Safety and Efficacy Trial of a Proposed NDA Formulation of Topical Cysteamine in the Treatment of Corneal Cystine Crystal Accumulation in Cystinosis
Cysteamine
Enfermedades metabólicas+2
+ Errores innatos del metabolismo
+ Enfermedades y Anomalías Congénitas, Hereditarias y Neonatales
Estudio de Tratamiento
Resumen
Fecha de inicio: 1 de mayo de 1998
Fecha en la que se inscribió al primer participante.The primary purpose of this study is to evaluate the safety and efficacy of a new formulation of cysteamine eye drops to treat children and adults with cystinosis and cystine crystal accumulation in the cornea. The safety of this new formulation will be evaluated among 30 children and adults with a history of cystinosis under treatment at the NIH with the current preparation of cysteamine eye drops. Patients will be randomized to the current preparation in one eye and the new formulation in the other eye. Vision, blurring, redness, pain, irritation, and itching will be evaluated in each eye at Week 1, Week 2, Week 4, and Month 6. Complete ophthalmic evaluation including corneal slit lamp examination with photographs will be performed at baseline and 6 months at the NEI Clinical Center. Efficacy will be evaluated among 15 patients (5 each from the NEI Clinical Center, Ann Arbor Michigan, and La Jolla, California) who have not received cysteamine eye drops prior to enrollment. Patients will be randomized to the current preparation in one eye and the new formulation in the other eye. Vision, blurring, redness, pain, irritation, and itching will be evaluated in each eye and a complete ophthalmic assessment including a corneal slit lamp examination with photographs will be performed at baseline, Month 3, Month 6, Month 9, and Month 12. The primary efficacy outcome measure will be the improvement in the new formulation treated eye by 1.00 unit on a corneal photograph scale from 0.00 to 3.00 in 0.25 increments compared to the baseline grade. This scale grades the density and number of crystals in the cornea. The safety and efficacy studies will be conducted concurrently.
Protocolo
Esta sección proporciona detalles del plan del estudio, incluyendo cómo está diseñado y qué se está evaluando.Se reclutarán 51 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.Voluntarios sanos no permitidos
Indica si personas sanas, sin la condición que se estudia, pueden participar.Condiciones
Patología
Criterios
Patients appearing for their scheduled visits under Protocol 86-EI-0062 will be offered enrollment into this study provided they fulfill all the following criteria: SAFETY STUDY: Patient diagnosed with cystinosis (greater than 2 nmole half-cystine/milligram protein). Patient has a clinical history consistent with cystinosis. Willing and able to tolerate photographs. Patient age greater than or equal to 1 year old. Willingness and ability to comply with treatment and follow-up procedures as demonstrated by a history of adherence with their current eye-drop and patient follow up schedule under protocol 86-EI-0062. Ability of the patient or the patient's parent or legal guardian to understand and sign an informed consent. Any crystal density score, including zero, on photographs, which has been stable or improved over the past year. EFFICACY STUDY: Patients must fulfill all of the following criteria to be eligible for the efficacy study. Patient diagnosed with cystinosis (greater than 2 nmole half-cystine/milligram protein). Patient has a clinical history consistent with cystinosis. Willing and able to tolerate photographs. Age greater than or equal to 2 years and less than or equal to 10 years. Willingness and the ability to comply with treatment and follow-up procedures. Ability of the patient or the patient's parent or legal guardian to understand and sign an informed consent form. Crystal density score greater than or equal to 1.00 on photographs. Photographs must be submitted to the NEI Clinical Center for quality and eligibility verification prior to enrollment. No prior use of cysteamine drops. Prior use of Cystagon for 6 months prior to enrollment.
Centros del Estudio
Estos son los hospitales, clínicas o centros de investigación donde se lleva a cabo el estudio. Puedes encontrar la ubicación más cercana a ti y su estado de reclutamiento.Este estudio tiene una ubicación
National Eye Institute (NEI)
Bethesda, United StatesAbrir National Eye Institute (NEI) en Google Maps