Long-term Psychological and Cognitive Evaluation of Children Treated With Allogeneic Hematopoieti… (NCT07766304) | Clinical Trial Compass
Not Yet RecruitingNot Applicable
Long-term Psychological and Cognitive Evaluation of Children Treated With Allogeneic Hematopoietic Stem Cell Transplantation for Immunodeficiency
France30 participantsStarted 2026-08
Plain-language summary
Primary immunodeficiencies (PIDs) are a large group of genetic diseases of the immune system with highly variable clinical presentations. Allogeneic hematopoietic stem cell transplantation (HSCT) is one of the treatments offered to some patients with PIDs. It is a curative but particularly demanding treatment, potentially life-threatening, requiring several months of hospitalization, prolonged limitations in social interactions for the patient, and impacting the entire family unit. The short-term complications of HSCT are numerous and well-known. However, few studies describe the long-term psychological and cognitive complications of HSCT, particularly in the context of PIDs. The few published studies in children concern patients transplanted for hematological malignancies, a context very different from that of primary immunodeficiencies.
This study is a pilot research project focusing on the multidimensional assessment of the neurocognitive, psychological, and psychosocial functioning of children between 6 and 8 years old who have received allogeneic hematopoietic stem cell transplantation for primary immunodeficiency for at least 2 years. These stringent criteria aim to limit biases related to the diversity of ages at which care is provided.
Who can participate
Age range
6 Years – 8 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:
* Patients who underwent allogeneic transplantation for a primary immunodeficiency at Necker Hospital, regardless of the genetic diagnosis, and at least 2 years post Allogeneic hematopoietic stem cell transplantation.
* Chronological age at the time of evaluation between 6 years and 8 years 11 months.
* Holders of parental authority and children or adolescents or adults' patients informed and consenting to participate in the study
Exclusion Criteria:
* Child transplanted for a condition other than the primary immunodeficiency or at a different center.
* Presence of an associated acquired or genetic neurological condition, independent of the PID, likely to significantly impair cognitive development.
* Severe uncorrected sensory impairment (auditory or visual) rendering the cognitive assessment uninterpretable.
* Refusal to participate by the child or those with parental authority.
* Child not fluent in French or not proficient enough in French to complete the cognitive tests and questionnaires.
* Profound intellectual disability.
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.