan.jamilloux@chu-lyon.fr for enrollment information.

Where is the NCT05319132 trial being conducted?

This trial is being conducted at Lyon, France, Angers, France, Paris, France, Reims, France and 1 additional location.

Who is sponsoring the NCT05319132 clinical trial?

NCT05319132 is sponsored by Hospices Civils de Lyon. The principal investigator is YVAN JAMILLOUX at Service de medecine interne - Hôpital de la Croix Rousse. The trial plans to enroll 4 participants.

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