Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis
Adeno-associated virus-CFTR vector
Enfermedades del Sistema Digestivo+6
+ Enfermedades del recién nacido
+ Enfermedades del pulmón
Estudio de Tratamiento
Resumen
Fecha de inicio: 1 de agosto de 1999
Fecha en la que se inscribió al primer participante.PROTOCOL OUTLINE: This is a randomized, dose escalation, double blind, placebo controlled, multicenter study. Patients are randomized to receive either adeno-associated virus-CFTR (AAV-CFTR) vector or placebo. Patients undergo tests on days -10 to -4 to rule out adenovirus and adeno-associated virus infections. Patients then receive AAV-CFTR vector intranasally to the right or left inferior turbinates and placebo to the other side of the nose. The next day, patients receive an endobronchial dose of AAV-CFTR vector to the superior segment of the right lower lobe. Cohorts of 2-4 patients each receive escalating doses of AAV-CFTR vector until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose level immediately preceding the dose at which at least 2 subjects develop dose limiting toxicity. Patients are followed at day 10, then at 1, 2, 3, 6, 9, and 12 months.
Protocolo
Esta sección proporciona detalles del plan del estudio, incluyendo cómo está diseñado y qué se está evaluando.Se reclutarán 19 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.A partir de 15 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-- Diagnosis of cystic fibrosis (CF) with mild to moderate lung disease; FVC at least 60% predicted; Schwachman-Kulczycki score at least 65; Sweat chloride greater than 60 mEq/L All CF genotypes allowed No sputum colonization with Burkholderia cepacia or another multiply resistant organism No recurrent or intermittent hemoptysis; At least 1 year since significant hemoptysis requiring transfusion or embolization OR Coughing of greater than 30 mL of blood more often than once a week --Prior/Concurrent Therapy-- At least 30 days since hospitalization or home intravenous antibiotic therapy for pulmonary exacerbation No concurrent investigational drugs or therapy --Patient Characteristics-- Other: No concurrent cigarette smoking; Not pregnant or nursing; Negative pregnancy test; Fertile patients must use effective contraception