Cystinosis Clinical Trial
Official title:
Safety and Efficacy Trial of a Proposed NDA Formulation of Topical Cysteamine in the Treatment of Corneal Cystine Crystal Accumulation in Cystinosis
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.
Status | Completed |
Enrollment | 51 |
Est. completion date | March 2001 |
Est. primary completion date | |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A and older |
Eligibility |
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. |
Endpoint Classification: Safety Study, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | National Eye Institute (NEI) | Bethesda | Maryland |
Lead Sponsor | Collaborator |
---|---|
National Eye Institute (NEI) |
United States,
Markello TC, Bernardini IM, Gahl WA. Improved renal function in children with cystinosis treated with cysteamine. N Engl J Med. 1993 Apr 22;328(16):1157-62. — View Citation
Thoene JG, Oshima RG, Crawhall JC, Olson DL, Schneider JA. Cystinosis. Intracellular cystine depletion by aminothiols in vitro and in vivo. J Clin Invest. 1976 Jul;58(1):180-9. — View Citation
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