Resumen

Determinar el potencial nefroprotector a largo plazo del tratamiento con sparsentan en comparación con un bloqueador del receptor de angiotensina en pacientes con glomeruloesclerosis focal y segmentaria (GEFS) primaria y genética.

Descripción detallada

Se trata de un estudio aleatorizado, multicéntrico, doble ciego, paralelo, con control activo. Se espera enrolar aproximadamente 300 pacientes de entre 8 y 75 años (inclusive) en el estudio. El estudio se llevará a cabo en aproximadamente 300 centros a nivel global. El medicamento en investigación (sparsentan) es un bloqueador dual del receptor de angiotensina y antagonista del receptor de endotelina. El control activo es irbesartán. Los pacientes que cumplan los criterios de elegibilidad deberán realizar un período de lavado de bloqueadores del sistema renina-angiotensina-aldosterona (SRAA), si corresponde, antes de recibir la primera dosis del medicamento del estudio. Los pacientes serán asignados aleatoriamente en una proporción 1:1 para recibir sparsentan o control activo (irbesartán). Tras completar la fase doble ciego del estudio, los pacientes podrán participar en la extensión abierta para recibir tratamiento con sparsentan si cumplen los criterios de elegibilidad. La fecha de finalización primaria representa la fecha de finalización anticipada de la fase doble ciego del estudio. La fecha de finalización del estudio representa la fecha de finalización anticipada de la fase de extensión abierta del estudio.

Elegibilidad

Edad mínima: 8 YearsEdad máxima: 75 YearsSexo: ALL

Criterios de inclusión

Sites within the US and UK: The patient is male or female aged 8 to 75 years, inclusive, weighing ≥20 kg at screening
Sites outside the US and UK: The patient is male or female aged 18 to 75 years, inclusive, weighing ≥20 kg at screening
Biopsy-proven focal segmental glomerulosclerosis (FSGS) lesion(s) or documentation of a genetic mutation in a podocyte protein associated with FSGS.
Urine protein/creatinine (UP/C) ≥1.5 g/g (170 mg/mmol) at screening
eGFR ≥30 mL/min/1.73 m2 at screening.
Women of childbearing potential must agree to the use of one highly reliable method of contraception from 7 days prior to the first dose of study medication until 90 days after the last dose of study medication, plus one additional barrier method during sexual activity
Complete participation in the double-blind period, including the Week 112 visit.
Patient received blinded study medication through the duration of the double-blind period (ie, did not permanently discontinue study medication)

Criterios de exclusión

FSGS secondary to another condition
Positive serological tests of another primary or secondary glomerular disease not consistent with a diagnosis of primary or genetic FSGS
History of type 1 diabetes mellitus, uncontrolled type 2 diabetes mellitus, or nonfasting blood glucose \>180 mg/dL (10.0 mmol/L)
Treated with rituximab, cyclophosphamide, or abatacept within ≤3 months prior to screening; if taking other chronic immunosuppressive medications, the dosage must be stable prior to screening
Documented history of heart failure, coronary artery disease, or cerebrovascular disease
Significant liver disease
Positive at screening for the human immunodeficiency virus or markers indicating acute or chronic hepatitis B virus infection or hepatitis C infection
History of malignancy other than adequately treated basal cell or squamous cell skin cancer or cervical carcinoma within the past 2 years
Screening hematocrit value \<27% (0.27 L/L) or hemoglobin value \<9 g/dL (90 g/L)
Screening potassium value of \>5.5 mEq/L (5.5 mmol/L)
Extreme obesity (ie, ≥18 years of age with a body mass index (BMI) \>40, or is \<18 years of age with a BMI in the 99th percentile plus 5 units at screening, in whom there is a causal relationship between obesity and the development of FSGS
History of alcohol or illicit drug use disorder
History of serious side effect or allergic response to any angiotensin II antagonist or endothelin receptor antagonist
Female patient is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.
Progression to end-stage renal disease requiring replacement therapy
The patient developed criteria for discontinuation between Week 108 and Week 112
The patient was unable to initiate, or developed contraindications to, treatment with RAAS inhibitors between Week 108 and Week 112
eGFR ≤20 mL/min/1.73 m2 at Week 108

Estudio aleatorizado, multicéntrico, doble ciego, paralelo, con control activo, sobre los efectos de sparsentan, un bloqueador dual del receptor de endotelina y del receptor de angiotensina, en los resultados renales en pacientes con GEFS primaria

Resumen

Descripción detallada

Elegibilidad

Criterios de inclusión

Criterios de exclusión

Intervenciones

sparsentan

Irbesartan

Ubicaciones

Travere Investigational Site

Travere Investigational Site

Travere Investigational Site

Travere Investigational Site

Patrocinadores