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

April 6, 2017 updated by: Celgene

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Greece
    • Goudi
      • Athens, Goudi, Greece, 11527
        • Aghia Sofia Children's Hosptial
  • Italy
      • Milano, Italy, 20122
        • Fondazione Irccs Ca Granda Ospedale Maggiore
  • Lebanon
      • Beirut, Lebanon, 4407 2020
        • American University of Beirut Medical Center
  • Thailand
    • Bangkoknoi
      • Bangkok, Bangkoknoi, Thailand, 10700
        • Faculty of Medicine Siriraj Hospital, Mahidol University
  • Turkey
      • Cukurova, Turkey, 1330
        • Cukurova University Medical Faculty Balcali Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

β-thalassemia subjects who are transfusion dependent (TD) and non-transfusion dependent (NTD)

Description

Inclusion Criteria:

For the transfusion dependent (TD) subjects:

  1. Male or female, >18 years of age at the time of signing the informed consent document (ICF);
  2. Documented diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia;
  3. 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
  4. Performance status: Eastern Cooperative Oncology Group (ECOG) score of 0 to 1.

For the non-transfusion dependent (NTD) subpopulation

  1. Male or female, >18 years of age at the time of signing the informed consent document (ICF).
  2. Documented diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia
  3. 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);
  4. Most recent hemoglobin ≤10 g/dL (hemoglobin values ≤ 21 days post-transfusion will be excluded)
  5. Performance status: ECOG score of 0 to 1.

Exclusion Criteria:

For TD Subpopulation:

  1. A diagnosis of hemoglobin S/β-thalassemia;
  2. Any significant psychiatric or medical conditions not related to thalassemia that would prevent the subject from participating in the study;
  3. Inability to read or understand the local official languages; or
  4. Participated in another clinical trial (interventional) <30 days prior to study participation

For NTD Subpopulation:

  1. Received RBC transfusion ≤ 8 weeks prior to study enrollment;
  2. A diagnosis of hemoglobin S/β-thalassemia;
  3. Any significant psychiatric or medical conditions not related to thalassemia that would prevent the subject from participating in the study;
  4. Inability to read or understand the local official languages; or
  5. Participated in another clinical trial (interventional) <30 days prior to study participation.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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.
Other: Quality of Life (QOL) questionnaires
Monitoring of FACT-AN, SF-36V2, TransQol, and NTD PRO assessments,
Other: Healthcare Resource Utilization
Monitoring and reviewing HealthCare Resource Utilization information.
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.
Other: Quality of Life (QOL) questionnaires
Monitoring of FACT-AN, SF-36V2, TransQol, and NTD PRO assessments,
Other: Healthcare Resource Utilization
Monitoring and reviewing HealthCare Resource Utilization information.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
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
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.
Up to 6 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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
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
Up to 6 months
Change in the total scores over the study period using the Tran-QOL questionnaire
Time Frame: Up to 6 months
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.
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
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.
Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of office visits
Time Frame: Up to 6 months
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.
Up to 6 months
Number of minutes spent in sedentary, lightly active, fairly active, or very active lifestyles
Time Frame: Up to 6 months
Number of minutes a patient will spend in sedentary, lightly active, fairly active, and very active levels.
Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of prescriptions dispensed
Time Frame: Up to 6 months
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
Up to 6 months
Changes in the annual Healthcare Resource Care (HRC) involving the number of procedures undergone
Time Frame: Up to 6 months
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
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
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.
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
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)
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
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
Up to 6 months
Percentage of time spent in sedentary, lightly active, fairly active, or very active lifestyles
Time Frame: Up to 6 months
Percentage of time a patient will spend in sedentary, lightly active, fairly active, or very active lifestyles
Up to 6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Celgene

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Xiaohan (Henry) HU, MD, MPH, PhD, Celgene

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 1, 2016

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 3, 2017

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 3, 2017

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 5, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 8, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 10, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 7, 2017

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 6, 2017

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ACE-536-B-THAL-003

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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