Clinical trial 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
Official title: Burden of Beta-Thalassemia - Quality of Life and Health Care Resource Utilization- A Prospective Observational Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
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
Fact-An
NTD beta-thalassemia PRO
Lead sponsor: Celgene
Sponsor class: INDUSTRY
First posted: 2015-12-10
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: β-thalassemia subjects who are transfusion dependent (TD) and non-transfusion dependent (NTD)

Brief 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 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.


Eligibility 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 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.

Primary outcomes:
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
timeFrame: Up to 6 months
measure: 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
timeFrame: Up to 6 months
Secondary outcomes:
measure: Change in the total scores over the study period using the The Functional Assessment of Cancer Therapy-Anemia (FACT-An) questionnaire
timeFrame: Up to 6 months
measure: Change in the total scores over the study period using the Tran-QOL questionnaire
timeFrame: Up to 6 months
measure: Change in the total scores over the study period using the Patient Reported Outcome (PRO) questionnaire in non-transfusion dependent (NTD) subjects
timeFrame: Up to 6 months
measure: Changes in the annual Healthcare Resource Care (HRC) involving the number of office visits
timeFrame: Up to 6 months
measure: Number of minutes spent in sedentary, lightly active, fairly active, or very active lifestyles
timeFrame: Up to 6 months
measure: Changes in the annual Healthcare Resource Care (HRC) involving the number of prescriptions dispensed
timeFrame: Up to 6 months
measure: Changes in the annual Healthcare Resource Care (HRC) involving the number of procedures undergone
timeFrame: Up to 6 months
measure: Changes in the annual Healthcare Resource Care (HRC) involving the number of lab assessments completed
timeFrame: Up to 6 months
measure: Changes in the annual Healthcare Resource Care (HRC) involving the number of days that a patient is hospitalized
timeFrame: Up to 6 months
measure: Changes in the annual Healthcare Resource Care (HRC) involving the number of emergency room visits
timeFrame: Up to 6 months
measure: Percentage of time spent in sedentary, lightly active, fairly active, or very active lifestyles
timeFrame: Up to 6 months
Other outcomes:
Locations:
city: Athens
country: Greece
facility: Aghia Sofia Children's Hosptial
state: Goudi
city: Milano
country: Italy
facility: Fondazione IRCCS Ca Granda Ospedale Maggiore
city: Beirut
country: Lebanon
facility: American University of Beirut Medical Center
city: Bangkok
country: Thailand
facility: Faculty of Medicine Siriraj Hospital, Mahidol University
state: Bangkoknoi
city: Cukurova
country: Turkey
facility: Cukurova University Medical Faculty Balcali Hospital
Study officials:
affiliation: Celgene
name: Xiaohan (Henry) HU, MD, MPH, PhD
role: STUDY_DIRECTOR
References: