Clinical trial NCT04313231: Sterile Inflammation and Molecular Aberrations in MDS
Official title: Sterile Inflammation and Molecular Aberrations in Myelodysplastic Syndrome: Determinants of Quality of Life and Vulnerability
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Myelodysplastic Syndromes
Keywords: anemia
health-related quality of life
inflammation
Lead sponsor: Medical University Innsbruck
Sponsor class: OTHER
Collaborators: name: University Hospital, Bonn
name: Universitätsklinikum Leipzig
First posted: 2020-03-18
Enrollment: 130
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: MDS, MDS/MPN diagnosis based on current WHO classification. CCUS and CHIP defined by Valent (Valent, Oncotarget, 2018) and by Stauder (Stauder, Blood, 2018)

Brief summary: The objective of this study is the description of the possible association between genetic mutation/aberration profiles, inflammatory tonus and clinical phenotype based on PROMs and HRQoL. Apart from gaining a better understanding of the causal correlation between genetics, sterile inflammatory processes and QoL (e.g. fatigue) in MDS, this study is supposed to identify potential novel biomarkers and, ultimately, therapeutic targets.


Eligibility criteria: Inclusion Criteria:

* Female and male patients \> 18 years
* MDS, MDS/MPN diagnosis based on current WHO classification. CCUS and CHIP defined by Valent (Valent, Oncotarget, 2018) and by Stauder (Stauder, Blood, 2018)
* Signed and dated declaration of consent by the patient according to ICH-GCP Guidelines

Exclusion Criteria:

* Any other illness, whether physical or mental, or any laboratory abnormalities which prevent a declaration of consent by the patient
* Patients with an acute and/or uncontrolled infection, including patients that are afebrile under treatment with antibiotic/antifungal/antiviral prophylactic medication
* Any pre-existing autoimmune disease requiring a systemic immunosuppression
* Steroid therapy (\>10mg Prednison/day or equivalent), regardless of its necessity up to 4 weeks before inclusion in the study
* Anamnestic and/or current therapy with hypomethylating agents (HMA) or immunomodulatory imide drugs (IMiDs)
* Status post allogenic stem cell transplantation
* Previous or ongoing chemotherapy
* Pregnancy or breastfeeding period

Primary outcomes:
measure: Definition of correlation between molecular aberrations and the sterile inflammatory tonus
timeFrame: baseline
measure: Definition how genetic aberrations and associated sterile inflammation impacts on health-related quality of life (HRQoL, e.g. fatigue) and functional activities in patients with MDS, MDS/MPN, or CHIP/CCUS
timeFrame: baseline
Secondary outcomes:
measure: Definition of correlation of sterile inflammatory tonus with clinical variables (e.g. progression to secondary acute myeloid leukemia (sAML), complications (e.g. infections), and survival)
timeFrame: baseline
Other outcomes:
Locations:
city: Innsbruck
country: Austria
facility: Medical University of Innsbruck
state: Tyrol
Study officials:
affiliation: Medical University Innsbruck
name: Domink Wolf, Univ.Prof.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Valent P, Stauder R, Theurl I, Geissler K, Sliwa T, Sperr WR, Bettelheim P, Sill H, Pfeilstocker M. Diagnosis, management and response criteria of iron overload in myelodysplastic syndromes (MDS): updated recommendations of the Austrian MDS platform. Expert Rev Hematol. 2018 Feb;11(2):109-116. doi: 10.1080/17474086.2018.1420473. Epub 2018 Jan 2.
citation: Stauder R, Valent P, Theurl I. Anemia at older age: etiologies, clinical implications, and management. Blood. 2018 Feb 1;131(5):505-514. doi: 10.1182/blood-2017-07-746446. Epub 2017 Nov 15.