Recruiting

Observational Study

Sponsor:

The Hospital for Sick Children

Code:

NCT02285582

Conditions

Rare Histiocytic Disorders (RHDs)

Erdheim-Chester Disease (ECD)

Rosai-Dorfman Disease (RDD)

Xanthogranuloma Family (XG)

Indeterminate Dendritic Cell Histiocytosis

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

Registry study

Study Details

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.

Conditions

Rare Histiocytic Disorders (RHDs)

Erdheim-Chester Disease (ECD)

Rosai-Dorfman Disease (RDD)

Xanthogranuloma Family (XG)

Indeterminate Dendritic Cell Histiocytosis

Study ID

NCT02285582

Start date

Oct, 2014

Status verified date

Jun, 2025

Completion date

Sep, 2028

Anticipated

Primary completion date

Sep, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Inclusion Criteria:

1. Any age at diagnosis.
2. Diagnosis of a rare histiocytic disorder, established before or after the opening of the registry.
3. Cases diagnosed from January - 01- 1995 until the present time and prospectively.
4. Signed informed consent by a patient, or parent/legal guardian.
5. Cognitively impaired patients can be included after consent by legal guardian/parent.
6. Deceased patients can be included if 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:

1. Informed consent has not been signed.
2. Diagnosis other than RHD.
3. Cases diagnosed before the year 1995.

Study Design

Enrollment

400 participants

Anticipated

Interventions and Outcome Measures

Interventions

Registry study

No intervention.

Primary outcome measure

  • 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. ]

Central Contacts and Locations

Central contacts

Oussama Abla, MD

416-813-7879oussama.abla@sickkids.ca

Locations

The University of Alabama at Birmingham

Recruiting

Birmingham, Alabama, United States

Contacts

Gaurav Goyal, MD

ggoyal@uabmc.edu

Principal Investigator:

Gaurav Goyal

Children's Hospital of Los Angeles

Recruiting

Los Angeles, California, United States, 90027

Contacts

Rima Jubran, MD

323-361-4624rjubran@chla.usc.edu

Principal Investigator:

Rima Jubran, MD

Valley Children's Hospital

Recruiting

Madera, California, United States, 93636

Contacts

Faisal Razzaqi, MD

559-353-3000frazzaqi@valleychildrens.org

Principal Investigator:

Faizal Razzaqi, MD

Dana-Farber Cancer Institute

Recruiting

Boston, Massachusetts, United States, 02215

Contacts

Barbara Degar, MD

barbara_degar@dfci.harvard.edu

Principal Investigator:

Barbara Degar, MD

Memorial Sloan Kettering Cancer Center

Recruiting

New York, New York, United States, 10022

Contacts

Eli Diamond, MD

212-610-0188diamone1@mskcc.org

Principal Investigator:

Eli Diamond, MD

University of Pittsburgh Medical Center

Recruiting

Pittsburgh, Pennsylvania, United States, 15219

Contacts

Steven Allen, MD

steven.allen@chp.edu

Principal Investigator:

Steven Allen, MD

The Hospital for Sick Children

Recruiting

Toronto, Ontario, Canada, M5G 1X8

Contacts

Oussama Abla, MD

416-813-7879oussama.abla@sickkids.ca

Arnelle Lardizabal, HBSc

416-813-6431arnelle.lardizabal@sickkids.ca

More Information

Sponsor

The Hospital for Sick Children

Last update posted

Jun 24, 2025

Last verified

Jun, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by The Hospital for Sick Children on 2025-06-24.