Resumen del Estudio
Este estudio clínico de Fase 3 busca evaluar un nuevo tratamiento oral llamado Rilzabrutinib para pacientes adultos diagnosticados con Anemia Hemolítica Autoinmune por anticuerpos calientes (wAIHA) primaria. Esta es una condición donde el sistema inmunológico destruye equivocadamente los glóbulos rojos. El estudio está diseñado para personas que no han logrado una respuesta sostenida con el tratamiento estándar de corticosteroides o que no los toleran. Los participantes serán asignados al azar para recibir Rilzabrutinib o un placebo durante 24 semanas con el objetivo de medir si el fármaco logra aumentar y mantener los niveles de hemoglobina de forma duradera. Aquellos que completen esta fase inicial podrán continuar recibiendo el medicamento activo en un periodo abierto extendido, buscando ofrecer una alternativa terapéutica eficaz y segura para esta enfermedad hematológica compleja.
Criterios de Elegibilidad
Resumen de Elegibilidad:Pueden participar hombres y mujeres adultos con diagnóstico confirmado de wAIHA primaria (mínimo 3 meses de evolución) que hayan fallado, sean dependientes o intolerantes a los corticosteroides. No pueden participar pacientes con wAIHA secundaria (por lupus, fármacos, etc.), infecciones activas por Hepatitis B, C o VIH, ni con historia reciente de neoplasias malignas.
- Male and female participants with a documented (confirmed) diagnosis of primary wAIHA for at least 3 months.
- Participants who have previously failed to maintain a sustained response after treatment with CS (CS-resistance \[defined as failure to obtain hemoglobin response within 3 weeks on at least 1 mg/kg or 60 mg prednisone or equivalent per day\], CS-dependent wAIHA \[defined as need to continue on prednisone or equivalent at a dose of \>10 mg/day to maintain a response\]), or are intolerant or ineligible to CS (defined as with contraindications, pre-existing medical conditions or CS-related complications that may render CS intolerant or ineligible per the best clinical judgement of the investigators).
- Participants with Eastern Cooperative Oncology Group (ECOG) performance status Grade 2 or lower.
- Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- Participants with clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator.
- Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years.
- Participants with symptomatic herpes zoster within 3 months prior to screening.
- Participants with secondary wAIHA from any cause including drugs, Evans Syndrome, lymphoproliferative disorders (low count monoclonal B-cell lymphocytosis is allowed), infectious or autoimmune disease, or active hematologic malignancies. Participants with positive antinuclear antibodies but without a definitive diagnosis of an autoimmune disease are allowed.
- Participants with history of myelodysplastic syndrome.
- Participants with uncontrolled or active HBV infection or Active HCV infection.
- HIV infection.
- Participants with history of solid organ transplant.
- Participants with a history of active or latent tuberculosis (TB).
- Concurrent treatment with other experimental/investigational drugs within 30 days or 5 half-lives, whichever is longer, prior to treatment start. Participants who previously received treatment with BTK inhibitors for wAIHA before Day 1 (randomization) are not eligible.
- Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study.