The Myeloid Neoplasms Biology and Outcome Project

Myeloid Neoplasm Clinical Trial

— MyBOP  

Official title:

A Prospective Registry Study on Biological Disease Profile, Intervention Type and Clinical Outcome in Patients With Myeloid Neoplasms

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05074316
• Other study ID #: MyBOP
• Status: Recruiting
• Start date: March 10, 2020
• Est. completion date: December 31, 2032

Study information

• Verified date: December 2023
• Source: University Hospital Heidelberg
• Contact: Editha Gnutzmann, M.A.
• Phone: +49 6221 56 36235
• Email: editha.gnutzmann[@]med.uni-heidelberg.de
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The Myeloid Neoplasms Biology and Outcome Project (MyBOP) aims to establish a registry study for patients with myeloid neoplasms. It integrates clinical data, biological samples, socio-demographic information, long-term follow-up and patient reported outcomes in a structured manner for scientific purposes.

The ultimate benefits are:

1. Improvement of evidence-based clinical management of patients with myeloid neoplasms through better understanding of the course of disease and prognostic and predictive parameters

2. Direct access to new and personalized treatment approaches through recruitment into clinical studies based on the myeloid neoplasms study platform

3. Quality assurance of participating centers by evaluating and comparing clinical outcomes and side effects of the MyBOP patients with published data.

Description:

During recent years, considerable progress has been made in deciphering the molecular genetic and epigenetic basis of myeloid neoplasms and in defining new diagnostic and prognostic as well as predictive markers. Myeloid neoplasms are categorized according to the current WHO Classification of Tumors of Haematopoietic and Lymphoid Tissues based on the revision of 2016 [1]. This includes Myeloproliferative neoplasms (MPN), Mastocytosis, Myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRA, PDGFRB, or FGFR1, or with PCM1-JAK2, Myelodysplastic/myeloproliferative neoplasms (MDS/MPN), Myelodysplastic syndromes (MDS), Myeloid neoplasms with germ line predisposition, Acute myeloid leukemia (AML) and related neoplasms (i.e. Myeloid sarcoma and Myeloid proliferations related to Down syndrome), Blastic plasmacytoid dendritic cell neoplasm and Acute leukemia of ambiguous lineage (Table 1).

A growing number of recurring genetic changes are recognized in the current WHO 2016 classification of myeloid neoplasms [2] and additional molecularly defined subgroups as well as new entities are expected to be included in future versions. Furthermore, novel therapies are now available and being developed, which target specific genetic lesions, and several surface antigens are being explored as targets for immunotherapy-based treatment strategies, e.g. CAR-T-cell therapy [3].

Although the WHO 2016 classification represents an enormous progress in terms of reliability, validity and objectivity, there are still huge diagnostic uncertainties left [4-18] and the field of targeted therapy [19-25] in myeloid neoplasms is just at its beginning. Furthermore, clonal evolution and transition from one entity to another is a clinically relevant issue [26-30].

Thus, key areas of interest are:

• Systematic collection and evaluation of comprehensive clinical information from patients with myeloid neoplasm, including morphomolecular disease subtype, as well as drug treatments, radiation therapy, surgical procedures and long-term follow-up data

• Systematic collection and evaluation of comprehensive biological specimens and information from patients with myeloid neoplasms, including data on the genomic, transcriptomic, epigenomic and proteomic "landscapes" as well as expression of surface antigens of myeloid disease subtypes, to identify novel prognostic and predictive parameters as well as entry points for targeted therapeutic interventions

• Regular assessment of patient reported outcomes

The above challenges are ideally met by a registry study with a sufficient population size in order to answer relevant questions in rare cancer entities. The aim is to set up a registry study that covers systematic and comprehensive clinical data acquisition. In addition, the banking of tumor and germline samples from patients with myeloid neoplasms is intended by all patients. This resource will spur patient-oriented investigations into relationships between clinical and biological parameters in myeloid neoplasms and lay the groundwork for novel, molecular mechanism- and immunotherapy-based treatment approaches in poorly understood and difficult-to-treat subsets.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1000
• Est. completion date: December 31, 2032
• Est. primary completion date: July 31, 2030
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Suspected or proven diagnosis of Myeloid Neoplasms according to the WHO Classification of Tumors of Haematopoietic and Lymphoid Tissues

• Age =18 years

• Ability to understand the nature and individual consequences of the registry

• Written informed consent

• Subjects who are physically or mentally capable of giving consent

Exclusion Criteria:

Severe neurological or psychiatric disorder interfering with the ability to give written informed consent

Related Conditions & MeSH terms

• Myeloid Neoplasm
• Neoplasms

Locations

Country	Name	City	State
Germany	UHHeidelberg	Heidelberg	Baden-Württemberg

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital Heidelberg

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	median overall survival (mOS)	Time period of survival from date of diagnosis of myeloid neoplasy	5 years
Primary	overall survival (mOS)	Time period of survival from date of diagnosis of myeloid neoplasy	10 years
Primary	event free survival (EFS)	Time period of event free survival from date of diagnosis of myeloid neoplasy	5 years
Primary	progression free survival (PFS)	Time period of progression survival from date of diagnosis of myeloid neoplasy	5 years
Secondary	Questionnaire for the health- related quality of life QLQ-C30	Standardized Quality of Life Assessment, Higher values are better	5 years
Secondary	Questionnaire for physical, cognitive and emotional aspects of cancer-related fatigue QLQ-FA12	Standardized Quality of Fatigue, Higher values are worse	5 years
Secondary	Questionnaire for anxiety and depression PHQ-4	Standardized Quality of anxiety and depression, Higher values are worse	5 years
Secondary	Functional Assessment of Cancer Therapy Fact-Cog	Standardized Quality of cognitive function, Higher values are better	5 years
Secondary	The Pittsburgh Sleep Quality Index PSQI	Standardized Quality of sleep, Higher values are worse	5 years