LeukemiaNCT01046305
Symptom Burden in Chronic Myeloid Leukemia (CML)
An observational study of Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
Objectives: The objective of this study is to measure and delineate the symptom burden experienced by patients with chronic myeloid leukemia (CML). The Primary Aim is to develop and validate an M. D. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Leukemia
- What is being tested?
- Interview & Questionnaires, Rating Questionnaire and MDASI-CML Questionnaire
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 210 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2009to 30 Nov 2020
- 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 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Chronic myeloid leukemia (CML) patients and their caregivers, 18 years of age or older, physicians and nurses.
Who may be able to join
- 18 years of age or older (Patient and family caregiver only)
- Ability to speak and read English (All participants)
- Live in the United States (Patient Phase 3 only)
- Diagnosis of Philadelphia chromosome positive CML confirmed by pathological and cytogenetic analysis (Patient only)
- Being followed at M.D. Anderson Cancer Center (Patient only)
- Consent to participate (All participants)
Show all 12Show fewer
- Treatment for CML for at least 1 year (Patient Phase 2 only)
- Family caregiver also willing to receive packet for expert panel participation (Patient Phase 2 only)
- Physician or nurse with at least 5 years experience caring for patients with CML (Professional expert only)
- At least one publication in the last 5 years dealing with CML (Physician professional expert only)
- Identification as a family caregiver by a patient with CML (Family caregiver only)
- Patient also willing to receive packet for expert panel participation (Family caregiver only)
Who cannot join
- Medical condition or impaired performance status that would preclude participation in the study (Patient only)
- Diagnosis of active psychosis or severe cognitive impairment as determined by primary physician (Patient only)
- Active treatment (systemic drug therapy or radiation therapy) for a second malignancy (Patient only Phase 3)
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: Symptom Burden of Patients With Chronic Myeloid Leukemia (CML)
Objectives:
The objective of this study is to measure and delineate the symptom burden experienced by patients with chronic myeloid leukemia (CML).
Read more
The Primary Aim is to develop and validate an M. D. Anderson Symptom Inventory (MDASI) module (the MDASI-CML), compliant with FDA standards for patient-reported outcomes (PROs), to measure the severity of multiple symptoms and the impact of these symptoms on daily functioning in patients with CML.
The Secondary Aims are:
- to develop a detailed description of the severity and interference with daily activities of symptoms experienced by patients with CML;
- to assess the impact of symptom severity in CML on standard functioning and quality of life (QOL) measures including Eastern Cooperative Oncology Group (ECOG) Performance Status and single-item QOL scale;
- to evaluate the MDASI-CML as an estimate of functional status and QOL in patients with CML;
- to identify common clusters of symptoms and symptom patterns occurring over multiple measurement time points in patients with CML;
5, to define the qualitative symptom burden of patients with CML;
- to explore the feasibility of the Interactive Voice Response (IVR) system in measuring symptom severity and interference with daily activities over time in patients with CML.
Read the detailed description
Part 1:
Interview:
If you agree to take part in this study, while you are at the clinic for a visit, you will be interviewed about what it is like to have CML. The interview should take about 30 minutes to complete.
Questionnaires:
After the interview, you will be asked to answer a symptom questionnaire, a quality-of-life questionnaire, and some demographic questions such as your age and marital status. It will take about 10 minutes to complete all 3 questionnaires.
Length of Study:
After you have completed the interview and questionnaires, your participation on this study will be over.
Additional Information:
The interview will be digitally recorded. The electronic record of the interview will not have your name linked to it and it will be kept on a secure server at M. D. Anderson Cancer Center that is password protected. All study materials will be destroyed after data analysis is completed and study results reported.
Information about your symptoms collected in this study is collected for research purposes only. If you are experiencing severe or troublesome symptoms, you should report them to your doctor or nurse as well as rating them on the symptom assessment questionnaire. If the data collector notices that you have rated a symptom as severe, the data collector will ask you if your doctor or nurse is aware of the symptom or if you intend to report the symptom to your doctor or nurse. If you have not or do not intend to report the symptom, the data collector will let you know that he or she will report the symptom to your doctor or nurse.
This is an investigational study.
Up to 218 participants will take part in this study. Up to 40 will be enrolled in Part 1 of this study. All will be enrolled at MD Anderson.
Part 3:
Symptom Questionnaires:
If you agree to take part in this study, you will complete a questionnaire about any symptoms you are having every 2 weeks for 1 year. On the questionnaire, you will be asked to rate the level of discomfort caused by the symptoms you are having and how the symptoms effect the activities of your daily life. The symptom questionnaire should take about 5 minutes to complete. You will complete the questionnaire during the first study visit and at your clinic visits every 3 months. In between visits, you will be called every 2 weeks to complete the questionnaire by an automated telephone system.
You will decide a convenient time and place for the automated phone system to call you. If you are not available when the automated system calls, it will call you again at a later time. You may also call the automated system if you miss a call. If you are not reached by the automated phone system after several tries, the study staff will call you.
You will be given a secure user name and identification number to use with the automated phone system. You will be shown how the automated phone system works by the study staff.
Two (2) days after the first visit, you will fill out the symptom questionnaire again. You will take it home with you from the clinic with a pre-addressed, stamped envelope given to you by the research staff so you can return it. The research staff will also ask for your telephone number so that they may contact you and remind you to complete the questionnaire.
Quality-of-Life/Demographic Questionnaires:
At the first visit and the 3-month clinic visits, you will also rate your overall quality-of-life. This should take about 1 minute to complete.
At the first visit, you will also be asked some demographic questions (such as your age and marital status). Answering these questions should take about 1 minute.
Opinion Questionnaire:
If you are 1 of the first 30 patients enrolled in the study, at the first visit you will also complete a questionnaire about your opinion of the symptom questionnaire. For example, you will be asked if the symptom questionnaire was easy to understand and to complete and if there were any other questions that should be included. The study staff will use your opinion to decide if other important symptom questions are missing from the questionnaire. This questionnaire should take about 5 minutes to complete.
Length of Study:
You will be on study for 1 year.
Additional Information:
The questionnaires will only use your study number and will not have your name linked to them. All questionnaires and study data will be kept in a locked cabinet at all times. All study materials will be destroyed after data analysis is completed and study results reported.
Information about your symptoms collected in this study is collected for research purposes only. If you are experiencing severe or troublesome symptoms, you should report them to your doctor or nurse as well as rating them on the symptom assessment questionnaire. If the data collector notices that you have rated a symptom as severe, the data collector will ask you if your doctor or nurse is aware of the symptom or if you intend to report the symptom to your doctor or nurse. If you have not or do not intend to report the symptom, the data collector will let you know that he or she will report the symptom to your doctor or nurse.
This is an investigational study.
Up to 218 participants will take part in this study. Up to 160 participants will be enrolled in Part 3 of this study. All will be enrolled at MD Anderson.
- Conditions
- Leukemia
- Being tested
- Interview & Questionnaires, Rating Questionnaire, MDASI-CML Questionnaire
- Keywords
- Chronic Myeloid Leukemia, CML, Symptom Burden, M. D. Anderson Symptom Inventory, MDASI-CML, Severity, Interference, Questionnaire, Interview
- Medical subject headings
- Leukemia, Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Interview & QuestionnairesBehavioural
Also called Survey
30 Minute Interview about what it is like to have CML, followed by symptom questionnaire, quality-of-life questionnaire, and demographic questions taking another 10 minutes for all 3 questionnaires.
Rating QuestionnaireBehavioural
Also called Survey, MDASI-CML
Questionnaire developed in Part 1 to rate the relevance of symptom items, given to 3-5 physicians, 3-5 nurses, 2-4 patients and their family caregivers, taking about 10 minutes.
MDASI-CML QuestionnaireBehavioural
Also called Survey, M. D. Anderson Symptom Inventory, MDASI, MDASI-CML
Patient questionnaires about CML symptoms over 1 year, completed in person or using Interactive Voice Response (IVR) system every 2 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Part 1: Interview & Questionnaires | Patients interviews and questionnaires about CML symptoms once. Receives: Behavioral: Interview & Questionnaires | |
| Part 2: Symptoms Rating | Importance of symptoms to CML patients rated by physicians, nurses, patients, and caregivers. Receives: Behavioral: Rating Questionnaire | |
| Part 3: MDASI-CML Questionnaires | Patients questionnaires about CML symptoms over 1 year using M. D. Anderson Symptom Inventory (MDASI) module (the MDASI-CML) Receives: Behavioral: MDASI-CML Questionnaire |
What is being measured
The primary outcome is the main question the study is designed to answer.
Symptom Burden Experienced by CML Patients
Measured: Patients followed over 1 year with MDASI-CML questionnaire
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jan 2010 (estimated)
- Study startNov 2009
- Primary completion30 Nov 2020 (estimated)
- Study completion30 Nov 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe 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
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 210 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Loretta A. Williams, PHD, MSN, Study chair, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01046305
- Sponsor's protocol ID
- 2008-0728
- Status last verified
- Jan 2020
- First submitted
- 8 Jan 2010
- Last updated
- 7 Jan 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
Publications
Williams 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 23777764
Study at a glance
From the registry record, last updated by the sponsor on 7 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Symptom Burden of Patients With Chronic Myeloid Leukemia (CML)
- ClinicalTrials.gov ID
- NCT01046305
- Acronym / sponsor ID
- 2008-0728
- Conditions
- Leukemia
- Keywords
- Chronic Myeloid Leukemia, CML, Symptom Burden, M. D. Anderson Symptom Inventory, MDASI-CML, Severity, Interference, Questionnaire
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 210 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Novartis Pharmaceuticals
- Study start
- Nov 2009 (actual)
- Primary completion
- 30 Nov 2020 (estimated)
- Study completion
- 30 Nov 2020 (estimated)
- First posted
- 11 Jan 2010 (estimated)
- 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.
Objectives:
The objective of this study is to measure and delineate the symptom burden experienced by patients with chronic myeloid leukemia (CML).
The Primary Aim is to develop and validate an M. D. Anderson Symptom Inventory (MDASI) module (the MDASI-CML), compliant with FDA standards for patient-reported outcomes (PROs), to measure the severity of multiple symptoms and the impact of these symptoms on daily functioning in patients with CML.
The Secondary Aims are:
- to develop a detailed description of the severity and interference with daily activities of symptoms experienced by patients with CML;
- to assess the impact of symptom severity in CML on standard functioning and quality of life (QOL) measures including Eastern Cooperative Oncology Group (ECOG) Performance Status and single-item QOL scale;
- to evaluate the MDASI-CML as an estimate of functional status and QOL in patients with CML;
- to identify common clusters of symptoms and symptom patterns occurring over multiple measurement time points in patients with CML;
5, to define the qualitative symptom burden of patients with CML;
- to explore the feasibility of the Interactive Voice Response (IVR) system in measuring symptom severity and interference with daily activities over time in patients with CML.
Detailed description
Part 1:
Interview:
If you agree to take part in this study, while you are at the clinic for a visit, you will be interviewed about what it is like to have CML. The interview should take about 30 minutes to complete.
Questionnaires:
After the interview, you will be asked to answer a symptom questionnaire, a quality-of-life questionnaire, and some demographic questions such as your age and marital status. It will take about 10 minutes to complete all 3 questionnaires.
Length of Study:
After you have completed the interview and questionnaires, your participation on this study will be over.
Additional Information:
The interview will be digitally recorded. The electronic record of the interview will not have your name linked to it and it will be kept on a secure server at M. D. Anderson Cancer Center that is password protected. All study materials will be destroyed after data analysis is completed and study results reported.
Information about your symptoms collected in this study is collected for research purposes only. If you are experiencing severe or troublesome symptoms, you should report them to your doctor or nurse as well as rating them on the symptom assessment questionnaire. If the data collector notices that you have rated a symptom as severe, the data collector will ask you if your doctor or nurse is aware of the symptom or if you intend to report the symptom to your doctor or nurse. If you have not or do not intend to report the symptom, the data collector will let you know that he or she will report the symptom to your doctor or nurse.
This is an investigational study.
Up to 218 participants will take part in this study. Up to 40 will be enrolled in Part 1 of this study. All will be enrolled at MD Anderson.
Part 3:
Symptom Questionnaires:
If you agree to take part in this study, you will complete a questionnaire about any symptoms you are having every 2 weeks for 1 year. On the questionnaire, you will be asked to rate the level of discomfort caused by the symptoms you are having and how the symptoms effect the activities of your daily life. The symptom questionnaire should take about 5 minutes to complete. You will complete the questionnaire during the first study visit and at your clinic visits every 3 months. In between visits, you will be called every 2 weeks to complete the questionnaire by an automated telephone system.
You will decide a convenient time and place for the automated phone system to call you. If you are not available when the automated system calls, it will call you again at a later time. You may also call the automated system if you miss a call. If you are not reached by the automated phone system after several tries, the study staff will call you.
You will be given a secure user name and identification number to use with the automated phone system. You will be shown how the automated phone system works by the study staff.
Two (2) days after the first visit, you will fill out the symptom questionnaire again. You will take it home with you from the clinic with a pre-addressed, stamped envelope given to you by the research staff so you can return it. The research staff will also ask for your telephone number so that they may contact you and remind you to complete the questionnaire.
Quality-of-Life/Demographic Questionnaires:
At the first visit and the 3-month clinic visits, you will also rate your overall quality-of-life. This should take about 1 minute to complete.
At the first visit, you will also be asked some demographic questions (such as your age and marital status). Answering these questions should take about 1 minute.
Opinion Questionnaire:
If you are 1 of the first 30 patients enrolled in the study, at the first visit you will also complete a questionnaire about your opinion of the symptom questionnaire. For example, you will be asked if the symptom questionnaire was easy to understand and to complete and if there were any other questions that should be included. The study staff will use your opinion to decide if other important symptom questions are missing from the questionnaire. This questionnaire should take about 5 minutes to complete.
Length of Study:
You will be on study for 1 year.
Additional Information:
The questionnaires will only use your study number and will not have your name linked to them. All questionnaires and study data will be kept in a locked cabinet at all times. All study materials will be destroyed after data analysis is completed and study results reported.
Information about your symptoms collected in this study is collected for research purposes only. If you are experiencing severe or troublesome symptoms, you should report them to your doctor or nurse as well as rating them on the symptom assessment questionnaire. If the data collector notices that you have rated a symptom as severe, the data collector will ask you if your doctor or nurse is aware of the symptom or if you intend to report the symptom to your doctor or nurse. If you have not or do not intend to report the symptom, the data collector will let you know that he or she will report the symptom to your doctor or nurse.
This is an investigational study.
Up to 218 participants will take part in this study. Up to 160 participants will be enrolled in Part 3 of this study. All will be enrolled at MD Anderson.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Part 1: Interview & Questionnaires | – | Behavioral: Interview & QuestionnairesPatients interviews and questionnaires about CML symptoms once. |
| Part 2: Symptoms Rating | – | Behavioral: Rating QuestionnaireImportance of symptoms to CML patients rated by physicians, nurses, patients, and caregivers. |
| Part 3: MDASI-CML Questionnaires | – | Behavioral: MDASI-CML QuestionnairePatients questionnaires about CML symptoms over 1 year using M. D. Anderson Symptom Inventory (MDASI) module (the MDASI-CML) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Chronic myeloid leukemia (CML) patients and their caregivers, 18 years of age or older, physicians and nurses.
Key inclusion criteria (12 listed)
- 18 years of age or older (Patient and family caregiver only)
- Ability to speak and read English (All participants)
- Live in the United States (Patient Phase 3 only)
- Diagnosis of Philadelphia chromosome positive CML confirmed by pathological and cytogenetic analysis (Patient only)
- Being followed at M.D. Anderson Cancer Center (Patient only)
Key exclusion criteria (3 listed)
- Medical condition or impaired performance status that would preclude participation in the study (Patient only)
- Diagnosis of active psychosis or severe cognitive impairment as determined by primary physician (Patient only)
- Active treatment (systemic drug therapy or radiation therapy) for a second malignancy (Patient only Phase 3)
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 |
|---|---|---|
| Interview & QuestionnairesAlso: Survey30 Minute Interview about what it is like to have CML, followed by symptom questionnaire, quality-of-life questionnaire, and demographic questions taking another 10 minutes for all 3 questionnaires. | Behavioural | Part 1: Interview & Questionnaires |
| Rating QuestionnaireAlso: Survey, MDASI-CMLQuestionnaire developed in Part 1 to rate the relevance of symptom items, given to 3-5 physicians, 3-5 nurses, 2-4 patients and their family caregivers, taking about 10 minutes. | Behavioural | Part 2: Symptoms Rating |
| MDASI-CML QuestionnaireAlso: Survey, M. D. Anderson Symptom Inventory, MDASI, MDASI-CMLPatient questionnaires about CML symptoms over 1 year, completed in person or using Interactive Voice Response (IVR) system every 2 weeks. | Behavioural | Part 3: MDASI-CML Questionnaires |
Primary outcome
Symptom Burden Experienced by CML Patients
Time frame: Patients followed over 1 year with MDASI-CML questionnaire
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jan 2020.
- Derived from this study: 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.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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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.
Loretta A. Williams, PHD, MSN study chair
M.D. Anderson Cancer Center
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 Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
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 12 inclusion and 3 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: status not confirmed, as of the sponsor's last update on 7 Jan 2020. 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 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?
Results are not available in the source record.
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?
Symptom Burden Experienced by CML Patients (time frame: Patients followed over 1 year with MDASI-CML questionnaire). 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 NCT01046305, which also lists 1 linked publication.
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 7 Jan 2020.