Myelodysplastic SyndromesNCT04676945
Analysis of Risk in MDS Over Time - Comparison of Treated vs Untreated Patients
An observational study of Myelodysplastic Syndromes, run by Michael Pfeilstöcker at 1 site in Austria.
Trial summary
A comparison of treated vs untreated patients with MDS with a sample size of approximately 8000 patients in 11 countries. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Myelodysplastic Syndromes
- How many people
- 9,179 participants
- Where
- 1 siteAustria
- Study dates
- 21 Aug 2020to 21 Nov 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- None
Trial locations and contacts
1 site in 1 country.
About this study
A comparison of treated vs untreated patients with MDS with a sample size of approximately 8000 patients in 11 countries.
Read the detailed description
Non-interventional multicentre retrospective study using chart reviews - study was submitted to the ethics committee of city of Vienna - due to the design of the study no specific approval necessary.
Inclusion of MDS (all subtypes and risk groups) patients according to WHO or oligoblastic AML (RAEB-T according to FAB), untreated and treated during their chronic MDS phase
The 26 centers of the IWG-PM have provided control data set of untreated, all centers will be asked for contribution of treated patients Data will be collected in Austria, Brazil, Czech Republic, France, Germany, Italy, Japan, Netherlands, Spain, UK, USA.
- Conditions
- Myelodysplastic Syndromes
- Keywords
- MDS, IPSS-R
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| untreated control group | NO MDS disease modifying therapy | |
| treated patients | ANY MDS disease modifying therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2020
- Study start21 Aug 2020
- Primary completion20 Aug 2021
- Study completion21 Nov 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 9,179 participants (actual)
Sponsor and investigators
- Lead sponsor
- Michael Pfeilstöcker
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene Corporation
- Responsible party
- Sponsor-investigator, Michael Pfeilstöcker, Mein Hanusch-Krankenhaus
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04676945
- Sponsor's protocol ID
- N/A-NI-MDSAML-PI-14023
- Status last verified
- Nov 2022
- First submitted
- 26 Nov 2020
- Last updated
- 22 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 22740453Lamarque 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.
PubMed 23243156Montalban-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.
PubMed 29214694Sekeres 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.
PubMed 25171203Della 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.
PubMed 24558201Pfeilstocker 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.
PubMed 21967831Pfeilstocker 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.
PubMed 27335276
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Analysis of Risk in MDS Over Time - Comparison of Treated vs Untreated Patients
- ClinicalTrials.gov ID
- NCT04676945
- Acronym / sponsor ID
- N/A-NI-MDSAML-PI-14023
- Conditions
- Myelodysplastic Syndromes
- Keywords
- MDS, IPSS-R
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 9,179 participants (actual)
- Lead sponsor
- Michael PfeilstöckerUniversity, hospital or other
- Collaborators
- Celgene Corporation
- Study start
- 21 Aug 2020 (actual)
- Primary completion
- 20 Aug 2021 (actual)
- Study completion
- 21 Nov 2022 (actual)
- First posted
- 21 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A comparison of treated vs untreated patients with MDS with a sample size of approximately 8000 patients in 11 countries.
Detailed description
Non-interventional multicentre retrospective study using chart reviews - study was submitted to the ethics committee of city of Vienna - due to the design of the study no specific approval necessary.
Inclusion of MDS (all subtypes and risk groups) patients according to WHO or oligoblastic AML (RAEB-T according to FAB), untreated and treated during their chronic MDS phase
The 26 centers of the IWG-PM have provided control data set of untreated, all centers will be asked for contribution of treated patients Data will be collected in Austria, Brazil, Czech Republic, France, Germany, Italy, Japan, Netherlands, Spain, UK, USA.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| untreated control group | – | –NO MDS disease modifying therapy |
| treated patients | – | –ANY MDS disease modifying therapy |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (1 listed)
- 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
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
1.Overall Survival
Time frame: 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.
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.
2.Time to transformation
Time frame: 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.
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2022.
- Background: PubMed 22740453Greenberg 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.
- Background: PubMed 23243156Lamarque 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.
- Background: PubMed 29214694Montalban-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.
- Background: PubMed 25171203Sekeres 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.
- Background: PubMed 24558201Della 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.
- Background: PubMed 21967831Pfeilstocker 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.
- Background: PubMed 27335276Pfeilstocker 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myelodysplastic Syndromes, run by Michael Pfeilstöcker at 1 site in Austria.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Austria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
1.Overall Survival (time frame: 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.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04676945, which also lists 7 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Nov 2022.