Safety and Efficacy Trial of a Proposed NDA Formulation of Topical Cysteamine in the Treatment of Corneal Cystine Crystal Accumulation in Cystinosis
Cysteamine
Maladies métaboliques+4
+ Erreurs innées du métabolisme
+ Maladies et anomalies congénitales, héréditaires et néonatales
Étude thérapeutique
Résumé
Date de début de l'étude : 1 mai 1998
Date à laquelle le premier participant a commencé l'étude.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.
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.51 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.Volontaires sains non autorisés
Indique si les individus en bonne santé et ne présentant pas la condition étudiée peuvent participer.Conditions
Pathologie
Critères
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.
Centres d'étude
Ce sont les hôpitaux, cliniques ou centres de recherche où l'essai est conduit. Vous pouvez trouver le site le plus proche de vous ainsi que son statut.Cette étude comporte 1 site
National Eye Institute (NEI)
Bethesda, United StatesOuvrir National Eye Institute (NEI) dans Google Maps