A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorBristol-Myers Squibb

About this trial

The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Male or female participants of any race aged at least 18 years at time of initiation of luspatercept treatment

Participants with documented diagnosis of transfusion-dependent β-thalassemia (TDT).

Participants who have been initiated on treatment with luspatercept as per the product's Summary of Product Characteristics (SmPC) no longer than 12 months prior to informed consent signature, and for whom therapy is ongoing.

Participants for whom the decision to prescribe luspatercept treatment is clearly separated from the physician's decision to include the participant in the current study.

Disqualifiers

Participants that meet any of the contraindications to the administration of luspatercept as outlined in the latest version of the locally approved SmPC.

Participants who are currently receiving or are planned to receive treatment with any investigational drug/device/intervention or who have received any investigational product within 1 month or 5 half-lives of the investigational agent (whichever is longer) prior to luspatercept therapy initiation.

Participants who are currently pregnant, breastfeeding, or planning a pregnancy during the study observation period.

Participants who have not provided signed informed consent for participating in the study and for collecting and analysing medical data pertinent to the objectives of this study.

Trial population

The study population will include adult patients with transfusion-dependent β-thalassemia (TDT) in the Middle East region who have initiated luspatercept treatment

Trial design

Design model

Cohort

Time perspective

Other

Treatments tested in this trial

  • Luspatercept

    Drug

    According to the product label

Treatment groups

200 Participants
are divided into 1 treatment group
Group A: Participants receiving luspatercept treatment1 intervention

Trial outcomes

Primary outcomes

1

Change in transfusion burden

Time frame
Baseline and up to week 144
2

Change in mean pre-transfusion hemoglobin level

Time frame
Baseline and up to week 144

Secondary outcomes

1

Change in transfusion-related visits

Time frame
Baseline and up to week 144
2

Proportion of participants achieving ≥33% reduction in red blood cell (RBC) transfusion burden (number of RBC units transfused) plus a reduction of ≥2 units

Time frame
Baseline and up to week 144
3

Proportion of participants achieving ≥50% reduction in red blood cell transfusion burden plus a reduction of ≥2 units

Time frame
Baseline and up to week 144
4

Time from first luspatercept dosing date to the first erythroid response

Time frame
Baseline and up to week 144

Other outcomes

Sponsors and contacts

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