Molecular Targets for the Treatment of Histiocytosis

NCT04437381 | Unknown

• 1 other identifier: APHP200137
• Study Type: observational
• Target: 1,800
• Locations: 1 country
• Sites: 3

Brief Summary

The primary objective: to develop technical and operating procedures for detection mutations of histiocytosis during clinical practice of no-specialized molecular platforms, for diagnosis and follow-up of the disease. The secondary objectives: to describe therapeutic target mutations in histiocytosis patients, and to develop the cellular tests to evaluate in vitro the sensibility of these mutations drive to inhibitors.

Conditions

Histiocytosis

Keywords

histiocytosis

Outcome Measures

Primary Outcomes (1)

• Molecular alterations in histiocytosis

  Characterize the molecular alterations of nucleic acids in histiocytosis. Assess the frequency and type of drugable mutations in the different sub-types and localizations of histiocytosis Molecular alterations will be assessed by next analysis of DNA or RNA extracted from histiocytosis tissue or blood samples.

  throughout of the study, an average of 3 years

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Paediatric and adult patients with histologically proven histiocytosis, included in either "Gene Histio" or "HISTIO target 2020" cohorts between 1997 and 2023.

You may qualify if:

• Histologically proven histiocytosis;
• Informed consent form signed by patients (or parents/legal tutors of children) to participate"Gene Histio" or "HISTIO target 2020".

You may not qualify if:

• \- Patient refusal.

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• National Cancer Institute, France: collaborator
• Direction de l'Hospitalisation et de l'Organisation des Soins: collaborator
• Versailles Saint-Quentin-en-Yvelines University, Paris-Saclay University: collaborator

Study Sites (3)

Biological research center, Ambroise Paré hospital, APHP

Boulogne-Billancourt, 92100, France

RECRUITING

Department of internal Medicine, Pitié-Salpétrière hospital, APHP

Paris, 75013, France

RECRUITING

Department of Pediatrics, Trousseau hospital, APHP

Paris, France

RECRUITING

Related Publications (1)

• Bigenwald C, Roos-Weil D, Pages A, Helias-Rodzewicz Z, Copie-Bergman C, Nashvi M, Khneisser P, Parrens M, Traverse-Glehen A, Ray-Coquard I, Ysebaert L, Marchand T, Razanamahery J, Charlotte F, Neel A, Damaj G, Dion J, Nazal-Traissac EM, Tardy S, Salmeron G, Monjanel H, Idbaih A, Heritier S, Haroche J, Donadieu J, Emile JF. Characterization and treatment outcomes of malignant histiocytoses in a retrospective series of 141 cases in France. Blood Adv. 2025 May 27;9(10):2530-2541. doi: 10.1182/bloodadvances.2024015208.

  PMID: 40009752 DERIVED

Biospecimen

Retention: SAMPLES WITHOUT DNA

Tissue and blood samples and derived nucleic acids.

MeSH Terms

Conditions

Histiocytosis

Condition Hierarchy (Ancestors)

Lymphatic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Jean-François EMILE, MD, PhD

  Department of Pathology, Ambroise Paré hospital, APHP

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jean-François EMILE, MD, PhD

CONTACT

+33 1 49 09 57 25; jean-francois.emile@uvsq.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 8, 2020
• First Posted: June 18, 2020
• Study Start: November 22, 2021
• Primary Completion: April 1, 2025
• Study Completion: May 1, 2025
• Last Updated: September 6, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

FR (3)