NCT04676945

Brief Summary

A comparison of treated vs untreated patients with MDS with a sample size of approximately 8000 patients in 11 countries.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
9,179

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Aug 2020

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 21, 2020

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

November 26, 2020

Completed
25 days until next milestone

First Posted

Study publicly available on registry

December 21, 2020

Completed
8 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 20, 2021

Completed
1.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 21, 2022

Completed
Last Updated

November 22, 2022

Status Verified

November 1, 2022

Enrollment Period

12 months

First QC Date

November 26, 2020

Last Update Submit

November 21, 2022

Conditions

Myelodysplastic Syndromes

Keywords

MDSIPSS-R

Outcome Measures

Primary Outcomes (2)

  • 1.Overall Survival

    In \*overall survival\* two possible events are defined: * \*death\* (regarded as complete observation) * \*end of follow up\* (regarded as censored observation) Time is calculated from diagnosis to the first occurrence of one of the above listed events.

    From date of diagnosis until the date of death or lost to follow-up, whichever came first. No administrative censoring will be applied to the retrospectively collected data, a minimum period of two months of stable disease will be required.

  • 2.Time to transformation

    In \*time to transformation\* three possible events are defined: * \*transformation into AML\* (regarded as complete observation) * \*death without transformation\* (regarded as censored observation) * \*end of follow up\* (regarded as censored observation) Time is calculated from diagnosis to the first occurrence of one of the above listed events. In case of \*transformation into AML\* this results in a complete observation, in case of \*death without transformation\* or \*end of follow up\* the observation is treated as censored.

    From date of diagnosis until the date of transformation,death or lost to follow-up, whichever came first. No administrative censoring will be applied to the retrospectively collected data,a minimum period of two months of stable disease will be required.

Study Arms (2)

untreated control group

NO MDS disease modifying therapy

treated patients

ANY MDS disease modifying therapy

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

MDS (all subtypes and risk groups) according to WHO or oligoblastic AML (RAEB-T according to FAB) patients untreated and treated during their chronic MDS phase Population derived from registries of participating centres.

You may qualify if:

  • MDS (all subtypes and risk groups) according to WHO or oligoblastic AML (RAEB-T according to FAB) patients untreated and treated during their chronic MDS phase

You may not qualify if:

  • None

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hanusch Krankenhaus, 3.Medizinische Abteilung

Vienna, 1140, Austria

Location

Related Publications (7)

  • Greenberg PL, Tuechler H, Schanz J, Sanz G, Garcia-Manero G, Sole F, Bennett JM, Bowen D, Fenaux P, Dreyfus F, Kantarjian H, Kuendgen A, Levis A, Malcovati L, Cazzola M, Cermak J, Fonatsch C, Le Beau MM, Slovak ML, Krieger O, Luebbert M, Maciejewski J, Magalhaes SM, Miyazaki Y, Pfeilstocker M, Sekeres M, Sperr WR, Stauder R, Tauro S, Valent P, Vallespi T, van de Loosdrecht AA, Germing U, Haase D. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012 Sep 20;120(12):2454-65. doi: 10.1182/blood-2012-03-420489. Epub 2012 Jun 27.

    PMID: 22740453BACKGROUND

  • Lamarque M, Raynaud S, Itzykson R, Thepot S, Quesnel B, Dreyfus F, Rauzy OB, Turlure P, Vey N, Recher C, Dartigeas C, Legros L, Delaunay J, Visanica S, Stamatoullas A, Fenaux P, Ades L. The revised IPSS is a powerful tool to evaluate the outcome of MDS patients treated with azacitidine: the GFM experience. Blood. 2012 Dec 13;120(25):5084-5. doi: 10.1182/blood-2012-09-453555. No abstract available.

    PMID: 23243156BACKGROUND

  • Montalban-Bravo G, Garcia-Manero G. Myelodysplastic syndromes: 2018 update on diagnosis, risk-stratification and management. Am J Hematol. 2018 Jan;93(1):129-147. doi: 10.1002/ajh.24930.

    PMID: 29214694BACKGROUND

  • Sekeres MA, Swern AS, Fenaux P, Greenberg PL, Sanz GF, Bennett JM, Dreyfus F, List AF, Li JS, Sugrue MM. Validation of the IPSS-R in lenalidomide-treated, lower-risk myelodysplastic syndrome patients with del(5q). Blood Cancer J. 2014 Aug 29;4(8):e242. doi: 10.1038/bcj.2014.62. No abstract available.

    PMID: 25171203BACKGROUND

  • Della Porta MG, Alessandrino EP, Bacigalupo A, van Lint MT, Malcovati L, Pascutto C, Falda M, Bernardi M, Onida F, Guidi S, Iori AP, Cerretti R, Marenco P, Pioltelli P, Angelucci E, Oneto R, Ripamonti F, Bernasconi P, Bosi A, Cazzola M, Rambaldi A; Gruppo Italiano Trapianto di Midollo Osseo. Predictive factors for the outcome of allogeneic transplantation in patients with MDS stratified according to the revised IPSS-R. Blood. 2014 Apr 10;123(15):2333-42. doi: 10.1182/blood-2013-12-542720. Epub 2014 Feb 20.

    PMID: 24558201BACKGROUND

  • Pfeilstocker M, Tuchler H, Schonmetzler A, Nosslinger T, Pittermann E. Time changes in predictive power of established and recently proposed clinical, cytogenetical and comorbidity scores for Myelodysplastic Syndromes. Leuk Res. 2012 Feb;36(2):132-9. doi: 10.1016/j.leukres.2011.09.007. Epub 2011 Oct 2.

    PMID: 21967831BACKGROUND

  • Pfeilstocker M, Tuechler H, Sanz G, Schanz J, Garcia-Manero G, Sole F, Bennett JM, Bowen D, Fenaux P, Dreyfus F, Kantarjian H, Kuendgen A, Malcovati L, Cazzola M, Cermak J, Fonatsch C, Le Beau MM, Slovak ML, Levis A, Luebbert M, Maciejewski J, Machherndl-Spandl S, Magalhaes SM, Miyazaki Y, Sekeres MA, Sperr WR, Stauder R, Tauro S, Valent P, Vallespi T, van de Loosdrecht AA, Germing U, Haase D, Greenberg PL. Time-dependent changes in mortality and transformation risk in MDS. Blood. 2016 Aug 18;128(7):902-10. doi: 10.1182/blood-2016-02-700054. Epub 2016 Jun 22.

    PMID: 27335276BACKGROUND

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Univ.Prof.Dr.med.univ.

Study Record Dates

First Submitted

November 26, 2020

First Posted

December 21, 2020

Study Start

August 21, 2020

Primary Completion

August 20, 2021

Study Completion

November 21, 2022

Last Updated

November 22, 2022

Record last verified: 2022-11

Data Sharing

IPD Sharing
Will not share

Locations

AU(1)