LCH in Adults: a Collaborative, Prospective-retrospective, Observational Study

Langerhans Cell Histiocytosis Clinical Trial

Official title:

Langerhans Cell Histiocytosis in Adults: a Collaborative, Prospective-retrospective, Observational GIMEMA Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04627090
• Other study ID #: RD0120
• Status: Recruiting
• Start date: March 15, 2021
• Est. completion date: March 2024

Study information

• Verified date: February 2024
• Source: Gruppo Italiano Malattie EMatologiche dell'Adulto
• Contact: Paola Fazi
• Phone: 06 70390528
• Email: p.fazi[@]gimema.it
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This is a multicenter, retrospective and prospective, observational, no profit study including adult patients with LCH, diagnosed starting from January 2001 to two years after the first enrolled patient. Each patient will be followed up to one year after the last enrolled patient. Each patient will be followed up to one year after the last enrolled patient.This study plans to collect clinical information at the time of diagnosis and at various follow-ups to evaluate the efficacy of first-line therapies. Diagnostic and therapeutic data will be collected from routine clinical evaluations and laboratory and instrumental investigations carried out during clinical practice

Description:

This is a multicenter, retrospective and prospective, observational, no profit study including adult patients with LCH, diagnosed starting from January 2001 to two years after the first enrolled patient. Data from patients, diagnosed from January 2001 to the date of patient enrolment, is considered retrospective, while data collected after patient enrolment, and up to two years after the first enrolled patient, is considered prospective. At least one year of follow-up is expected for each patient. Each patient will be followed up to one year after the last enrolled patient. This study plans to collect clinical information at the time of diagnosis and at various follow-ups to evaluate the efficacy of first-line therapies. Diagnostic and therapeutic data will be collected from routine clinical evaluations and laboratory and instrumental investigations carried out during clinical practice. A sub-cohort of patients with available biological data on somatic mutations of the RAF-MEK-ERK genes will also be evaluated.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 186
• Est. completion date: March 2024
• Est. primary completion date: March 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Confirmed histological and immunohistochemical diagnosis of LCH (CD1a+, S-100+, CD207+), from January 2001 up to two years after the first enrolled patient. Patients with isolated vertebra plana, not related to a malignancy, and without a soft tissue component, are included without a histological and immunohistochemical diagnosis;

• Age =18 years at the time of definitive diagnosis;

• Signed, written informed consent, according to ICH/EU/GCP, and national, local laws.

Exclusion Criteria:

• Age =18 years and presumptive diagnosis of LCH, or definitive diagnosis of non-Langerhans Histiocytosis (Juvenile xantogranuloma, Rosai-Dorfman disease, Erdheim-Chester disease, etc.).

Related Conditions & MeSH terms

• Histiocytosis
• Histiocytosis, Langerhans-Cell
• Langerhans Cell Histiocytosis

Intervention

Other:
• Data collection
Diagnostic and therapeutic data of adult patients with LCH, collected from clinical and laboratory evaluations, carried out during clinical practice.

Locations

Country	Name	City	State
Italy	Ao Brotzu, Presidio Ospedaliero A. Businco - Cagliari - Sc Ematologia E Ctmo	Cagliari
Italy	Ctc U.O Di Ematologia Con Trapianto Di Midollo Osseo - Catania	Catania
Italy	Fondazione Irccs "Istituto Nazionale Tumori" - Milano - Sc Ematologia	Milano
Italy	Ao Regionale S. Carlo - Potenza - Sic Ematologia	Potenza
Italy	Fondazione Policlinico Universitario Agostino Gemelli Irccs	Roma
Italy	Università Degli Studi Di Roma "Sapienza" - Dipartimento Di Medicina Traslazionale E Di Precisione - U.O.C. Ematologia	Roma
Italy	Aou "San Giovanni Di Dio E Ruggi D'Aragona" - Salerno - Uoc Ematologia E Trapianti Di Cellule Staminali Emopoietiche	Salerno
Italy	Aou Di Sassari - Cliniche Universitarie - Stabilimento Cliniche Di San Pietro - Uoc Ematologia	Sassari

Sponsors (1)

Lead Sponsor	Collaborator
Gruppo Italiano Malattie EMatologiche dell'Adulto

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evaluation of efficacy of first-line therapies	Number of patients who achive complete/intermediate response	up to 3 months