Hereditary Spherocytosis and Vascular Function (NCT04451785) | Clinical Trial Compass
CompletedNot Applicable
Hereditary Spherocytosis and Vascular Function
France72 participantsStarted 2020-08-26
Plain-language summary
Background : Patients with hereditary spherocytosis (HS) are characterized by increased red blood cell (RBC) fragility and a loss of RBC deformability. While the clinical variability of the disease may be heterogenous from one patient to another, some studies reported the occurrence of vascular complications, notably in patients who have been splenectomized.
Purpose : The aim of the study is to test the associations between the degree of vascular dysfunction and the extent of hemolysis, the amount of circulating microparticles, the level of erythrosis and the degree of RBC biophysical alterations.
Abstract : Recent studies reported the occurrence of vascular complications in patients with HS, notably in patients who have previously been splenectomized. However, the exact reasons of these complications are unknown and no study investigated the vascular function in HS patients.
Main objective Highlight the presence of altered vascular function in HS patients and test the relationships with the level of hemolysis and circulating microparticles.
Secondary objectives To evaluate the associations between clinical severity and 1) the level of vascular dysfunction and 2) several biomarkers (hemolysis, hematological parameters, circulating microparticles, erythrosis, RBC biophysical properties).
Who can participate
Age range
6 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:
Healthy controls:
* age ≥ 6 years old
* written, informed and signed consent by the healthy volunteer, or by both parents or legal guardian if the healthy volunteer is a minor
* Healthy volunteer affiliated to a social security scheme or assimilated
* Healthy volunteer not subject to any legal protection measure
Patients with hereditary spherocytosis:
* age ≥ 6 years old
* Patient with hereditary or non-splenectomized spherocytosis
* Written, informed and signed consent by the patient, or by at least one of the two parents or legal guardian if the patient is a minor
* Patient affiliated to a social security scheme or assimilated
* Patient not subject to any legal protection measure
Exclusion Criteria:
Healthy controls:
* Pregnant or lactating woman
* Subjects with hereditary spherocytosis or other characterized condition by chronic hemolysis
* Subjects with known pathology affecting the vascular system
* Blood donation (less than a month old)
* Not affiliated to a social security scheme
* Patient participating in another interventional research protocol that may interfere with this protocol (according to the investigator's judgment).
Patients with hereditary spherocytosis:
* Patient who received a blood transfusion in the 3 months preceding
* Pregnant or lactating woman
* Any disease or condition other than hereditary spherocytosis, chronic or not, likely to induce chronic or acute intravascular hemolysis
* Patient participating in another interve…
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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.