Safety and Efficacy Trial of a Proposed NDA Formulation of Topical Cysteamine in the Treatment of Corneal Cystine Crystal Accumulation in Cystinosis
Cysteamine
Metabolic Diseases+4
+ Metabolism, Inborn Errors
+ Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Treatment Study
Summary
Study start date: May 1, 1998
Actual date on which the first participant was enrolled.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.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.51 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
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.
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 1 location