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Trial record 1 of 1 for:    NCT02285582
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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

Tracking Information
First Submitted Date November 3, 2014
First Posted Date November 7, 2014
Last Update Posted Date April 20, 2018
Study Start Date October 2014
Estimated Primary Completion Date September 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: November 4, 2014)
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 ]
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT02285582 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: November 4, 2014)
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 ]
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title International Rare Histiocytic Disorders Registry (IRHDR)
Official Title International Rare Histiocytic Disorders Registry (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 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]
Study Design Observational Model: Case-Only
Time Perspective: Other
Target Follow-Up Duration 10 Years
Biospecimen Not Provided
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
Condition
  • 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)
Intervention Other: Registry study
No intervention.
Study Groups/Cohorts Not Provided
Publications * Weitzman S, Jaffe R. Uncommon histiocytic disorders: the non-Langerhans cell histiocytoses. Pediatr Blood Cancer. 2005 Sep;45(3):256-64. Review.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: November 4, 2014)
80
Original Estimated Enrollment Same as current
Estimated Study Completion Date September 2024
Estimated Primary Completion Date September 2024   (Final data collection date for primary outcome measure)
Eligibility 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
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Listed Location Countries Argentina,   Austria,   Canada,   Czechia,   Germany,   Italy,   Poland,   Russian Federation,   Spain,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02285582
Other Study ID Numbers 1000045224
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Oussama Abla, The Hospital for Sick Children
Study Sponsor The Hospital for Sick Children
Collaborators Not Provided
Investigators
Principal Investigator: Oussama Abla, MD The Hospital for Sick Children
PRS Account The Hospital for Sick Children
Verification Date April 2018