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

NCT ID: NCT02626689

Last Updated: 2017-04-07

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 100 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2016-03-01
• Study Completion Date: 2017-01-03

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.

Conditions

Beta-Thalassemia

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

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.

Quality of Life (QOL) questionnaires

Intervention Type: OTHER

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

Healthcare Resource Utilization

Intervention Type: OTHER

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.

Quality of Life (QOL) questionnaires

Intervention Type: OTHER

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

Healthcare Resource Utilization

Intervention Type: OTHER

Monitoring and reviewing HealthCare Resource Utilization information.

Interventions

Quality of Life (QOL) questionnaires

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

Intervention Type: OTHER

Healthcare Resource Utilization

Monitoring and reviewing HealthCare Resource Utilization information.

Intervention Type: OTHER

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Celgene

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Xiaohan (Henry) HU, MD, MPH, PhD

Role: STUDY_DIRECTOR

Celgene

Locations

Aghia Sofia Children's Hosptial

Athens, Goudi, Greece

Fondazione IRCCS Ca Granda Ospedale Maggiore

Milan, , Italy

American University of Beirut Medical Center

Beirut, , Lebanon

Faculty of Medicine Siriraj Hospital, Mahidol University

Bangkok, Bangkoknoi, Thailand

Cukurova University Medical Faculty Balcali Hospital

Çukurova, , Turkey (Türkiye)

Countries

Greece; Italy; Lebanon; Thailand; Turkey (Türkiye)

Other Identifiers

ACE-536-B-THAL-003

Identifier Type: -

Identifier Source: org_study_id