Leukemia, Chronic MyeloidNCT04384848
The EMPATHY Pilot Study
A study testing EMPATHY Pilot in people with Leukemia, Chronic Myeloid and Cancer, run by Northwestern University at 16 sites in 2 countries.
Trial summary
The proposed study, may significantly contribute to improve healthcare delivery in patients with Chronic Myeloid Leukemia (CML) treated with modern tyrosine kinase inhibitors (TKIs) in two ways. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia, Chronic Myeloid, Cancer
- What is being tested?
- EMPATHY Pilot
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 94 participants
- Where
- 16 sitesin 2 countries
- Study dates
- 1 Nov 2019to 31 Jul 2022
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of Philadelphia chromosome positive and/or BCR-ABL positive CML confirmed by cytogenetic and/or molecular analysis;
- Newly diagnosed chronic phase (CP)-CML Patients planned to receive one of the following TKI approved as first line treatment: imatinib, dasatinib, nilotinib or bosutinib;
- Adult Patients (≥18 years) at the time of study entry; Children under the age of 18 will be excluded from the study. The exclusion of children is justified by the following circumstances: a) The condition is relatively rare in children, as compared to adults; b) Issues of study preclude direct applicability of hypotheses and/or interventions to both adults and children.
- Written informed consent.
- Written informed consent from Patient's physician as a participant.
- Newly diagnosed Chronic Phase (CP)-CML Patients who are within 4 weeks of first line TKI therapy (anyone of the TKI approved in the USA and Europe, that is: imatinib, dasatinib, nilotinib or bosutinib).
Show all 14Show fewer
- Ability to read/converse in English (Northwestern University and Augusta University Sites). Ability to read/converse in Italian (GIMEMA Centers).
- Patient exclusion criteria will include:
- Major cognitive deficits or psychiatric problems hampering a self-reported evaluation;
- Having received any CML treatment - other than TKI - for more than 3 months prior to receiving current TKI therapy.
- This project will focus on CML, which affect both men and women. Therefore, there are no exclusion/inclusion criteria based on sex/gender. In addition, there are no exclusion/inclusion criteria based on race and ethnicity.
- Physician inclusion criteria will include:
- 1)Provider of clinical care for Patient who meets inclusion criteria for the study
- Please note that Physician consent is requisite for the Patient to be enrolled as a participant in the study.
Who cannot join
- Major cognitive deficits or psychiatric problems hampering a self-reported evaluation
- Having received any CML treatment prior to therapy with imatinib, dasatinib, bosutinib or nilotinib for more than three months
Trial locations and contacts
16 sites in 2 countries.
16 sites in 2 countries
Italy14 sites
Policlinico Sant'Orsola Malpighi - UOC Ematologia - Azienda Ospedaliero-Universitaria di Bologna
Bologna
Ospedale "A. Businco" - Struttura Complessa di Ematologia e CTMO - Azienda Ospedaliera G. Brotzu
Cagliari
Milano Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - UOC Oncoematologia - Padiglione Marcora
Milan
Azienda Ospedaliera Universitaria - Università degli Studi di Napoli "Federico II" - UOC Ematologia
Naples
Azienda Ospedaliera Universitaria Maggiore della Carità di Novara - SCDU Ematologia
Novara
AUSL Reggio Emilia - Arcispedale S. Maria Nuova, IRCSS - SC Ematologia
Reggio Emilia
Azienda Ospedaliera Universitaria Policlinico Umberto I - Università degli Studi "Sapienza" - UOC Ematologia
Roma
Ospedale Sant'Eugenio
Roma
The GIMEMA Foundation (Italian Group for Adult Hematologic Diseases)
Rome, 00161
Azienda Ospedaliero-Universitario Città della Salute e della Scienza di Torino - Ospedale S. Giovanni Battista Molinette - SC Ematologia
Torino
Ospedale Mauriziano Umberto I - Torino - SCDU Ematologia
Torino
ASUI di Udine - Presidio Ospedaliero "Santa Maria della Misericordia" - Clinica Ematologica
Udine
Azienda Ospedaliera Universitario Integrata di Verona, Policlinico G.B. Rossi - UOC Ematologia
Verona
USL 6 - Ospedale San Bortolo - Vicenza
Vicenza
United States2 sites
Augusta University, Hematology and Oncology
Augusta, Georgia, 30912
Northwestern University, Robert H. Lurie Comprehensive Cancer Center Comprehensive Cancer Center (RHLCCC)
Chicago, Illinois, 60611
About this study
Official title: Evaluating Patient-Reported Outcomes Monitoring in Routine Care of Patients With Chronic Myeloid Leukemia for Increasing Adherence and Clinical Response to THerapY: The EMPATHY Pilot Study
The proposed study, may significantly contribute to improve healthcare delivery in patients with Chronic Myeloid Leukemia (CML) treated with modern tyrosine kinase inhibitors (TKIs) in two ways. First, it may provide novel empirical data on the positive effects of systematically monitoring of patient-reported adverse events (AEs) in routine practice for improving symptom management and adherence to therapy. Second, it will inform the development of a large international randomized controlled trial (RCT) to test whether systematic collection of patient-reported AEs, could improve clinical response to TKI therapy.
Read the detailed description
The evolution in the understanding of the biology of Chronic Myeloid Leukemia (CML), that eventually translated into highly effective molecular targeted therapies, is unparalleled in cancer medicine. This knowledge led the development of a number of orally active molecule tyrosine kinase inhibitors (TKIs) that have dramatically improved clinical outcomes. In less than two decades, the 10-year survival probability increased from 20 to 53% with previous (IFN)-therapies to about 90% in the TKI era. Life expectancy of these patients now approaches that of the general population. While oral TKIs are now the standard of care for CML, it should be considered that therapy is lifelong and patients are requested to take medication on a daily basis. Importantly, there is convincing evidence that full adherence to therapy is a critical factor to obtain and maintain an optimal response to therapy. However, non-adherence is a major challenge in CML, since side effects induced by these drugs negatively impact on patient's quality of life (QoL), seriously undermine full adherence with treatment schedule and thereby often lead to sub-optimal clinical responses to drugs. From previous Preliminary Data we extrapolated the following evidences: Systematic monitoring of Patient-Reported Outcomes (PRO) in routine care has several advantages, such as improved symptom control, enhanced patient-physician communication, as well as patient satisfaction and wellbeing. Furthermore, it was found that Adverse Events (AEs) are the most frequent cause for non-adherence to CML therapy and that even experienced physicians tend to underestimate burden of TKIs of their patients. This mismatch might have major clinical implications in disease management, as physicians might not be able to early identify those patients who might be at heightened risk of poor adherence behavior. Given these findings, we hypothesized that systematic electronic monitoring of Patient-Reported AEs in CML routine practice may improve adherence to therapy, quality of life, and clinical response to therapy. This hypothesis will be addressed in the experiments of the following Specific Aims: 1) To develop an online platform for systematic monitoring of patient-reported AE assessment that is tailored to the unique demands of TKI therapy for CML. 2) To assess patient and physician acceptability and satisfaction with use of this platform in CML routine practice and evaluate its value in improving symptom management, quality of life, adherence to therapy as well as preliminary efficacy. We anticipate this study will provide unprecedented information on the value of systematically collecting patient-reported AEs information in CML routine care. If positive, our results will inform the development of large international randomized controlled trial (RCT) to investigate whether this experimental approach can improve depth and rates of clinical responses to TKI therapies in CML patients.
Dr. Fabio Efficace is also a Principal Investigator on this study. He is also a Professor in the Department of Medical Social Sciences at Northwestern University, Feinberg School of Medicine.
- Conditions
- Leukemia, Chronic Myeloid, Cancer
- Being tested
- EMPATHY Pilot
- Keywords
- Cancer, Leukemia, Chronic Myeloid, Health-Related Quality Of Life, Patient Reported Outcome
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EMPATHY PilotOther
The goal of this pilot is to develop and pilot a tailored monitoring intervention targeting symptomatic, Patient-reported adverse events (AEs) in chronic myeloid leukemia (CML) Patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy. The tailored monitoring intervention will draw primarily from the PRO-CTCAE Item Library, with additional items drawn from the FACIT and EORTC Item libraries as necessary. After identifying the full set of AEs to be monitored, we will load the Patient assessment and report program into tablets for electronic administration in the busy clinic setting. We will then pilot a six-month intervention aimed to monitor and manage emerging AEs, coupled with assessment of intervention feasibility, Patient acceptability and satisfaction, provider acceptability and clinical management utility, adherence to TKI therapy, and HRQoL. We expect that adherence to therapy, clinical response, and HRQoL will be enhanced by such an intervention.
| Study group | Role | What they receive |
|---|---|---|
| Chronic myeloid leukemia (CML) patients | Experimental | CML patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy Receives: Other: EMPATHY Pilot |
What is being measured
The primary outcome is the main question the study is designed to answer.
Medication Adherence
Adherence to Refills and Medications Scale (ARMS). The ARMS-7 consists of seven items evaluating adherence to taking medications and refilling prescriptions. Items are rated on a four-point Likert scale ranging between 7 and 28, with higher scores indicating lower medication adherence.
Measured: 6 months
Number of Participants Adherent To Their Medication
Prescription refill data extracted from hospital pharmacy records. Prescription refill data were evaluated over 6 months. Missed refills, for reasons other than mortality or physician change of medication, were counted as non-adherence. Adherence was defined as the number of days medicine not available between each refill over the number of days between first prescription and last refill during the study period.
Measured: 6 months
Secondary outcomes (3)
Health-Related Quality of Life
Functional Assessment of Cancer Therapy-General. The possible score range of this measure is 0-128. Higher scores denote better quality of life
Measured: 6 months
Fatigue
Functional Assessment of Chronic Illness Therapy-Fatigue. the possible score range of this measure is 0-52. Higher scores reflect less fatigue
Measured: 6 months
Cytogenetic Response
Cytogenetic response (CyR) is defined based on the percentage of Ph pos metaphases, as evaluated by chromosome banding analysis of at least 20 marrow cell metaphases (cytogenetics). Complete CyR is when the percentage of Ph pos metaphases is 0; Partial CyR is when the percentage of Ph pos metaphases ranges from 1 to 35, Minor CyR if the percentage of Ph pos metaphases ranges from 36 to 65, minimal CyR if the percentage of Ph pos metaphases ranges from 66 to 95, No CyR if the percentage of Ph pos metaphases is higher than 95.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 May 2020
- Study start1 Nov 2019
- Primary completion10 Jun 2022
- Study completion31 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (35)Papers the sponsor has linked to this study.
- Other trials for Leukemia, Chronic MyeloidStudies of the same condition, open and under way.
- More trials run by Northwestern UniversityThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 94 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwestern University
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI), Fondazione GIMEMA, Augusta University
- Responsible party
- Principal investigator, David Cella, Northwestern University
- Investigators
- David Cella, PhD, Principal investigator, Northwestern UniversityFabio Efficace, PhD, Principal investigator, Fondazione GIMEMA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04384848
- Sponsor's protocol ID
- 1R21CA230367-01A1
- Status last verified
- Feb 2024
- First submitted
- 17 Apr 2020
- Last updated
- 4 Mar 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Druker BJ, Sawyers CL, Kantarjian H, Resta DJ, Reese SF, Ford JM, Capdeville R, Talpaz M. Activity of a specific inhibitor of the BCR-ABL tyrosine kinase in the blast crisis of chronic myeloid leukemia and acute lymphoblastic leukemia with the Philadelphia chromosome. N Engl J Med. 2001 Apr 5;344(14):1038-42. doi: 10.1056/NEJM200104053441402.
PubMed 11287973Druker BJ, Talpaz M, Resta DJ, Peng B, Buchdunger E, Ford JM, Lydon NB, Kantarjian H, Capdeville R, Ohno-Jones S, Sawyers CL. Efficacy and safety of a specific inhibitor of the BCR-ABL tyrosine kinase in chronic myeloid leukemia. N Engl J Med. 2001 Apr 5;344(14):1031-7. doi: 10.1056/NEJM200104053441401.
PubMed 11287972Rosti G, Castagnetti F, Gugliotta G, Baccarani M. Tyrosine kinase inhibitors in chronic myeloid leukaemia: which, when, for whom? Nat Rev Clin Oncol. 2017 Mar;14(3):141-154. doi: 10.1038/nrclinonc.2016.139. Epub 2016 Oct 18.
PubMed 27752053Cella D, Nowinski CJ, Frankfurt O. The impact of symptom burden on patient quality of life in chronic myeloid leukemia. Oncology. 2014;87(3):133-47. doi: 10.1159/000362816. Epub 2014 Jul 8.
PubMed 25012261Bower H, Bjorkholm M, Dickman PW, Hoglund M, Lambert PC, Andersson TM. Life Expectancy of Patients With Chronic Myeloid Leukemia Approaches the Life Expectancy of the General Population. J Clin Oncol. 2016 Aug 20;34(24):2851-7. doi: 10.1200/JCO.2015.66.2866. Epub 2016 Jun 20.
PubMed 27325849Efficace F, Baccarani M, Breccia M, Cottone F, Alimena G, Deliliers GL, Barate C, Specchia G, Di Lorenzo R, Luciano L, Turri D, Martino B, Stagno F, Dabusti M, Bergamaschi M, Leoni P, Simula MP, Levato L, Fava C, Veneri D, Sica S, Rambaldi A, Rosti G, Vignetti M, Mandelli F. Chronic fatigue is the most important factor limiting health-related quality of life of chronic myeloid leukemia patients treated with imatinib. Leukemia. 2013 Jul;27(7):1511-9. doi: 10.1038/leu.2013.51. Epub 2013 Feb 18.
PubMed 23417029Guerin A, Chen L, Ionescu-Ittu R, Marynchenko M, Nitulescu R, Hiscock R, Keir C, Wu EQ. Impact of low-grade adverse events on health-related quality of life in adult patients receiving imatinib or nilotinib for newly diagnosed Philadelphia chromosome positive chronic myelogenous leukemia in chronic phase. Curr Med Res Opin. 2014 Nov;30(11):2317-28. doi: 10.1185/03007995.2014.944973. Epub 2014 Aug 5.
PubMed 25025755Williams LA, Garcia Gonzalez AG, Ault P, Mendoza TR, Sailors ML, Williams JL, Huang F, Nazha A, Kantarjian HM, Cleeland CS, Cortes JE. Measuring the symptom burden associated with the treatment of chronic myeloid leukemia. Blood. 2013 Aug 1;122(5):641-7. doi: 10.1182/blood-2013-01-477687. Epub 2013 Jun 18.
PubMed 23777764Noens L, Hensen M, Kucmin-Bemelmans I, Lofgren C, Gilloteau I, Vrijens B. Measurement of adherence to BCR-ABL inhibitor therapy in chronic myeloid leukemia: current situation and future challenges. Haematologica. 2014 Mar;99(3):437-47. doi: 10.3324/haematol.2012.082511.
PubMed 24598855Marin D, Bazeos A, Mahon FX, Eliasson L, Milojkovic D, Bua M, Apperley JF, Szydlo R, Desai R, Kozlowski K, Paliompeis C, Latham V, Foroni L, Molimard M, Reid A, Rezvani K, de Lavallade H, Guallar C, Goldman J, Khorashad JS. Adherence is the critical factor for achieving molecular responses in patients with chronic myeloid leukemia who achieve complete cytogenetic responses on imatinib. J Clin Oncol. 2010 May 10;28(14):2381-8. doi: 10.1200/JCO.2009.26.3087. Epub 2010 Apr 12.
PubMed 20385986Noens L, van Lierde MA, De Bock R, Verhoef G, Zachee P, Berneman Z, Martiat P, Mineur P, Van Eygen K, MacDonald K, De Geest S, Albrecht T, Abraham I. Prevalence, determinants, and outcomes of nonadherence to imatinib therapy in patients with chronic myeloid leukemia: the ADAGIO study. Blood. 2009 May 28;113(22):5401-11. doi: 10.1182/blood-2008-12-196543. Epub 2009 Apr 6.
PubMed 19349618Ibrahim AR, Eliasson L, Apperley JF, Milojkovic D, Bua M, Szydlo R, Mahon FX, Kozlowski K, Paliompeis C, Foroni L, Khorashad JS, Bazeos A, Molimard M, Reid A, Rezvani K, Gerrard G, Goldman J, Marin D. Poor adherence is the main reason for loss of CCyR and imatinib failure for chronic myeloid leukemia patients on long-term therapy. Blood. 2011 Apr 7;117(14):3733-6. doi: 10.1182/blood-2010-10-309807. Epub 2011 Feb 23.
PubMed 21346253Efficace F, Cannella L. The value of quality of life assessment in chronic myeloid leukemia patients receiving tyrosine kinase inhibitors. Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):170-179. doi: 10.1182/asheducation-2016.1.170.
PubMed 27913477Experts in Chronic Myeloid Leukemia. The price of drugs for chronic myeloid leukemia (CML) is a reflection of the unsustainable prices of cancer drugs: from the perspective of a large group of CML experts. Blood. 2013 May 30;121(22):4439-42. doi: 10.1182/blood-2013-03-490003. Epub 2013 Apr 25.
PubMed 23620577Kotronoulas G, Kearney N, Maguire R, Harrow A, Di Domenico D, Croy S, MacGillivray S. What is the value of the routine use of patient-reported outcome measures toward improvement of patient outcomes, processes of care, and health service outcomes in cancer care? A systematic review of controlled trials. J Clin Oncol. 2014 May 10;32(14):1480-501. doi: 10.1200/JCO.2013.53.5948. Epub 2014 Apr 7.
PubMed 24711559
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluating Patient-Reported Outcomes Monitoring in Routine Care of Patients With Chronic Myeloid Leukemia for Increasing Adherence and Clinical Response to THerapY: The EMPATHY Pilot Study
- ClinicalTrials.gov ID
- NCT04384848
- Acronym / sponsor ID
- 1R21CA230367-01A1
- Conditions
- Leukemia, Chronic Myeloid, Cancer
- Keywords
- Cancer, Leukemia, Chronic Myeloid, Health-Related Quality Of Life, Patient Reported Outcome
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 94 participants (actual)
- Lead sponsor
- Northwestern UniversityUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI), Fondazione GIMEMA, Augusta University
- Study start
- 1 Nov 2019 (actual)
- Primary completion
- 10 Jun 2022 (actual)
- Study completion
- 31 Jul 2022 (actual)
- First posted
- 12 May 2020 (actual)
- Results
- Posted 4 Mar 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The proposed study, may significantly contribute to improve healthcare delivery in patients with Chronic Myeloid Leukemia (CML) treated with modern tyrosine kinase inhibitors (TKIs) in two ways. First, it may provide novel empirical data on the positive effects of systematically monitoring of patient-reported adverse events (AEs) in routine practice for improving symptom management and adherence to therapy. Second, it will inform the development of a large international randomized controlled trial (RCT) to test whether systematic collection of patient-reported AEs, could improve clinical response to TKI therapy.
Detailed description
The evolution in the understanding of the biology of Chronic Myeloid Leukemia (CML), that eventually translated into highly effective molecular targeted therapies, is unparalleled in cancer medicine. This knowledge led the development of a number of orally active molecule tyrosine kinase inhibitors (TKIs) that have dramatically improved clinical outcomes. In less than two decades, the 10-year survival probability increased from 20 to 53% with previous (IFN)-therapies to about 90% in the TKI era. Life expectancy of these patients now approaches that of the general population. While oral TKIs are now the standard of care for CML, it should be considered that therapy is lifelong and patients are requested to take medication on a daily basis. Importantly, there is convincing evidence that full adherence to therapy is a critical factor to obtain and maintain an optimal response to therapy. However, non-adherence is a major challenge in CML, since side effects induced by these drugs negatively impact on patient's quality of life (QoL), seriously undermine full adherence with treatment schedule and thereby often lead to sub-optimal clinical responses to drugs. From previous Preliminary Data we extrapolated the following evidences: Systematic monitoring of Patient-Reported Outcomes (PRO) in routine care has several advantages, such as improved symptom control, enhanced patient-physician communication, as well as patient satisfaction and wellbeing. Furthermore, it was found that Adverse Events (AEs) are the most frequent cause for non-adherence to CML therapy and that even experienced physicians tend to underestimate burden of TKIs of their patients. This mismatch might have major clinical implications in disease management, as physicians might not be able to early identify those patients who might be at heightened risk of poor adherence behavior. Given these findings, we hypothesized that systematic electronic monitoring of Patient-Reported AEs in CML routine practice may improve adherence to therapy, quality of life, and clinical response to therapy. This hypothesis will be addressed in the experiments of the following Specific Aims: 1) To develop an online platform for systematic monitoring of patient-reported AE assessment that is tailored to the unique demands of TKI therapy for CML. 2) To assess patient and physician acceptability and satisfaction with use of this platform in CML routine practice and evaluate its value in improving symptom management, quality of life, adherence to therapy as well as preliminary efficacy. We anticipate this study will provide unprecedented information on the value of systematically collecting patient-reported AEs information in CML routine care. If positive, our results will inform the development of large international randomized controlled trial (RCT) to investigate whether this experimental approach can improve depth and rates of clinical responses to TKI therapies in CML patients.
Dr. Fabio Efficace is also a Principal Investigator on this study. He is also a Professor in the Department of Medical Social Sciences at Northwestern University, Feinberg School of Medicine.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Chronic myeloid leukemia (CML) patients | Experimental | Other: EMPATHY PilotCML patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Diagnosis of Philadelphia chromosome positive and/or BCR-ABL positive CML confirmed by cytogenetic and/or molecular analysis;
- Newly diagnosed chronic phase (CP)-CML Patients planned to receive one of the following TKI approved as first line treatment: imatinib, dasatinib, nilotinib or bosutinib;
- Adult Patients (≥18 years) at the time of study entry; Children under the age of 18 will be excluded from the study. The exclusion of children is justified by the following circumstances: a) The condition is relatively rare in children, as compared to adults; b) Issues of study preclude direct applicability of hypotheses and/or interventions to both adults and children.
- Written informed consent.
- Written informed consent from Patient's physician as a participant.
Key exclusion criteria (2 listed)
- Major cognitive deficits or psychiatric problems hampering a self-reported evaluation
- Having received any CML treatment prior to therapy with imatinib, dasatinib, bosutinib or nilotinib for more than three months
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EMPATHY PilotThe goal of this pilot is to develop and pilot a tailored monitoring intervention targeting symptomatic, Patient-reported adverse events (AEs) in chronic myeloid leukemia (CML) Patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy. The tailored monitoring intervention will draw primarily from the PRO-CTCAE Item Library, with additional items drawn from the FACIT and EORTC Item libraries as necessary. After identifying the full set of AEs to be monitored, we will load the Patient assessment and report program into tablets for electronic administration in the busy clinic setting. We will then pilot a six-month intervention aimed to monitor and manage emerging AEs, coupled with assessment of intervention feasibility, Patient acceptability and satisfaction, provider acceptability and clinical management utility, adherence to TKI therapy, and HRQoL. We expect that adherence to therapy, clinical response, and HRQoL will be enhanced by such an intervention. | Other | Chronic myeloid leukemia (CML) patients |
Primary outcome
Medication Adherence
Time frame: 6 months
Adherence to Refills and Medications Scale (ARMS). The ARMS-7 consists of seven items evaluating adherence to taking medications and refilling prescriptions. Items are rated on a four-point Likert scale ranging between 7 and 28, with higher scores indicating lower medication adherence.
Number of Participants Adherent To Their Medication
Time frame: 6 months
Prescription refill data extracted from hospital pharmacy records. Prescription refill data were evaluated over 6 months. Missed refills, for reasons other than mortality or physician change of medication, were counted as non-adherence. Adherence was defined as the number of days medicine not available between each refill over the number of days between first prescription and last refill during the study period.
3 secondary outcomes
Health-Related Quality of Life
Time frame: 6 months
Fatigue
Time frame: 6 months
Cytogenetic Response
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 11287973Druker BJ, Sawyers CL, Kantarjian H, Resta DJ, Reese SF, Ford JM, Capdeville R, Talpaz M. Activity of a specific inhibitor of the BCR-ABL tyrosine kinase in the blast crisis of chronic myeloid leukemia and acute lymphoblastic leukemia with the Philadelphia chromosome. N Engl J Med. 2001 Apr 5;344(14):1038-42. doi: 10.1056/NEJM200104053441402.
- Background: PubMed 11287972Druker BJ, Talpaz M, Resta DJ, Peng B, Buchdunger E, Ford JM, Lydon NB, Kantarjian H, Capdeville R, Ohno-Jones S, Sawyers CL. Efficacy and safety of a specific inhibitor of the BCR-ABL tyrosine kinase in chronic myeloid leukemia. N Engl J Med. 2001 Apr 5;344(14):1031-7. doi: 10.1056/NEJM200104053441401.
- Background: PubMed 27752053Rosti G, Castagnetti F, Gugliotta G, Baccarani M. Tyrosine kinase inhibitors in chronic myeloid leukaemia: which, when, for whom? Nat Rev Clin Oncol. 2017 Mar;14(3):141-154. doi: 10.1038/nrclinonc.2016.139. Epub 2016 Oct 18.
- Background: PubMed 25012261Cella D, Nowinski CJ, Frankfurt O. The impact of symptom burden on patient quality of life in chronic myeloid leukemia. Oncology. 2014;87(3):133-47. doi: 10.1159/000362816. Epub 2014 Jul 8.
- Background: PubMed 27325849Bower H, Bjorkholm M, Dickman PW, Hoglund M, Lambert PC, Andersson TM. Life Expectancy of Patients With Chronic Myeloid Leukemia Approaches the Life Expectancy of the General Population. J Clin Oncol. 2016 Aug 20;34(24):2851-7. doi: 10.1200/JCO.2015.66.2866. Epub 2016 Jun 20.
- Background: PubMed 23417029Efficace F, Baccarani M, Breccia M, Cottone F, Alimena G, Deliliers GL, Barate C, Specchia G, Di Lorenzo R, Luciano L, Turri D, Martino B, Stagno F, Dabusti M, Bergamaschi M, Leoni P, Simula MP, Levato L, Fava C, Veneri D, Sica S, Rambaldi A, Rosti G, Vignetti M, Mandelli F. Chronic fatigue is the most important factor limiting health-related quality of life of chronic myeloid leukemia patients treated with imatinib. Leukemia. 2013 Jul;27(7):1511-9. doi: 10.1038/leu.2013.51. Epub 2013 Feb 18.
- Background: PubMed 25025755Guerin A, Chen L, Ionescu-Ittu R, Marynchenko M, Nitulescu R, Hiscock R, Keir C, Wu EQ. Impact of low-grade adverse events on health-related quality of life in adult patients receiving imatinib or nilotinib for newly diagnosed Philadelphia chromosome positive chronic myelogenous leukemia in chronic phase. Curr Med Res Opin. 2014 Nov;30(11):2317-28. doi: 10.1185/03007995.2014.944973. Epub 2014 Aug 5.
- Background: PubMed 23777764Williams LA, Garcia Gonzalez AG, Ault P, Mendoza TR, Sailors ML, Williams JL, Huang F, Nazha A, Kantarjian HM, Cleeland CS, Cortes JE. Measuring the symptom burden associated with the treatment of chronic myeloid leukemia. Blood. 2013 Aug 1;122(5):641-7. doi: 10.1182/blood-2013-01-477687. Epub 2013 Jun 18.
- Background: PubMed 24598855Noens L, Hensen M, Kucmin-Bemelmans I, Lofgren C, Gilloteau I, Vrijens B. Measurement of adherence to BCR-ABL inhibitor therapy in chronic myeloid leukemia: current situation and future challenges. Haematologica. 2014 Mar;99(3):437-47. doi: 10.3324/haematol.2012.082511.
- Background: PubMed 20385986Marin D, Bazeos A, Mahon FX, Eliasson L, Milojkovic D, Bua M, Apperley JF, Szydlo R, Desai R, Kozlowski K, Paliompeis C, Latham V, Foroni L, Molimard M, Reid A, Rezvani K, de Lavallade H, Guallar C, Goldman J, Khorashad JS. Adherence is the critical factor for achieving molecular responses in patients with chronic myeloid leukemia who achieve complete cytogenetic responses on imatinib. J Clin Oncol. 2010 May 10;28(14):2381-8. doi: 10.1200/JCO.2009.26.3087. Epub 2010 Apr 12.
- Background: PubMed 19349618Noens L, van Lierde MA, De Bock R, Verhoef G, Zachee P, Berneman Z, Martiat P, Mineur P, Van Eygen K, MacDonald K, De Geest S, Albrecht T, Abraham I. Prevalence, determinants, and outcomes of nonadherence to imatinib therapy in patients with chronic myeloid leukemia: the ADAGIO study. Blood. 2009 May 28;113(22):5401-11. doi: 10.1182/blood-2008-12-196543. Epub 2009 Apr 6.
- Background: PubMed 21346253Ibrahim AR, Eliasson L, Apperley JF, Milojkovic D, Bua M, Szydlo R, Mahon FX, Kozlowski K, Paliompeis C, Foroni L, Khorashad JS, Bazeos A, Molimard M, Reid A, Rezvani K, Gerrard G, Goldman J, Marin D. Poor adherence is the main reason for loss of CCyR and imatinib failure for chronic myeloid leukemia patients on long-term therapy. Blood. 2011 Apr 7;117(14):3733-6. doi: 10.1182/blood-2010-10-309807. Epub 2011 Feb 23.
- FACIT Searchable Library and Custom Form Developer (Build-a-PRO)Link provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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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.
David Cella, PhD principal investigator
Northwestern University
Fabio Efficace, PhD principal investigator
Fondazione GIMEMA
16 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing EMPATHY Pilot in people with Leukemia, Chronic Myeloid and Cancer, run by Northwestern University at 16 sites in 2 countries.
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 14 inclusion and 2 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 4 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in Italy, United States. 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?
Yes. Results are posted in the registry (first posted 4 Mar 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Medication Adherence (time frame: 6 months). 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 NCT04384848, which also lists 35 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 4 Mar 2024.