Evaluate DF-003 in ex Vivo Assays Using Peripheral Blood Mononuclear Cell From Subjects With ROSA… (NCT05319132) | Clinical Trial Compass
RecruitingNot Applicable
Evaluate DF-003 in ex Vivo Assays Using Peripheral Blood Mononuclear Cell From Subjects With ROSAH Syndrome
France4 participantsStarted 2022-09-06
Plain-language 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this trial is listed as Phase NA and seems to involve lab-based cytokine release assays using my blood cells rather than giving me a new treatment directly, can you explain what that actually means for me as a participant — is this more like a blood donation for research than a treatment study?
2ROSAH syndrome is very rare, so how confident are you that my diagnosis qualifies me for this study, and what would the process look like to confirm my eligibility?
3The trial is measuring cytokine release, which relates to immune system signaling — can you explain what researchers are hoping to learn about ROSAH syndrome from this, and how it might eventually affect treatment options for me or others with this condition?
4Since this appears to be an ex vivo study using my peripheral blood mononuclear cells, what does the blood collection process involve, how much blood would be taken, and are there any real risks I should be aware of even if I'm not receiving an experimental drug?
5Before I consider this trial, is there any standard of care treatment or other research study I should explore first that might more directly address my symptoms right now?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.