Long-term心理 and cognitive check after stem cell transplant for immune deficiencies

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study follows children aged 6 to just under 9 years who underwent allogeneic hematopoietic stem cell transplantation for a primary immunodeficiency at least two years ago. The assessment focuses on neurocognitive, psychological, and psychosocial functioning during a routine follow-up day hospital visit at Necker-Enfants Malades Hospital in Paris. The pilot aims to describe the multidimensional outcomes and identify potential factors associated with long-term well-being after transplantation. Enrollment is limited to patients treated at the Necker hospital who meet specific age and language criteria, with informed consent from guardians. No experimental interventions are being tested; standardized assessments are used to characterize outcomes over time.

Why it matters

Who can join: the full rules

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.

Where it's running

• Hôpital Necker-Enfants Malades
  Paris, France Recruiting

Study contacts

• Principal investigator: Agathe Escudier, MD — Assistance Publique - Hôpitaux de Paris
• Study coordinator: Agathe Escudier, M.D.
• Email: agathe.escudier@aphp.fr
• Phone: 0144498222

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.