The Myeloid Neoplasms Biology and Outcome Project

Studien-Informationen Einschlusskriterien Studien-Rationale

Rekrutierend

NCT-Nummer:
NCT05074316

Studienbeginn:
März 2020

Letztes Update:
12.12.2023

Wirkstoff:
-

Indikation (Clinical Trials):
Neoplasms

Geschlecht:
Alle

Altersgruppe:
Erwachsene (18+)

Phase:
-

Sponsor:
University Hospital Heidelberg

Collaborator:
-

Studienleiter

Richard F Schlenk, M.D.
Principal Investigator
University Hospital Heidelberg, Department of Internal Medicine V, German Cancer Research Center

Kontakt

Editha Gnutzmann, M.A.
Kontakt:
Phone: +49 6221 56 36235
E-Mail: editha.gnutzmann@med.uni-heidelberg.de» Kontaktdaten anzeigen

Studienlocations
(1 von 1)

UHHeidelberg
69120 Heidelberg
(Baden-Württemberg)
GermanyRekrutierend» Google-Maps
Ansprechpartner:
Editha Gnutzmann
Phone: +4962215636235
E-Mail: editha.gnutzmann@med.uni-heidelberg.de» Ansprechpartner anzeigen

Studien-Informationen

Detailed 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.

Ein-/Ausschlusskriterien

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

Studien-Rationale

Primary outcome:

1. median overall survival (mOS) (Time Frame - 5 years):
Time period of survival from date of diagnosis of myeloid neoplasy

2. overall survival (mOS) (Time Frame - 10 years):
Time period of survival from date of diagnosis of myeloid neoplasy

3. event free survival (EFS) (Time Frame - 5 years):
Time period of event free survival from date of diagnosis of myeloid neoplasy

4. progression free survival (PFS) (Time Frame - 5 years):
Time period of progression survival from date of diagnosis of myeloid neoplasy

Secondary outcome:

1. Questionnaire for the health- related quality of life QLQ-C30 (Time Frame - 5 years):
Standardized Quality of Life Assessment, Higher values are better

2. Questionnaire for physical, cognitive and emotional aspects of cancer-related fatigue QLQ-FA12 (Time Frame - 5 years):
Standardized Quality of Fatigue, Higher values are worse

3. Questionnaire for anxiety and depression PHQ-4 (Time Frame - 5 years):
Standardized Quality of anxiety and depression, Higher values are worse

4. Functional Assessment of Cancer Therapy Fact-Cog (Time Frame - 5 years):
Standardized Quality of cognitive function, Higher values are better

5. The Pittsburgh Sleep Quality Index PSQI (Time Frame - 5 years):
Standardized Quality of sleep, Higher values are worse

Quelle: ClinicalTrials.gov

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