Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) International Registry

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Clinical Trial

Official title:

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) International Registry

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05430971
• Other study ID #: IMMONC0002
• Status: Recruiting
• Start date: July 1, 2022
• Est. completion date: July 2032

Study information

• Verified date: August 2023
• Source: Immune Oncology Research Institute
• Contact: Astghik Voskanyan, MD
• Phone: +374 10 28 38 00
• Email: astghikvos[@]gmail.com, astghik.voskanyan[@]blood.am
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) is a very rare hematologic malignancy. Despite recent advances, at present there is no consensus on the optimal treatment of BPDCN. The optimal therapy of disease remains to be determined, and due to the rarity of cases, there is a need for international collaboration to collect data on BPDCN clinical presentations, diagnostics, treatment regimens and outcomes. Therefore, the objectives of this study are: (1) to build a large database of patients with BPDCN, (2) to investigate the characteristics and outcome of the disease with different treatment regimens, (3) to evaluate prognostic factors, and (4) to generate data-based prospective treatment recommendations.

Description:

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematologic malignancy. In 2008, it was recognized by the WHO as a distinct entity and separately listed in the group of acute myeloid leukemias and related precursor neoplasms. The final diagnosis of BPDCN relies on a compatible immunophenotype. The triple positive CD4+CD56+CD123+ phenotype associated with negativity for lineage-specific markers is a minimum requirement for defining BPDCN. The highly specific marker BDCA2/CD303, as well as other plasmacytoid dendritic cell-associated antigens (e.g. TCL1 and CD2AP), might be of great support to exclude potential mimickers of BPDCN (acute myeloid and monocytic leukemias, precursor lymphoblastic T-cell leukemia/lymphomas and T- and NK/T cell lymphomas. At present, there is no consensus on the optimal treatment of BPDCN. The majority of patients receive multi-agent chemotherapy with AML or ALL treatment regimens, while a few patients undergo allogeneic haematopoietic stem cell transplantation (HSCT). In recent years, different novel and innovative therapies are in development to target surface molecules in BPDCN. The patients are still in need of better treatments and the optimal therapy of disease remains to be determined. This is a multicenter, international prospective and retrospective registry with the aim of collecting data of patients with a diagnosis of BPDCN globally. Patients will be recruited directly by the national study groups / participating centers. Participating centers will collect and verify informed consent of all prospective patients enrolled at their center. The following data will be collected through questionnaires: 1. Patient characteristics 2. BPDCN characteristics 3. Treatment details 4. Outcomes 5. Cause of death 6. End of data collection Quality control and data management will be conducted by the Immune Oncology Research Institute.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: July 2032
• Est. primary completion date: July 2032
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Diagnosis of BPDCN
• Signed informed consent form for prospective patients Exclusion Criteria: -

Related Conditions & MeSH terms

• Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
• Neoplasms

Locations

Country	Name	City	State
Armenia	Hematology Center named after prof. R. Yeolyan	Yerevan
Canada	University of Calgary	Calgary	Alberta
Canada	Children's Hospital of Eastern Ontario, University of Ottawa	Ottawa	Ontario
Cyprus	Cyprus Society of Haematology	Nicosia
Egypt	Oncology Center, Mansoura University Faculty of Medicine	Mansoura
Georgia	M. Iashvili Children's Central Hospital	Tbilisi
India	Department of Medical Oncology, Dr. B.R.A Institute Rotary Cancer Hospital, All India Institute of Medical Sciences	New Delhi
Iraq	Pediatric Hematology Oncology, Children's Welfare Teaching Hospital, Medical City, College of Medicine, University of Baghdad	Baghdad
Italy	University of Perugia - Azienda Ospedaliera Perugia	Perugia
Italy	Department of Biomedicine and Prevention, University of Rome Tor Vergata	Roma
Kuwait	Department of hematology, Kuwait Cancer Control Center	Kuwait
Taiwan	China Medical University Children's Hospital	Taichung
Turkey	Turkish Pediatric Cancer Registry	Ankara
Turkey	Istanbul University, Oncology Institute	Istanbul
Turkey	University of Health Sciences, Umraniye Research and Education Hospital, Pediatric Hematology and Oncology Department, Pediatric Bone Marrow Transplantation Unit	Istanbul
United Kingdom	Broomfield Hospital, Haematology Mid and South Essex University Hospitals Group	Chelmsford	Essex
United States	Sylvester Comprehensive Cancer Center, University of Miami	Miami	Florida
United States	Seattle Children's Cancer and Blood Disorders Center	Seattle	Washington
United States	Moffitt Cancer Center	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Immune Oncology Research Institute

Countries where clinical trial is conducted

United States,  Armenia,  Canada,  Cyprus,  Egypt,  Georgia,  India,  Iraq,  Italy,  Kuwait,  Taiwan,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall survival	Time from diagnosis to death	5 years
Secondary	Complete remission rate	The proportion of patients with complete remission	5 years
Secondary	Mean duration of the first remission	Time from first remission to disease progression or death	5 years
Secondary	Event-free survival	Time from diagnosis to occurrence of a complication	5 years