New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis
Status:
Completed
Trial end date:
2001-03-01
Target enrollment:
Participant gender:
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.