New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis

Launched by NATIONAL EYE INSTITUTE (NEI) · Dec 9, 2002

Keywords

ClinConnect Summary

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...

Gender

ALL

Eligibility 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.