Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dichlorphenamide for Periodic Paralyses and Associated Sodium Channel Disorders
dichlorphenamide
Enfermedades metabólicas+12
+ Errores innatos del metabolismo
+ Errores innatos del metabolismo de metales
Estudio de Tratamiento
Resumen
Fecha de inicio: 1 de junio de 1992
Fecha en la que se inscribió al primer participante.PROTOCOL OUTLINE: This is a randomized, double-blind study. Patients are stratified by participating institution and diagnosis. The weekly attack rate is determined during an 8-week assessment prior to therapy initiation and at crossover. Patients are randomly assigned to oral dichlorphenamide (DCP) or placebo for 9 weeks and then cross to the alternate treatment. Patients on DCP at baseline continue on the same dose; those on acetazolamide (ACZ) at baseline receive a DCP dose equivalent to one fifth of the ACZ dose.
Protocolo
Esta sección proporciona detalles del plan del estudio, incluyendo cómo está diseñado y qué se está evaluando.Se reclutarán 64 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.De 10 a 75 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-- Hypokalemic periodic paralysis Typical clinical profile Normal serum thyroxine Hypokalemia during spontaneous or glucose-induced paralytic attack in subject or affected family member Periodic paralysis associated with sodium channel 17q alpha-subunit, e.g.: Hyperkalemic periodic paralysis with or without myotonia Paramyotonia congenita with periodic paralysis Distinct, regular episodes of weakness at least once a week and no more than 3 times a day No history of worsening symptoms with carbonic anhydrase inhibitor No history of life-threatening weakness episodes prior to treatment No atypical periodic paralysis without demonstrable 17q alpha-subunit defect --Prior/Concurrent Therapy-- No requirement for the following agents, unless for periodic paralysis: Diuretics Antiepileptics Antiarrhythmics Magnesium supplements Steroids Calcium supplements Beta-blockers Potassium supplements Calcium channel blockers --Patient Characteristics-- Hepatic: No hepatic disease Renal: No renal failure No nephrolithiasis Cardiovascular: No heart disease No cardiac arrhythmia Pulmonary: No restrictive or obstructive lung disease Other: No active thyroid disease No pregnant women