Sickle cell anemia without crisis Sickle cell disorders
Conditions
Interventions
This is a randomized, controlled, open-label clinical trial with two arms, involving a total of 80 participants, with 40 participants in the dapagliflozin-treated group and 40 participants in the untr
s resources. Recognizing that the absence of masking may introduce performance and measurement biases, strict measures will be adopted to minimize this risk. Primary outcomes will be objective and mea
Sponsors
Centro de Hematologia e Hemoterapia da Unicamp – Hemocentro da UNICAMP
Centro de Hematologia e Hemoterapia da Unicamp – Hemocentro da UNICAMP
Eligibility
Age
18 Years to 70 Years
Inclusion criteria
Inclusion criteria: Adults aged 18 to 70 years; confirmed diagnosis of sickle cell disease of the HbSS or HbSB0 genotypes; estimated glomerular filtration rate (eGFR) of 15 mL/min/1.73 m² or higher at the time of screening; hemoglobin of 5 g/dL or higher; History of at least 2 vaso-occlusive crises in the past 12 months or at least 1 severe episode (acute chest syndrome, priapism, or need for transfusion) in the past year; Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2; Stable use of hydroxyurea for at least 90 days prior to enrollment, or documentation of the reason why the patient is not taking the medication; ability to provide written informed consent
Exclusion criteria
Exclusion criteria: Pregnancy or lactation; severe hepatic impairment (Child-Pugh C); current use or use within the past 3 months of any SGLT2 inhibitor; history of diabetic ketoacidosis or diagnosis of type 1 diabetes mellitus; systolic blood pressure below 95 mmHg at screening or symptomatic hypotension; history of hematopoietic stem cell transplantation; participation in another interventional study with an investigational agent or use of any experimental therapy; recurrent urinary tract infections (3 or more episodes in the past year) or active genital infection at the time of screening; inability to adhere to the study protocol or to attend scheduled visits
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| A reduction in plasma levels of NT-proBNP (N-terminal pro-B-type natriuretic peptide) is expected after treatment with dapagliflozin, verified by standardized laboratory testing, based on a statistically significant difference between the groups (treated and untreated) in the pre- and post-intervention variation. | — |
Secondary
| Measure | Time frame |
|---|---|
| A reduction in left ventricular mass is expected to be found, verified by transthoracic echocardiography, based on the differences between the groups in the variation of the values obtained before and after the intervention ;An improvement in left ventricular ejection fraction is expected, verified by transthoracic echocardiography, based on differences between the groups in the variation of values obtained before and after the intervention ;An improvement in the E/e ratio is expected, verified by transthoracic echocardiography, based on differences between the groups in the variation of values obtained before and after the intervention ;An increase in the distance covered in the six-minute walk test is expected, verified by a standardized test, based on differences between the groups in the variation of distances measured before and after the intervention ;An improvement in the lean mass/fat mass ratio is expected, verified by electrical bioimpedance, based on differences between the groups in the variation of values measured before and after the intervention ;A reduction in microalbuminuria values is expected, verified by quantitative laboratory testing, based on differences between the groups in the variation in levels measured before and after the intervention | — |
Countries
BR
Contacts
Public ContactMurilo Lutfi
Centro de Hematologia e Hemoterapia da Unicamp – Hemocentro da UNICAMP
Outcome results
None listed