To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) ß-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02626689
• Date of registration: 05/11/2015
• Prospective Registration: Yes
• Primary sponsor: Celgene
• Public title: To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) ß-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care
• Scientific title: Burden of Beta-Thalassemia - Quality of Life and Health Care Resource Utilization- A Prospective Observational Study
• Date of first enrolment: March 1, 2016
• Target sample size: 100
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02626689
• Study type: Observational
• Phase: N/A

Countries of recruitment

Greece, Italy, Lebanon, Thailand, Turkey

Contacts

• Name: Xiaohan (Henry) HU, MD, MPH, PhD
• Affiliation: Celgene

Key inclusion & exclusion criteria

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 ß-thalassemia or hemoglobin E/ ß-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 ß-thalassemia or hemoglobin E/ ß-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/ß-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/ß-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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Beta-Thalassemia

Intervention(s)

Other: Quality of Life (QOL) questionnaires

Other: Healthcare Resource Utilization

Primary Outcome(s)

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]

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]

Secondary Outcome(s)

Percentage of time spent in sedentary, lightly active, fairly active, or very active lifestyles [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]

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]

Changes in the annual Healthcare Resource Care (HRC) involving the number of emergency room visits [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 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]

Change in the total scores over the study period using the Tran-QOL questionnaire [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]

Changes in the annual Healthcare Resource Care (HRC) involving the number of procedures undergone [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]

Secondary ID(s)

ACE-536-B-THAL-003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors