Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
calcitriol
+ interferon gamma
Enfermedades de los Huesos+6
+ Enfermedades Óseas del Desarrollo
+ Enfermedades del sistema musculoesquelético
Estudio de Tratamiento
Resumen
Fecha de inicio: 1 de noviembre de 1999
Fecha en la que se inscribió al primer participante.PROTOCOL OUTLINE: This is a randomized, placebo controlled, open label study. Patients are randomized to one of two arms (interferon gamma in combination with calcitriol or calcitriol alone). Arm I: Patients receive calcitriol once daily. Interferon gamma is administered by subcutaneous injection three times a week. Arm II: Patients receive calcitriol once daily. Patients may continue treatment in the absence of toxicity and disease progression. If disease progression is diagnosed in the control group, patients will then receive interferon gamma in combination with calcitriol. Patients are followed every 4 weeks.
Protocolo
Esta sección proporciona detalles del plan del estudio, incluyendo cómo está diseñado y qué se está evaluando.Se reclutarán 30 pacientes
Número total de participantes que el ensayo clínico espera reclutar.Estudio de Tratamiento
Elegibilidad
Los investigadores buscan pacientes que cumplan ciertos criterios, conocidos como criterios de elegibilidad: estado general de salud o tratamientos previos.Cualquier sexo
Sexo biológico de los participantes elegibles para inscribirse.Hasta 10 Años
Rango de edades de los participantes que pueden unirse al estudio.Voluntarios sanos no permitidos
Indica si personas sanas, sin la condición que se estudia, pueden participar.Condiciones
Patología
Criterios
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Biopsy and x-ray confirmed primary osteopetrosis Presence of anemia and/or cranial nerve compression --Prior/Concurrent Therapy-- Biologic therapy: No prior/concurrent bone marrow transplantation No prior interferon gamma No other investigational biologic agents Chemotherapy: No prior/concurrent chemotherapeutic agents for bone marrow transplantation Endocrine therapy: Prior/concurrent corticosteroid as supportive therapy allowed Radiotherapy: Not specified Surgery: At least 5 days since major surgery Other: Prior/concurrent calcitriol as supportive therapy allowed Prior/concurrent transfusion as supportive therapy allowed Prior/concurrent dietary therapy allowed --Patient Characteristics-- Age: 2 months to 10 years Performance status: Not specified Life expectancy: At least 6 months Hematopoietic: Not specified Hepatic: Bilirubin less than 2 mg/dL Renal: Creatinine less than 1.5 mg/dL OR Creatinine clearance greater than 50 mL/min Pulmonary: No uncorrected airway obstruction Other: No active infection requiring intravenous antibiotics No known seizure disorder not related to hypocalcemia No uncorrected hydrocephalus No MRI evidence of cerebral atrophy Must maintain or gain body weight No sleep apnea No thrombocytopenia No massive splenomegaly