New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis

Inclusion 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.