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International Rare Histiocytic Disorders Registry (IRHDR) (IRHDR)

This study is currently recruiting participants.
See Contacts and Locations
Verified April 2016 by Oussama Abla, The Hospital for Sick Children
Sponsor:
Information provided by (Responsible Party):
Oussama Abla, The Hospital for Sick Children
ClinicalTrials.gov Identifier:
NCT02285582
First received: November 3, 2014
Last updated: April 25, 2016
Last verified: April 2016
  Purpose
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 Intervention
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

Study Type: Observational [Patient Registry]
Study Design: Observational Model: Case-Only
Target Follow-Up Duration: 10 Years
Official Title: International Rare Histiocytic Disorders Registry (IRHDR)

Resource links provided by NLM:


Further study details as provided by Oussama Abla, The Hospital for Sick Children:

Primary Outcome Measures:
  • 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:
  • 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 ]

Estimated Enrollment: 80
Study Start Date: October 2014
Estimated Study Completion Date: September 2024
Estimated Primary Completion Date: September 2024 (Final data collection date for primary outcome measure)
Intervention Details:
    Other: Registry study
    No intervention.
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 Rregistry (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.
  Eligibility

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02285582

Locations
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   
Sponsors and Collaborators
The Hospital for Sick Children
Investigators
Principal Investigator: Oussama Abla, MD The Hospital for Sick Children
  More Information

Publications:
Responsible Party: Oussama Abla, Staff Physician, The Hospital for Sick Children
ClinicalTrials.gov Identifier: NCT02285582     History of Changes
Other Study ID Numbers: 1000045224
Study First Received: November 3, 2014
Last Updated: April 25, 2016

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

ClinicalTrials.gov processed this record on June 23, 2017