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

Celgene

Status: Completed

Conditions: Beta-Thalassemia

Treatments: Other: Quality of Life (QOL) questionnaires; Other: Healthcare Resource Utilization

Study type: Observational

Funder types: Industry

Identifiers

NCT02626689

ACE-536-B-THAL-003

Details and patient eligibility

About

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.

Enrollment: 100 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

100 participants in 2 patient groups

β-thalassemia transfusion dependent subjects

Description:

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.

Treatment:

Other: Quality of Life (QOL) questionnaires

Other: Healthcare Resource Utilization

β-thalassemia Non Transfusion Dependent (NTD) subjects

Description:

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.

Treatment:

Other: Quality of Life (QOL) questionnaires

Other: Healthcare Resource Utilization