International Rare Histiocytic Disorders Registry (IRHDR)

Juvenile Xanthogranuloma (JXG) Clinical Trial

— IRHDR  

Official title:

International Rare Histiocytic Disorders Registry (IRHDR)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02285582
• Other study ID #: 1000045224
• Status: Active, not recruiting
• Start date: October 2014
• Est. completion date: September 2024

Study information

• Verified date: December 2022
• Source: The Hospital for Sick Children
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 169
• Est. completion date: September 2024
• Est. primary completion date: September 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Erdheim-Chester Disease
• Erdheim-Chester Disease (ECD)
• Granuloma
• Histiocytoma
• Juvenile Xanthogranuloma (JXG)
• Multi-system Rosai-Dorfman Disease (RDD)
• Multicentric Reticulohistiocytosis (MRH)
• Rare Histiocytic Disorders (RHDs)
• Reticulohistiocytoma (Epithelioid Histiocytoma)
• Systemic Juvenile Xanthogranuloma
• Xanthoma Disseminatum (XD)

Intervention

Other:
• Registry study
No intervention.

Locations

Country	Name	City	State
Argentina	Hospital Nacional de Pediatria Garrahan	Buenos Aires
Austria	ELISABETHINEN Hospital Linz GmbH	Linz
Canada	Centre hospitalier universitaire Sainte-Justine	Montréal	Quebec
Canada	The Hospital for Sick Children	Toronto	Ontario
Czechia	University Hospital Brno	Brno
Germany	University Children's Hospital Rostock	Rostock
Italy	Azienda Ospedaliero-Universitaria Meyer	Florence
Poland	Children's Memorial Health Institute	Warsaw
Russian Federation	Morozov Children's Hospital	Moscow
Spain	Hospital Universitario Cruces	Barakaldo
United States	Children's Hospital of Los Angeles	Los Angeles	California
United States	Valley Children's Hospital	Madera	California
United States	Memorial Sloan Kettering Cancer Center	New York	New York
United States	Staten Island University Hospital	Staten Island	New York

Sponsors (1)

Lead Sponsor	Collaborator
The Hospital for Sick Children

Countries where clinical trial is conducted

United States,  Argentina,  Austria,  Canada,  Czechia,  Germany,  Italy,  Poland,  Russian Federation,  Spain, 

References & Publications (1)

Weitzman S, Jaffe R. Uncommon histiocytic disorders: the non-Langerhans cell histiocytoses. Pediatr Blood Cancer. 2005 Sep;45(3):256-64. doi: 10.1002/pbc.20246. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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		Data will be analysed on average yearly, the registry will be ongoing for 10 years
Secondary	Develop treatment guidelines for the RHD based on solid clinical trial data		Data will be analysed on average yearly, the registry will be ongoing for 10 years