Austrian Myeloid Registry

Myeloid Diseases Clinical Trial

— aMYELOIDr  

Official title:

Austrian Myeloid Registry

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04438889
• Other study ID #: AGMT_aMYELOIDr
• Status: Recruiting
• Start date: July 13, 2020
• Est. completion date: April 2030

Study information

• Verified date: March 2024
• Source: Arbeitsgemeinschaft medikamentoese Tumortherapie
• Contact: Lisa Pleyer, MD
• Phone: +43676899758271
• Email: dr.lisa.pleyer[@]gmail.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

The Austrian Myeloid Registry (aMYELOIDr) is a non-interventional study. It collects data from patients with the myeloid diseases, primarily myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML).The aMYELOIDr is multi-center database collecting data at various sites in Austria and potentially also at other centers in other countries in future. The registry has an electronic case report form (eCRF), where all data is entered by clinical trial personnel and/or physicians. It is set up to collect real-world experience in the management of patients with these diseases in Austria.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 3000
• Est. completion date: April 2030
• Est. primary completion date: April 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Age >17 years

• Diagnosis of myeloid disease according to WHO 2016

• Signed patient informed consent (IC)

Exclusion Criteria:

• Patient is unable or unwilling to sign IC

Related Conditions & MeSH terms

• Myeloid Diseases

Intervention

Other:
• Non-interventional
Only routine clinical data, which has already been recorded in the patient's medical chart, will be documented. Any other data assessments (e.g. quality of life analyses such as EQ-5D and QLQ-C30 have been approved by the Ehtikkommission für das Bundesland Salzburg and are optional.

Locations

Country	Name	City	State
Austria	LKH Feldkirch, Innere Medizin II, Interne E: Hämatologie und Onkologie	Feldkirch
Austria	KH der Barmherzigen Brüder, Innere Medizin I	Graz
Austria	Medizinische Universität Graz, Universitätsklinik für Innere Medizin, Klinische Abteilung für Hämatologie	Graz
Austria	Universitätsklinik Innsbruck, Univ.-Klinik für Innere Medizin V, Hämatologie und Onkologie	Innsbruck
Austria	Universitätsklinikum Krems, Innere Medizin II Hämato-Onkologie	Krems
Austria	LKH Hochsteiermark, Abteilung für Hämato-Onkologie	Leoben
Austria	Kepler Universitätsklinikum Linz, Med. Campus III., Univ.-Klinik für Hämatologie und Internistische Onkologie	Linz
Austria	Ordensklinikum Linz GmbH, Barmherzige Schwestern, Interne I: Internistische Onkologie, Hämatologie und Gastroenterologie	Linz
Austria	Ordensklinikum Linz GmbH, Elisabethinen, I. Interne Abteilung Hämato-Onkologie	Linz
Austria	Universitätsklinik für Innere Med. III, PMU Salzburg	Salzburg
Austria	Universitätsklinikum St. Pölten, Klinische Abteilung für Innere Medizin 1	St. Pölten
Austria	Klinikum Steyr, Innere Medizin II	Steyr
Austria	Klinik Donaustadt: 2. Medizinische Abteilung	Vienna
Austria	Klinikum Wels-Grieskirchen, Abteilung für Innere Medizin IV	Wels
Austria	Hanusch KH, 3. Med. Abteilung	Wien
Austria	Klinik Ottakring, 1. Med. Abteilung	Wien

Sponsors (1)

Lead Sponsor	Collaborator
Arbeitsgemeinschaft medikamentoese Tumortherapie

Country where clinical trial is conducted

Austria, 

References & Publications (19)

Almeida AM, Prebet T, Itzykson R, Ramos F, Al-Ali H, Shammo J, Pinto R, Maurillo L, Wetzel J, Musto P, Van De Loosdrecht AA, Costa MJ, Esteves S, Burgstaller S, Stauder R, Autzinger EM, Lang A, Krippl P, Geissler D, Falantes JF, Pedro C, Bargay J, Deben G — View Citation

Falantes J, Pleyer L, Thepot S, Almeida AM, Maurillo L, Martinez-Robles V, Stauder R, Itzykson R, Pinto R, Venditti A, Bargay J, Burgstaller S, Martinez MP, Seegers V, Cortesao E, Foncillas MA, Gardin C, Montesinos P, Musto P, Fenaux P, Greil R, Sanz MA, — View Citation

Huemer F, Weiss L, Faber V, Neureiter D, Egle A, Geissler K, Voskova D, Zebisch A, Burgstaller S, Pichler A, Stauder R, Sperr W, Lang A, Pfeilstocker M, Machherndl-Spandl S, Stampfl M, Greil R, Pleyer L. Establishment and validation of a novel risk model — View Citation

Jansko-Gadermeir B, Leisch M, Gassner FJ, Zaborsky N, Dillinger T, Hutter S, Risch A, Melchardt T, Egle A, Drost M, Larcher-Senn J, Greil R, Pleyer L. Myeloid NGS Analyses of Paired Samples from Bone Marrow and Peripheral Blood Yield Concordant Results: A — View Citation

Leisch M, Pfeilstocker M, Stauder R, Heibl S, Sill H, Girschikofsky M, Stampfl-Mattersberger M, Tinchon C, Hartmann B, Petzer A, Schreder M, Kiesl D, Vallet S, Egle A, Melchardt T, Piringer G, Zebisch A, Machherndl-Spandl S, Wolf D, Keil F, Drost M, Greil — View Citation

Leisch M, Weiss L, Lindlbauer N, Jungbauer C, Egle A, Rohde E, Greil R, Grabmer C, Pleyer L. Red blood cell alloimmunization in 184 patients with myeloid neoplasms treated with azacitidine - A retrospective single center experience. Leuk Res. 2017 Aug;59: — View Citation

Melchardt T, Weiss L, Pleyer L, Steinkirchner S, Auberger J, Hopfinger G, Greil R, Egle A. Complications of 5-azacytidine: Three cases of severe ischemic colitis in elderly patients with myelodysplastic syndrome. Oncol Lett. 2013 Dec;6(6):1756-1758. doi: — View Citation

Pleyer L, Burgstaller S, Girschikofsky M, Linkesch W, Stauder R, Pfeilstocker M, Schreder M, Tinchon C, Sliwa T, Lang A, Sperr WR, Krippl P, Geissler D, Voskova D, Schlick K, Thaler J, Machherndl-Spandl S, Theiler G, Eckmullner O, Greil R. Azacitidine in — View Citation

Pleyer L, Burgstaller S, Stauder R, Girschikofsky M, Sill H, Schlick K, Thaler J, Halter B, Machherndl-Spandl S, Zebisch A, Pichler A, Pfeilstocker M, Autzinger EM, Lang A, Geissler K, Voskova D, Geissler D, Sperr WR, Hojas S, Rogulj IM, Andel J, Greil R. — View Citation

Pleyer L, Dohner H, Dombret H, Seymour JF, Schuh AC, Beach CL, Swern AS, Burgstaller S, Stauder R, Girschikofsky M, Sill H, Schlick K, Thaler J, Halter B, Machherndl Spandl S, Zebisch A, Pichler A, Pfeilstocker M, Autzinger EM, Lang A, Geissler K, Voskova — View Citation

Pleyer L, Germing U, Sperr WR, Linkesch W, Burgstaller S, Stauder R, Girschikofsky M, Schreder M, Pfeilstocker M, Lang A, Sliwa T, Geissler D, Schlick K, Placher-Sorko G, Theiler G, Thaler J, Mitrovic M, Neureiter D, Valent P, Greil R. Azacitidine in CMML — View Citation

Pleyer L, Heibl S, Tinchon C, Vallet S, Schreder M, Melchardt T, Stute N, Fohrenbach Quiroz KT, Leisch M, Egle A, Scagnetti L, Wolf D, Beswick R, Drost M, Larcher-Senn J, Grochtdreis T, Vaisband M, Hasenauer J, Zaborsky N, Greil R, Stauder R. Health-Relat — View Citation

Pleyer L, Leisch M, Kourakli A, Padron E, Maciejewski JP, Xicoy Cirici B, Kaivers J, Ungerstedt J, Heibl S, Patiou P, Hunter AM, Mora E, Geissler K, Dimou M, Jimenez Lorenzo MJ, Melchardt T, Egle A, Viniou AN, Patel BJ, Arnan M, Valent P, Roubakis C, Bern — View Citation

Pleyer L, Sekeres MA. An early glimpse at azacitidine plus venetoclax for myelodysplastic syndromes. Lancet Haematol. 2022 Oct;9(10):e714-e716. doi: 10.1016/S2352-3026(22)00252-6. Epub 2022 Sep 2. No abstract available. — View Citation

Pleyer L, Stauder R, Burgstaller S, Schreder M, Tinchon C, Pfeilstocker M, Steinkirchner S, Melchardt T, Mitrovic M, Girschikofsky M, Lang A, Krippl P, Sliwa T, Egle A, Linkesch W, Voskova D, Angermann H, Greil R. Azacitidine in patients with WHO-defined — View Citation

Pleyer L, Vaisband M, Drost M, Pfeilstocker M, Stauder R, Heibl S, Sill H, Girschikofsky M, Stampfl-Mattersberger M, Pichler A, Hartmann B, Petzer A, Schreder M, Schmitt CA, Vallet S, Melchardt T, Zebisch A, Pichler P, Zaborsky N, Machherndl-Spandl S, Wol — View Citation

Ramos F, Thepot S, Pleyer L, Maurillo L, Itzykson R, Bargay J, Stauder R, Venditti A, Seegers V, Martinez-Robles V, Burgstaller S, Recher C, Deben G, Gaidano G, Gardin C, Musto P, Greil R, Sanchez-Guijo F, Fenaux P; European ALMA Investigators. Azacitidin — View Citation

Valentiny C, Mitrovic M, Pleyer L, Steurer M, Willenbacher W, Stauder R. Complete remission after a single cycle of azacitidine in a case of relapsed acute myeloid leukemia. Wien Klin Wochenschr. 2013 Jan;125(1-2):50-3. doi: 10.1007/s00508-012-0319-6. Epu — View Citation

Weiss L, Melchardt T, Neureiter D, Kemmerling R, Moshir S, Pleyer L, Greil R, Egle A. Complete remission of Waldenstrom macroglobulinemia with azacitidine and rituximab. J Clin Oncol. 2011 Aug 20;29(24):e696-8. doi: 10.1200/JCO.2011.35.8283. Epub 2011 Jul — View Citation

* Note: There are 19 references in all — Click here to view all references

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Minimal residual disease	Assess how many and which patients in Austria have minimal residual disease (MRD) assessments, assessed by which techniques and at which timepoints.	Through study completion, median expected within 100 months
Other	MRD-negativity on OS	Assess the impact of MRD-negativity on OS. MRD assessment via flow-cytometry and polymerase chain reactions according to the current ELN guidelines 2017 shall be exclusively based on the risk/benefit estimation of the treating physician.	Through study completion, median expected within 100 months
Other	Prognostic and predictive markers	Analyses of various factors known or thought to influence OS, ORR, EFS, PFS and time to next treatment in order to validate existing or establish novel prognostic and predictive markers	Through study completion, median expected within 100 months
Primary	To assess the treatment patterns (therapeutic landscape) of patients with myeloid diseases.	Due to the non-interventional nature of the aMYELOIDr, treatment indication, the decision to offer treatment, treatment choice, dose, schedule and dose reductions/escalations shall be exclusively based on the risk/benefit estimation of the treating physician. We recommend compliance current guidelines.	Through study completion, median expected within 100 months
Secondary	Impact of front-line treatment on overall survival (OS)		Through study completion, median expected within 100 months
Secondary	Impact of number and choice of treatment lines on OS as of initial diagnosis and/or as of treatment start		Through study completion, median expected within 100 months
Secondary	Overall response rate (ORR)	Response will be assessed according to current guidelines for the respective disease. Due to the non-interventional nature of the aMYELOIDr, timepoints and types of response assessment shall be exclusively based on the risk/benefit estimation of the treating physician. We recommend compliance current guidelines.	Through study completion, median expected within 100 months
Secondary	Event free survival (EFS)	Events include treatment failure, progressive disease, relapse after CR/CRi, death from any cause. Patients lost to-follow-up or still alive ans without event will be censored at last follow-up date	Through study completion, median expected within 100 months
Secondary	AML transformation	Time to transformation to AML for patients with a non-AML initial diagnosis	Through study completion, median expected within 100 months
Secondary	Treatment safety	Investigators should report adverse reactions (for which a causal role of a medicine is suspected) to the concerned competent authorities following regulations in the current or future versions of Austrian legislation (Pharmakovigilanz-Verordnung 2013 (PhVO, Regulation on Pharmacovigilance), Österreichisches Arzneimittel Gesetz (AMG, Austrian Medicinal Products Act). Participation in this registry does not exempt the participating center from their legal reporting obligations. Documentation of causality, duration, frequency and severity of adverse events (AEs) according to Common Terminology Criteria for AE (CTCAE v.5).	Through study completion, median expected within 100 months
Secondary	Concomitant treatments	Concomitant treatments (number of administration of e.g. prophylactic antibiotics, antivirals, antifungals)	Through study completion, median expected within 100 months
Secondary	Treatment characteristics	Among others, the following treatment characteristics will be assessed for each treatment line: substance	Through study completion, median expected within 100 months
Secondary	Treatment characteristics	Among others, the following treatment characteristics will be assessed for each treatment line: application date	Through study completion, median expected within 100 months
Secondary	Treatment characteristics	Among others, the following treatment characteristics will be assessed for each treatment line: route	Through study completion, median expected within 100 months
Secondary	Treatment characteristics	Among others, the following treatment characteristics will be assessed for each treatment line: dose	Through study completion, median expected within 100 months
Secondary	Treatment characteristics	Among others, the following treatment characteristics will be assessed for each treatment line: inpatient or outpatient setting	Through study completion, median expected within 100 months
Secondary	Concomitant treatments best supportive care (BSC)	Concomitant best supportive care (BSC) measures (e.g. number of transfusions, growth factors, iron chelators)	Through study completion, median expected within 100 months
Secondary	Quality of life assessment EQ-5D-5L (optional)	EuroQol-5 Dimensions with 5 Levels. The descriptive system comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The patient is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the five dimensions.	Through study completion, median expected within 100 months
Secondary	EORTC Quality of life assessment QLQ-C30 (optional)	European Organisation for Research and Treatment of Cancer Quality of life 30-item questionnaire of cancer patients incorporates five functional scales (physical, role, cognitive, emotional, and social), three symptom scales (fatigue, pain, and nausea and vomiting), a global health status / QoL scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnoea, loss of appetite, insomnia, constipation anddiarrhoea) and perceived financial impact of the disease. It has four-point scale which are coded with "Not at all", "A little", "Quite a bit" and "Very much"	Through study completion, median expected within 100 months

External Links

•   Sponsor website