Ignite Creation Date:
2024-05-06 @ 3:24 AM
Last Modification Date:
2024-10-26 @ 11:33 AM
Study NCT ID:
NCT02285582
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2022-12-15
First Post:
2014-11-03
Brief Title:
International Rare Histiocytic Disorders Registry IRHDR
Sponsor:
The Hospital for Sick Children
Organization:
The Hospital for Sick Children
Study Overview
Official Title:
International Rare Histiocytic Disorders Registry IRHDR
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2022-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
IRHDR
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
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 RHDs 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None