Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of Porphyria
heme arginate
+ tin mesoporphyrin
Maladies métaboliques+7
+ Maladies et anomalies congénitales, héréditaires et néonatales
+ Maladies nutritionnelles et métaboliques
Étude thérapeutique
Résumé
Date de début de l'étude : 1 janvier 1998
Date à laquelle le premier participant a commencé l'étude.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.
Protocole
Cette section fournit des détails sur le plan de l'étude, y compris la manière dont l'étude est conçue et ce qu'elle évalue.20 participants à inclure
Nombre total de participants que l'essai clinique vise à recruter.Traitement
Éligibilité
Les chercheurs recherchent des patients correspondant à une certaine description appelée critères d'éligibilité : état de santé général ou traitements antérieurs du patient.Tout sexe
Le sexe biologique des participants éligibles à s'inscrire.À partir de 18 ans
Tranche d'âge des participants éligibles à participer.Volontaires sains non autorisés
Indique si les individus en bonne santé et ne présentant pas la condition étudiée peuvent participer.Conditions
Pathologie
Critères
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