β-thalassemia
Conditions
Keywords
Transfusion-dependent β-thalassemia
Brief summary
The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East
Interventions
DRUGLuspatercept
According to the product label
Sponsors
Bristol-Myers Squibb
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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. * Participants who have provided signed informed consent for participating in the study and for collecting and analyzing medical data pertinent to the objectives of this study
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in transfusion burden | Baseline and up to week 144 |
| Change in mean pre-transfusion hemoglobin level | Baseline and up to week 144 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in transfusion-related visits | Baseline and up to week 144 | — |
| Proportion of participants achieving ≥33% reduction in red blood cell (RBC) transfusion burden (number of RBC units transfused) plus a reduction of ≥2 units | Baseline and up to week 144 | — |
| Proportion of participants achieving ≥50% reduction in red blood cell transfusion burden plus a reduction of ≥2 units | Baseline and up to week 144 | — |
| Time from first luspatercept dosing date to the first erythroid response | Baseline and up to week 144 | — |
| Time from the date the erythroid response is first observed until the last day of response | Baseline and up to week 144 | — |
| Participant pretransfusion hemoglobin level | Baseline and up to week 144 | — |
| Participant baseline transfusion burden | Baseline | — |
| Proportion of participants without red blood cell transfusion during any consecutive 12-week or 24-week treatment period | Baseline and up to week 144 | — |
| Time from first luspatercept dosing date to first onset of red blood cell-transfusion independence ≥12 weeks and ≥24 weeks | Baseline and up to week 144 | — |
| Time from the date a 12-week and 24-week red blood cell-transfusion independence is first observed until the date the participant has a subsequently documented red blood cell transfusion | Baseline and up to week 144 | — |
| Change in mean serum ferritin (SF) level | Baseline and up to week 144 | — |
| Change in proportion of participants with mean serum ferritin <1,000, 1000-2500, and >2,500 μg/L | Baseline and up to week 144 | — |
| Types (drug formulation - mono and combination therapy) of iron chelation therapy received | Baseline and up to week 144 | — |
| Change in mean daily dose of iron chelation therapy from baseline | Baseline and up to week 144 | — |
| Number of medical encounters (inpatient hospitalizations, emergency department attendances, hospital outpatient visits, visits at office-based physicians) | Baseline and up to week 144 | — |
| Inpatient length of stay | Baseline and up to week 144 | — |
| Proportion of participants remaining on luspatercept treatment | Up to week 144 | — |
| Length of time from initiation to discontinuation of luspatercept treatment | Up to week 144 | — |
| Frequencies of reasons for discontinuation of luspatercept treatment | Up to week 144 | — |
| Participant sociodemographics | Baseline | Sociodemographics of participants such as age, sex, ethnicity, country of treatment, height, weight, and body mass index |
| Participant disease characteristics | Baseline | Disease characteristics of participants, describing age of diagnosis of β-thalassemia, genotype (β0/β0, non-β0/β0), splenectomy status (yes/no), pretransfusion hemoglobin level (mean of all documented), transfusion burden (total number of red blood cell units transfused). |
| Participant comorbidities | Baseline | Disease- and non-disease-related comorbid conditions |
| Concomitant treatment(s) received | Baseline | Disease-related best supportive care (BSC) and treatments for other comorbidities. |
| Number of luspatercept doses administered | Up to week 144 | — |
| Number of luspatercept dose modifications | Up to week 144 | — |
| Frequencies of reasons for luspatercept dose modifications | Up to week 144 | — |
Countries
Kuwait, Oman, Saudi Arabia, United Arab Emirates
Contacts
CONTACTBMS Clinical Trials Contact Center www.BMSClinicalTrials.com
CONTACTFirst line of the email MUST contain NCT # and Site #.
STUDY_DIRECTORBristol Myers Squibb
Bristol-Myers Squibb
Outcome results
None listed