Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of Porphyria
heme arginate
+ tin mesoporphyrin
Enfermedades metabólicas+4
+ Enfermedades y Anomalías Congénitas, Hereditarias y Neonatales
+ Enfermedades Nutricionales y Metabólicas
Estudio de Tratamiento
Resumen
Fecha de inicio: 1 de enero de 1998
Fecha en la que se inscribió al primer participante.PROTOCOL OUTLINE: This is an unblinded study. Patients start a preventive heme regimen and are followed for 6 months prior to tin mesoporphyrin. The frequency of attacks is recorded. Patients receive tin mesoporphyrin once a week for 6 months. Patients must avoid sunlight during treatment and for 2 months thereafter. The frequency of heme infusions is reduced by half and the frequency of attacks of porphyria is monitored. Patients are followed for 6 months after last treatment.
Protocolo
Esta sección proporciona detalles del plan del estudio, incluyendo cómo está diseñado y qué se está evaluando.Se reclutarán 20 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 18 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-- Documented acute intermittent porphyria, variegate porphyria, or hereditary coproporphyria causing 6 or more attacks per year Fewer than 2 attacks in the preceding 6 months if already on preventive heme therapy --Prior/Concurrent Therapy-- See Disease Characteristics --Patient Characteristics-- Performance status: Not specified Life expectancy: Not specified Hematopoietic: Not specified Hepatic: Not specified Renal: Urinary porphobilinogen at least 20 mg/24 hours during acute symptoms Other: Not pregnant Fertile female patients must use effective contraception during and for 6 months prior to study No recurrent symptoms due to another illness No continuous (for longer than 1 month) symptoms of porphyria unless it is in addition to acute exacerbations No acute hemorrhagic disorder such as: Gastrointestinal bleeding Intracerebral hemorrhage No known hypersensitivity to tin mesoporphyrin No known hypersensitivity to any intravenous heme preparation No other condition which may increase risk to patient