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International Rare Histiocytic Disorders Registry (IRHDR) (IRHDR)

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ClinicalTrials.gov Identifier: NCT02285582
Recruitment Status : Recruiting
First Posted : November 7, 2014
Last Update Posted : April 20, 2018
Sponsor:
Information provided by (Responsible Party):
Oussama Abla, The Hospital for Sick Children

Brief Summary:
The rare histiocytic disorders (RHDs) are characterized by the infiltration of one or more organs by non-LCH histiocytes. They can range from localized disease that resolves spontaneously, to progressive disseminated forms that can be sometimes life-threatening. Since they are extremely rare, there is limited understanding of their causes and best treatment options. Physicians, patients and parents of children with RHDs frequently consult members of the Histiocyte Society regarding the best management of these disorders. Very often, no specific recommendation can be made due to the lack of prospective outcome data, or even large retrospective case series. The creation of an international rare histiocytic disorders registry (IRHDR) could facilitate a uniform diagnosis of the RHDs, as well as the collection and analysis of the clinical, epidemiological, treatment and survival data of patients with RHD. The registry may also lead to future therapeutic recommendations, provide a framework for future clinical trials and create excellent research opportunities.

Condition or disease Intervention/treatment
Rare Histiocytic Disorders (RHDs) Juvenile Xanthogranuloma (JXG) Reticulohistiocytoma (Epithelioid Histiocytoma) Xanthoma Disseminatum (XD) Multicentric Reticulohistiocytosis (MRH) Systemic Juvenile Xanthogranuloma Erdheim-Chester Disease (ECD) Multi-system Rosai-Dorfman Disease (RDD) Other: Registry study

Detailed Description:
Histiocytoses are rare diseases caused by an excess of cells called Histiocytes, which can infiltrate the skin, bones, lungs, liver, spleen and the central nervous system. These disorders can range from localized involvement that resolves spontaneously, to progressive disseminated forms that can be debilitating and sometimes life-threatening. The rare histiocytic disorders (RHD), or non-Langerhans cell disorders, are a diverse group of disorders defined by the accumulation of histiocytes that do not meet the criteria for Langerhans cell histiocytosis (LCH) or hemophagocytic lymphohistiocytosis (HLH). They include: Juvenile xanthogranuloma family, Erdheim-Chester disease, Multifocal Reticulohistiocytosis, Rosai-Dorfman disease and the Malignant Histiocytoses. Since they are so rare, there is limited understanding of their causes and treatments. Physicians, patients and parents of children with rare histiocytoses frequently consult members of the Histiocyte Society on the management of these disorders. Very often, no specific recommendation about treatment can be made due to the lack of prospective outcome data for these rare entities. The creation of an International Rare Histiocytic Disorders Registry (IRHDR) will facilitate a uniform diagnosis of the RHD's, as well as the collection and analysis of the clinical, epidemiological, treatment and survival data of patients with RHD. The registry will also provide expert pathology reviews and may lead to future therapeutic recommendations. Furthermore, the IRHDR can provide a framework for future clinical trials, thus, creating excellent research opportunities. Lastly, a de-identified link between clinical data and companion biology studies can potentially be accomplished in the future through the IRHDR. This may further help in understanding the etiology of these rare diseases, as well as identifying potential therapeutic targets.

Study Type : Observational [Patient Registry]
Estimated Enrollment : 80 participants
Observational Model: Case-Only
Time Perspective: Other
Target Follow-Up Duration: 10 Years
Official Title: International Rare Histiocytic Disorders Registry (IRHDR)
Study Start Date : October 2014
Estimated Primary Completion Date : September 2024
Estimated Study Completion Date : September 2024



Intervention Details:
    Other: Registry study
    No intervention.


Primary Outcome Measures :
  1. Collecting data on disease presentation, treatments used and treatment outcomes over time for patients diagnosed with RHD to better understand the diseases and optimize the treatments [ Time Frame: Data will be analysed on average yearly, the registry will be ongoing for 10 years ]

Secondary Outcome Measures :
  1. Develop treatment guidelines for the RHD based on solid clinical trial data [ Time Frame: Data will be analysed on average yearly, the registry will be ongoing for 10 years ]


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Patient with rare histiocytic disorder.

Rare histiocytic disorders include:

  1. Cutaneous non-LCH: such as the Juvenile Xanthogranuloma (JXG) family and reticulohistiocytoma (epithelioid histiocytoma).
  2. Cutaneous with a major systemic component

    • Xanthoma disseminatum (XD)
    • Multicentric reticulohistiocytosis (MRH)
  3. Systemic non-LCH

    • Systemic juvenile xanthogranuloma
    • Erdheim-Chester disease (ECD)
    • Multi-system Rosai-Dorfman disease (RDD).
  4. Malignant Histiocytoses

    • Histiocytic sarcoma
    • Langerhans cell sarcoma
    • Interdigitating dendritic cell sarcoma
    • Follicular dendritic cell sarcoma
    • Indeterminate dendritic cell tumour
    • Fibroblastic reticular cell tumour
Criteria

Inclusion Criteria:

  • Any age at diagnosis.
  • Diagnosis of a rare histiocytic disorder, established before or after the opening of the registry.
  • Cases diagnosed from January - 01- 1995 until the present time and prospectively.
  • Suitable pathology sample available for central review.
  • Signed informed consent by a patient, or parent/legal guardian.
  • Cognitively impaired patients can be included after consent by legal guardian/parent.
  • Deceased patients can be included provided that they are contacted at least 6 months after the death of their child and not on their child's birthday or anniversary of death.

Exclusion Criteria:

  • Informed consent has not been signed.
  • Diagnosis other than RHD.
  • Patients with no pathology sample available for central review.
  • Cases diagnosed before the year 1995

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02285582


Locations
United States, California
Children's Hospital of Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Rima Jubran, MD    323-361-4624      
Principal Investigator: Rima Jubran, MD         
Valley Children's Hospital Recruiting
Madera, California, United States, 93636
Contact: David Samuel, MD    559-353-5488      
Principal Investigator: David Samuel, MD         
United States, New York
Memorial Sloan Kettering Cancer Center Recruiting
New York, New York, United States, 10022
Contact: Eli Diamond, MD    212-610-0188      
Principal Investigator: Eli Diamond, MD         
Staten Island University Hospital Recruiting
Staten Island, New York, United States, 10305
Contact: Sarah Vaiselbuh, MD    718-226-6129      
Principal Investigator: Sarah Vaiselbuh, MD         
Argentina
Hospital Nacional de Pediatria Garrahan Recruiting
Buenos Aires, Argentina
Contact: Jorge Braier, MD    5411 41226254      
Principal Investigator: Jorge Braier, MD         
Austria
ELISABETHINEN Hospital Linz GmbH Recruiting
Linz, Austria
Contact: Michael Girschikofsky, MD    0043 732 7676 - 4425      
Principal Investigator: Michael Girschikofsky, MD         
Canada, Ontario
The Hospital for Sick Children Recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Oussama Abla, MD    416-813-7879    oussama.abla@sickkids.ca   
Contact: Prerna Chopra    416-813-7654 ext 202043    prerna.chopra@sickkids.ca   
Canada, Quebec
Centre hospitalier universitaire Sainte-Justine Recruiting
Montréal, Quebec, Canada, H3T 1C5
Contact: Anne-Sophie Carret, MD       anne-sophie.carret@umontreal.ca   
Principal Investigator: Anne-Sophie Carret, MD         
Czechia
University Hospital Brno Recruiting
Brno, Czechia
Contact: Zdenka Křenová, MD       Krenova.Z@seznam.cz   
Principal Investigator: Zdenka Křenová, MD         
Germany
University Children's Hospital Rostock Not yet recruiting
Rostock, Germany
Contact: Carl Friedrich Classen, MD       carl-friedrich.classen@med.uni-rostock.de   
Principal Investigator: Carl Friedrich Classen, MD         
Italy
Azienda Ospedaliero-Universitaria Meyer Not yet recruiting
Florence, Italy
Contact: Elena Sieni, MD       elena.sieni@meyer.it   
Principal Investigator: Elena Sieni, MD         
Poland
Children's Memorial Health Institute Recruiting
Warsaw, Poland
Contact: Olga Gryniewicz-Kwiatkowska, MD       O.Gryniewicz@IPCZD.PL   
Principal Investigator: Olga Gryniewicz-Kwiatkowska, MD         
Russian Federation
Morozov Children's Hospital Not yet recruiting
Moscow, Russian Federation
Contact: Gleb Bronin, MD       gleb-bronin@yandex.ru   
Principal Investigator: Gleb Bronin, MD         
Spain
Hospital Universitario Cruces Recruiting
Barakaldo, Spain
Contact: Itziar Astigarraga, MD    34 946006000      
Principal Investigator: Itziar Astigarraga, MD         
Sponsors and Collaborators
The Hospital for Sick Children
Investigators
Principal Investigator: Oussama Abla, MD The Hospital for Sick Children

Publications:
Responsible Party: Oussama Abla, Staff Physician, The Hospital for Sick Children
ClinicalTrials.gov Identifier: NCT02285582     History of Changes
Other Study ID Numbers: 1000045224
First Posted: November 7, 2014    Key Record Dates
Last Update Posted: April 20, 2018
Last Verified: April 2018

Additional relevant MeSH terms:
Erdheim-Chester Disease
Disease
Histiocytoma
Granuloma
Xanthomatosis
Xanthogranuloma, Juvenile
Histiocytosis, Sinus
Histiocytosis
Histiocytosis, Non-Langerhans-Cell
Pathologic Processes
Neoplasms, Fibrous Tissue
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms
Lymphatic Diseases
Lymphoproliferative Disorders
Lipid Metabolism Disorders
Metabolic Diseases
Skin Diseases