Identifying genetic targets for treating histiocytosis
Molecular Targets for the Treatment of Histiocytosis
This study is trying to find genetic changes linked to histiocytosis to help with diagnosis and treatment for people with the disease.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 1800 (estimated) |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris Academic / other |
| Locations | 3 sites (Boulogne Billancourt and 2 other locations) |
| Trial ID | NCT04437381 on ClinicalTrials.gov |
What this trial studies
This observational study aims to establish procedures for detecting mutations associated with histiocytosis using non-specialized molecular platforms, which will aid in diagnosis and monitoring of the disease. It is part of the French 'Gene Histio' cohort and 'HISTIO target 2020' cohort, involving multiple teams responsible for histological diagnosis, clinical data collection, and molecular analysis. The study will also explore therapeutic target mutations and develop cellular tests to assess the sensitivity of these mutations to inhibitors.
Who should consider this trial
Good fit: Ideal candidates include individuals with histologically confirmed histiocytosis who have provided informed consent.
Not a fit: Patients who refuse to participate in the study will not benefit from its findings.
Why it matters
Potential benefit: If successful, this study could enhance the accuracy of histiocytosis diagnosis and improve treatment options for patients.
How similar studies have performed: Other studies have shown promise in identifying genetic mutations in similar conditions, suggesting a potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Histologically proven histiocytosis; * Informed consent form signed by patients (or parents/legal tutors of children) to participate"Gene Histio" or "HISTIO target 2020". Exclusion Criteria: - Patient refusal.
Where this trial is running
Boulogne Billancourt and 2 other locations
- Biological research center, Ambroise Paré hospital, APHP — Boulogne Billancourt, France (Recruiting)
- Department of internal Medicine, Pitié-Salpétrière hospital, APHP — Paris, France (Recruiting)
- Department of Pediatrics, Trousseau hospital, APHP — Paris, France (Recruiting)
Study contacts
- Principal investigator: Jean-François EMILE, MD, PhD — Department of Pathology, Ambroise Paré hospital, APHP
- Study coordinator: Jean-François EMILE, MD, PhD
- Email: jean-francois.emile@uvsq.fr
- Phone: +33 1 49 09 57 25
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.