Resumen

Este estudio está abierto a adultos con determinadas enfermedades renales, incluidas glomeruloesclerosis segmentaria y focal secundaria (sFSGS), enfermedad por cambios mínimos primaria resistente al tratamiento (TR-pMCD), síndrome de Alport (AS) y nefropatía membranosa primaria resistente al tratamiento (TR-pMN). Los adolescentes con MCD primaria resistente al tratamiento también pueden participar. El propósito de este estudio es determinar si un medicamento llamado BI 764198 ayuda a personas con estas enfermedades renales. Los participantes son asignados aleatoriamente a 2 grupos, es decir, por azar. Un grupo toma comprimidos de BI 764198 y el otro grupo toma comprimidos de placebo. Los comprimidos de placebo tienen la misma apariencia que los de BI 764198, pero no contienen ningún medicamento. Los participantes toman un comprimido una vez al día durante 20 semanas. Todos los participantes continúan además con su medicación habitual para su enfermedad renal durante el estudio. Los participantes tienen el doble de probabilidades de ser asignados al grupo de BI 764198 que al grupo de placebo. Los participantes permanecen en el estudio durante aproximadamente 7 meses. Durante este tiempo, visitan el sitio del estudio 6 veces y realizan 3 llamadas telefónicas. Los médicos evalúan regularmente los niveles de proteína en la orina de los participantes mediante la recolección de muestras de orina. También evalúan la función renal mediante análisis de sangre. Los resultados se comparan entre los dos grupos para verificar si el tratamiento funciona. Los médicos también controlan regularmente la salud de los participantes y registran cualquier efecto no deseado.

Elegibilidad

Edad mínima: 12 YearsSexo: ALL

Criterios de inclusión

Male or female participants ≥18 years of age (≥12 years of age for Treatment resistant primary Minimal Change Disease (TR-pMCD)) on the day of signing informed consent/assent (Visit 1)
Body Mass Index (BMI) of ≤40 kg/m2 at screening visit (Visit 1)
Weight of ≥40 kg at screening
Estimated glomerular filtration rate (eGFR) ≥25 mL/min/1.73 m2 (chronic kidney disease (CKD) EPI formula based on serum cystatin C) at screening visit
For adult participants (≥18); ≥25 mL/min/1.73 m2 (CKD-EPI formula based on serum cystatin C) at the screening visit
For adolescent participants (\<18); ≥25 mL/min/1.73 m2 (chronic kidey disease under 25 years (CKiD U25) formula using height and serum cystatin C) at the screening visit
Seated blood pressure (mean of 3 values) systolic blood pressure (SBP) ≤160 mmHg (adult participants ≥18) or SBP ≤140 mmHg (participants \<18) at the screening visit (Visit 1). A participant with a documented history of white coat hypertension may be included as long as the participant is considered medically stable by the investigator and "true" blood pressure can be considered to be ≤160 mmHg (adult participants ≥18) or ≤140 mmHg (adolescent participants \<18)
Participants should be treated with angiotensin converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs), at a stable optimised dose for at least 8 weeks prior to the screening visit (Visit 1), with no plan to change the dose until the end of the randomised treatment period (i.e. end of trial (EoT), Week 20) unless not tolerated or indicated as per the discretion of the investigator
If treated with (non-steroidal) mineralocorticoid receptor antagonist (MRA), endothelin receptor antagonists (ERA), glucagon-like peptide-1 (GLP-1) or Sodium-glucose co-transporter-2 (SGLT2) inhibitors (SGLT2i), participants must be on a stable dose for at least 8 weeks prior to the screening visit (Visit 1), preferably with no plan to change the dose until the end of the randomised double-blind treatment period (i.e. EoT, Week 20)
Participants treated with oral immunosuppressive therapy except glucocorticoids (e.g. Calcineurin inhibitor(s) (CNI), mycophenolate mofetil/-sodium, cyclophosphamide) must be on a stable dose for at least 12 weeks prior to the screening visit (Visit 1) with no plans to change their dose during the trial treatment period
Patients treated/to be treated with oral glucocorticoids have to be at a dose ≤10 mg/d prednisolone or equivalent for ≥4 weeks prior to screening with no plan to increase the dose during the treatment period.

Criterios de exclusión

A history of organ transplantation or planned transplantation during the course of the study
Use of intravenous immunosuppressive agents (e.g. cyclophosphamide, rituximab, obinutuzumab) in the past 6 months prior to screening visit (Visit 1)
Participants in whom initiation of oral or IV immunosuppression is anticipated during the course of the trial
Treatment with metformin or dofetilide (multidrug and toxin extrusion protein 1 (MATE1) substrates) within one week prior to randomisation visit (Visit 2) through 5 days after the EoT visit
Treatment with strong inhibitors or strong inducers of cytochrome P450 3A4/5 (CYP3A4/5) within one week or 5 half-lives (whichever is longer) prior to randomisation visit (Visit 2)
Alanine aminotransferase (ALT)/aspartate aminotransferase (AST) \>3X the upper limit of normal (ULN) at screening visit (Visit 1)
Clinically significant laboratory abnormalities or medical conditions which pose a safety risk for the participant or may interfere with the trial objectives in the investigator's opinion (except for renal function tests or deviation of clinical laboratory values that are related to the podocytopathy in question) at screening visit

PODOMOUNT-Basket: ensayo canasta de fase II, multicéntrico, aleatorizado, de 2 grupos paralelos, doble ciego y controlado con placebo para evaluar la seguridad, tolerabilidad, farmacocinética y eficacia de BI 764198 en cuatro nefropatías proteinúricas

Resumen

Elegibilidad

Criterios de inclusión

Criterios de exclusión

Intervenciones

BI 764198

Placebo matching BI 764198

Ubicaciones

Clinica Privada Velez Sarfield

CardioAlem Investigaciones

Patrocinadores

Contactos

Boehringer Ingelheim