New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis

• Status: Completed
• Trial end date: 2001-03-01
• Target enrollment: 0
• Participant gender: All

Summary

This study will evaluate the safety and effectiveness of a new formulation of eye drops used to treat cystine crystals that form in the corneas of patients with cystinosis. Cystinosis is an inherited disease caused by a defective enzyme, in which excessive amounts of the amino acid cystine accumulate in the body. Among others, symptoms include poor growth and development of kidney failure. In addition, after 10 to 20 years, the cornea-the outside covering of the eye over the iris and pupils-becomes so packed with cystine crystals that small, painful breaks may develop.

This corneal condition is treated with cysteamine eye drops. This study is designed to provide additional information about this medication that the Food and Drug Administration requires before approving it for marketing. The study will examine, in two separate but simultaneous investigations, the safety and effectiveness of a new cysteamine formulation. In both studies, before treatment begins, patients will have a complete eye examination, and photographs of the eye will be taken using a bright flash.

Safety Study

Children and adults currently enrolled in a cystinosis study at the National Institutes of Health may participate in this trial. They will receive the current cysteamine formulation in one eye and the new preparation in the other eye. The drops will be given every hour during waking hours. Patients will be observed daily for the first week of treatment and will be called at 2 weeks and 4 weeks to check on side effects, if any. At 6 months, they will undergo a repeat eye examination. Patients (or their parents) will keep a daily diary recording the condition of each eye.

Effectiveness Study

Children and adults from Ann Arbor, Michigan, LaJolla, California, and the NEI clinic may be enrolled in this study. Participants will receive medication as described above for the safety trial. They will be observed daily for the first week and will have repeat eye examinations, including photographs, at months 3, 6, 9 and 12 to see if the crystals have decreased. Patients will keep a daily diary of the condition of both eyes.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Eye Institute (NEI)
• Treatments: Cysteamine; Methamphetamine

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.