Beta-ThalassemiaNCT02626689
To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care
An observational study of Beta-Thalassemia, run by Celgene at 5 sites in 5 countries.
Trial summary
This is a multi-site, prospective, observational study implemented in β-thalassemia treatment centers from 5 countries (Italy, Turkey, Greece, Lebanon, and Thailand). Approximately one to two study sites will be identified per country and approximately 20 β-thalassemia subjects will be enrolled per country (10… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- Quality of Life (QOL) questionnaires and Healthcare Resource Utilization
- How many people
- 100 participants
- Where
- 5 sitesin 5 countries
- Study dates
- 1 Mar 2016to 3 Jan 2017
- 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
- No healthy volunteers
- Adult, Older adult
Study population: β-thalassemia subjects who are transfusion dependent (TD) and non-transfusion dependent (NTD)
Who may be able to join
- For the transfusion dependent (TD) subjects:
- Male or female, >18 years of age at the time of signing the informed consent document (ICF);
- Documented diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia;
- TD - defined as: ≥6 Red Blood Cells (RBC) units in the 24 weeks prior to study participation and no transfusion free period for ≥35 days during that period; and
- Performance status: Eastern Cooperative Oncology Group (ECOG) score of 0 to 1.
- For the non-transfusion dependent (NTD) subpopulation
Show all 11Show fewer
- Male or female, >18 years of age at the time of signing the informed consent document (ICF).
- Documented diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia
- NTD - defined as: 0 to 5 RBC units received during the 24-week period prior to study participation (not including RBC transfusions units administered for elective surgery);
- Most recent hemoglobin ≤10 g/dL (hemoglobin values ≤ 21 days post-transfusion will be excluded)
- Performance status: ECOG score of 0 to 1.
Who cannot join
- For TD Subpopulation:
- A diagnosis of hemoglobin S/β-thalassemia;
- Any significant psychiatric or medical conditions not related to thalassemia that would prevent the subject from participating in the study;
- Inability to read or understand the local official languages; or
- Participated in another clinical trial (interventional) <30 days prior to study participation
- For NTD Subpopulation:
Show all 11Show fewer
- Received RBC transfusion ≤ 8 weeks prior to study enrollment;
- A diagnosis of hemoglobin S/β-thalassemia;
- Any significant psychiatric or medical conditions not related to thalassemia that would prevent the subject from participating in the study;
- Inability to read or understand the local official languages; or
- Participated in another clinical trial (interventional) <30 days prior to study participation.
Trial locations and contacts
5 sites in 5 countries.
5 sites in 5 countries
Thailand1 site
Faculty of Medicine Siriraj Hospital, Mahidol University
Bangkok, Bangkoknoi, 10700
Turkey (Türkiye)1 site
Cukurova University Medical Faculty Balcali Hospital
Çukurova, 1330
About this study
Official title: Burden of Beta-Thalassemia - Quality of Life and Health Care Resource Utilization- A Prospective Observational Study
This is a multi-site, prospective, observational study implemented in β-thalassemia treatment centers from 5 countries (Italy, Turkey, Greece, Lebanon, and Thailand). Approximately one to two study sites will be identified per country and approximately 20 β-thalassemia subjects will be enrolled per country (10 transfusion dependent (TD) and 10 Non-transfusion dependent (NTD) with a total of approximately 100 subjects. This study will not interfere with or influence the routine clinical management of β-thalassemia patients. Outcomes of interest will be collected prospectively for up to 6 months.
- Conditions
- Beta-Thalassemia
- Being tested
- Quality of Life (QOL) questionnaires, Healthcare Resource Utilization
- Keywords
- Beta-Thalassemia, Quality of Life, Healthcare Utilization, Transfusion dependent (TD) β-thalassemia, Non-transfusion dependent β-thalassemia, Standard of care, TransQoL, SF36v2, Fact-An, NTD beta-thalassemia PRO
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Quality of Life (QOL) questionnairesOther
Monitoring of FACT-AN, SF-36V2, TransQol, and NTD PRO assessments,
Healthcare Resource UtilizationOther
Monitoring and reviewing HealthCare Resource Utilization information.
| Study group | Role | What they receive |
|---|---|---|
| β-thalassemia transfusion dependent subjects | Participants will complete 3 quality of life instruments (i.e. FACT-AN, the SF-36v2, and the TranQol) once every 3 weeks, in addition to a TranQol instrument on the day of a RBC transfusion. Continuous monitoring of Healthcare Resource Utilization will be conducted throughout the course of the study at each clinical visit. Receives: Other: Quality of Life (QOL) questionnaires; Other: Healthcare Resource Utilization | |
| β-thalassemia Non Transfusion Dependent (NTD) subjects | Participants will complete 2 quality of life instruments (i.e. FACT-An and the the SF-36v2l) once every 3 weeks, in addition to completing the non-transfusion dependent Patient Recorded Outcome (PRO) tool on a daily basis. Continuous monitoring of Healthcare Resource Utilization will be conducted throughout the course of the study at each clinical visit. Receives: Other: Quality of Life (QOL) questionnaires; Other: Healthcare Resource Utilization |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in the Physical component score (PSC) over the study period versus the country specific population norms using the 36-item Short Form (SF-36) Quality of Life instrument
Short Form 36-Item Health Survey, Version 2 (SF-36) is a self-administered instrument that measures the impact of disease on overall quality of life and consists of 36 questions in eight domains (physical function, pain, general and mental health, vitality, social function, physical and emotional health). Norm-based scores were used in analyses, calibrated so that 50 is the average score and the standard deviation equals 10. Higher scores indicate a higher level of functioning. The physical functioning domain assesses limitations in physical activities because of health problems. A positive change from Baseline score indicates an improvement.
Measured: Up to 6 months
Change in the Mental component score (MCS) over the study period versus the country specific population norms using the 36-item Short Form (SF-36) Quality of Life instrument
Short Form 36-Item Health Survey, Version 2 (SF-36) is a self-administered instrument that measures the impact of disease on overall quality of life and consists of 36 questions in eight domains (physical function, pain, general and mental health, vitality, social function, physical and emotional health). Norm-based scores were used in analyses, calibrated so that 50 is the average score and the standard deviation equals 10. Higher scores indicate a higher level of functioning. The physical functioning domain assesses limitations in physical activities because of health problems. A positive change from Baseline score indicates an improvement.
Measured: Up to 6 months
Secondary outcomes (11)
Change in the total scores over the study period using the The Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire
The FACT-An questionaire is used to assess leath-related quality of life (HRQol). It measures the impact of fatigue and other anemia-related symptoms on patient functioning. The overall score range for the FACT-An is 0-188. Higher scores indicate better HRQoL. In addition to general HRQoL, the FACT-An measures the impact of fatigue and other anemia-related symptoms on patient functioning. Patients with higher hemoglobin levels and better performance status reported significantly higher scores on these instruments (including the newly created subscales) than did those with lower hemoglobin levels and poorer performance status
Measured: Up to 6 months
Change in the total scores over the study period using the Tran-QOL questionnaire
The Tran Qol is a new disease specific QOL measure for adults with B-Thalassemia with a score ranging from 0-100. It measures the quality of life issues of patients with B-Thalassemia by assessing physical health, emotional health, family functioning and school/career functioning. Higher scores indicate better health related quality of life (HRQOL). A positive change from baseline indicates an improvement in overall HRQol.
Measured: Up to 6 months
Change in the total scores over the study period using the Patient Reported Outcome (PRO) questionnaire in non-transfusion dependent (NTD) subjects
For NTD subjects, they will complete a Patient Reported Outcome (PRO) PRO via e-diary each evening before bedtime. For all subjects, physical activities will be recorded continuously via Fitbit for the entire study period.
Measured: Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of office visits
Healthcare resource utilization will be measured by the number of office visits a B-thal patient has over the course of 6 months while involved in the study.
Measured: Up to 6 months
Number of minutes spent in sedentary, lightly active, fairly active, or very active lifestyles
Number of minutes a patient will spend in sedentary, lightly active, fairly active, and very active levels.
Measured: Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of prescriptions dispensed
Healthcare resource utilization will be measured by the number of prescriptions dispensed for a B-thal patient over the course of 6 months while involved in the study
Measured: Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of procedures undergone
Healthcare resource utilization will be measured by the number of procedures undergone for a B-thal patient over the course of 6 months while involved in the study
Measured: Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of lab assessments completed
Healthcare resource utilization will be measured by the number of lab assessments completed for a B-thal patient over the course of 6 months while involved in the study, relating to the following lab assessments: hematology, clinical chemistry, renal, hepatic, iron parameters, endocrine parameters, and urinalysis.
Measured: Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of days that a patient is hospitalized
Healthcare resource utilization will be measured by the number of days the B-thal patient was hospitalized over the course of 6 months while involved with the study, including the number of days that the patient was in the Intensive Care Unit (ICU)
Measured: Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of emergency room visits
Healthcare resource utilization will be measured by the number of emergency room visits the B-thal patient had over the course of the 6 months while involved with the study
Measured: Up to 6 months
Percentage of time spent in sedentary, lightly active, fairly active, or very active lifestyles
Percentage of time a patient will spend in sedentary, lightly active, fairly active, or very active lifestyles
Measured: Up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2015 (estimated)
- Study start1 Mar 2016
- Primary completion3 Jan 2017
- Study completion3 Jan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-ThalassemiaStudies of the same condition, open and under way.
- More trials run by CelgeneThe 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Celgene
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Xiaohan (Henry) HU, MD, MPH, PhD, Study director, Celgene
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02626689
- Sponsor's protocol ID
- ACE-536-B-THAL-003
- Status last verified
- Apr 2017
- First submitted
- 5 Nov 2015
- Last updated
- 7 Apr 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Burden of Beta-Thalassemia - Quality of Life and Health Care Resource Utilization- A Prospective Observational Study
- ClinicalTrials.gov ID
- NCT02626689
- Acronym / sponsor ID
- ACE-536-B-THAL-003
- Conditions
- Beta-Thalassemia
- Keywords
- Beta-Thalassemia, Quality of Life, Healthcare Utilization, Transfusion dependent (TD) β-thalassemia, Non-transfusion dependent β-thalassemia, Standard of care, TransQoL, SF36v2
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- CelgeneIndustry (pharma, biotech, devices)
- Study start
- 1 Mar 2016 (actual)
- Primary completion
- 3 Jan 2017 (actual)
- Study completion
- 3 Jan 2017 (actual)
- First posted
- 10 Dec 2015 (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.
This is a multi-site, prospective, observational study implemented in β-thalassemia treatment centers from 5 countries (Italy, Turkey, Greece, Lebanon, and Thailand). Approximately one to two study sites will be identified per country and approximately 20 β-thalassemia subjects will be enrolled per country (10 transfusion dependent (TD) and 10 Non-transfusion dependent (NTD) with a total of approximately 100 subjects. This study will not interfere with or influence the routine clinical management of β-thalassemia patients. Outcomes of interest will be collected prospectively for up to 6 months.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| β-thalassemia transfusion dependent subjects | – | Other: Quality of Life (QOL) questionnaires, Other: Healthcare Resource UtilizationParticipants will complete 3 quality of life instruments (i.e. FACT-AN, the SF-36v2, and the TranQol) once every 3 weeks, in addition to a TranQol instrument on the day of a RBC transfusion. Continuous monitoring of Healthcare Resource Utilization will be conducted throughout the course of the study at each clinical visit. |
| β-thalassemia Non Transfusion Dependent (NTD) subjects | – | Other: Quality of Life (QOL) questionnaires, Other: Healthcare Resource UtilizationParticipants will complete 2 quality of life instruments (i.e. FACT-An and the the SF-36v2l) once every 3 weeks, in addition to completing the non-transfusion dependent Patient Recorded Outcome (PRO) tool on a daily basis. Continuous monitoring of Healthcare Resource Utilization will be conducted throughout the course of the study at each clinical visit. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- β-thalassemia subjects who are transfusion dependent (TD) and non-transfusion dependent (NTD)
Key inclusion criteria (11 listed)
- Male or female, >18 years of age at the time of signing the informed consent document (ICF);
- Documented diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia;
- TD - defined as: ≥6 Red Blood Cells (RBC) units in the 24 weeks prior to study participation and no transfusion free period for ≥35 days during that period; and
- Performance status: Eastern Cooperative Oncology Group (ECOG) score of 0 to 1.
- For the non-transfusion dependent (NTD) subpopulation
Key exclusion criteria (11 listed)
- A diagnosis of hemoglobin S/β-thalassemia;
- Any significant psychiatric or medical conditions not related to thalassemia that would prevent the subject from participating in the study;
- Inability to read or understand the local official languages; or
- Participated in another clinical trial (interventional) <30 days prior to study participation
- Received RBC transfusion ≤ 8 weeks prior to study enrollment;
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 |
|---|---|---|
| Quality of Life (QOL) questionnairesMonitoring of FACT-AN, SF-36V2, TransQol, and NTD PRO assessments, | Other | β-thalassemia Non Transfusion Dependent (NTD) subjects, β-thalassemia transfusion dependent subjects |
| Healthcare Resource UtilizationMonitoring and reviewing HealthCare Resource Utilization information. | Other | β-thalassemia Non Transfusion Dependent (NTD) subjects, β-thalassemia transfusion dependent subjects |
Primary outcome
Change in the Physical component score (PSC) over the study period versus the country specific population norms using the 36-item Short Form (SF-36) Quality of Life instrument
Time frame: Up to 6 months
Short Form 36-Item Health Survey, Version 2 (SF-36) is a self-administered instrument that measures the impact of disease on overall quality of life and consists of 36 questions in eight domains (physical function, pain, general and mental health, vitality, social function, physical and emotional health). Norm-based scores were used in analyses, calibrated so that 50 is the average score and the standard deviation equals 10. Higher scores indicate a higher level of functioning. The physical functioning domain assesses limitations in physical activities because of health problems. A positive change from Baseline score indicates an improvement.
Change in the Mental component score (MCS) over the study period versus the country specific population norms using the 36-item Short Form (SF-36) Quality of Life instrument
Time frame: Up to 6 months
Short Form 36-Item Health Survey, Version 2 (SF-36) is a self-administered instrument that measures the impact of disease on overall quality of life and consists of 36 questions in eight domains (physical function, pain, general and mental health, vitality, social function, physical and emotional health). Norm-based scores were used in analyses, calibrated so that 50 is the average score and the standard deviation equals 10. Higher scores indicate a higher level of functioning. The physical functioning domain assesses limitations in physical activities because of health problems. A positive change from Baseline score indicates an improvement.
11 secondary outcomes
Change in the total scores over the study period using the The Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire
Time frame: Up to 6 months
Change in the total scores over the study period using the Tran-QOL questionnaire
Time frame: Up to 6 months
Change in the total scores over the study period using the Patient Reported Outcome (PRO) questionnaire in non-transfusion dependent (NTD) subjects
Time frame: Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of office visits
Time frame: Up to 6 months
Number of minutes spent in sedentary, lightly active, fairly active, or very active lifestyles
Time frame: Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of prescriptions dispensed
Time frame: Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of procedures undergone
Time frame: Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of lab assessments completed
Time frame: Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of days that a patient is hospitalized
Time frame: Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of emergency room visits
Time frame: Up to 6 months
Percentage of time spent in sedentary, lightly active, fairly active, or very active lifestyles
Time frame: Up to 6 months
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 7 Apr 2017.
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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.
Xiaohan (Henry) HU, MD, MPH, PhD study director
Celgene
5 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Beta-Thalassemia, run by Celgene at 5 sites in 5 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 11 inclusion and 11 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 7 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Greece, Italy, Lebanon, Thailand, Turkey (Türkiye). 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?
Change in the Physical component score (PSC) over the study period versus the country specific population norms using the 36-item Short Form (SF-36) Quality of Life instrument (time frame: Up to 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?
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 Apr 2017.