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NCT Number: NCT02626689

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

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.

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Key information

Conditions

Beta-Thalassemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Aghia Sofia Children's Hosptial, Athens, Goudi, Greece

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

  • 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.

Exclusion criteria

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:

  • 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.

Treatment and study plan

Quality of Life (QOL) Questionnaires

Other

Monitoring of FACT-AN, SF-36V2, TransQol, and NTD PRO assessments,

Healthcare Resource Utilization

Other

Monitoring and reviewing HealthCare Resource Utilization information.

Primary outcomes

  1. 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.

  2. 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.

Secondary outcomes

  1. 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

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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

  7. 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

  8. 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.

  9. 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)

  10. 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

  11. 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

Sponsors and collaborators

Lead sponsor

Celgene

Industry

Registry information

Official study title

Burden of Beta-Thalassemia - Quality of Life and Health Care Resource Utilization- A Prospective Observational Study

Important dates

Study start
2016
Primary completion
2017
Study completion
2017
First posted
Dec 10, 2015
Registry last updated
Apr 7, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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