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New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis

Primary Purpose

Cystinosis

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Cysteamine
Sponsored by
National Eye Institute (NEI)
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystinosis focused on measuring Corneal Crystals, Cysteamine, Cystinosis, Eye

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • National Eye Institute (NEI)

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
November 3, 1999
Last Updated
March 3, 2008
Sponsor
National Eye Institute (NEI)
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1. Study Identification

Unique Protocol Identification Number
NCT00001736
Brief Title
New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis
Official Title
Safety and Efficacy Trial of a Proposed NDA Formulation of Topical Cysteamine in the Treatment of Corneal Cystine Crystal Accumulation in Cystinosis
Study Type
Interventional

2. Study Status

Record Verification Date
April 2000
Overall Recruitment Status
Completed
Study Start Date
May 1998 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
March 2001 (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
National Eye Institute (NEI)

4. Oversight

5. Study Description

Brief 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.
Detailed Description
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 at Week 1, Week 2, Week 4, and Month 6. Complete ophthalmic evaluation including corneal slit lamp examination with photographs will be performed at baseline and 6 months at the NEI Clinical Center. Efficacy will be evaluated among 15 patients (5 each from the NEI Clinical Center, Ann Arbor Michigan, and La Jolla, California) who have not received cysteamine eye drops prior to enrollment. 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 and a complete ophthalmic assessment including a corneal slit lamp examination with photographs will be performed at baseline, Month 3, Month 6, Month 9, and Month 12. The primary efficacy outcome measure will be the improvement in the new formulation treated eye by 1.00 unit on a corneal photograph scale from 0.00 to 3.00 in 0.25 increments compared to the baseline grade. This scale grades the density and number of crystals in the cornea. The safety and efficacy studies will be conducted concurrently.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystinosis
Keywords
Corneal Crystals, Cysteamine, Cystinosis, Eye

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Enrollment
51 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Cysteamine

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
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.
Facility Information:
Facility Name
National Eye Institute (NEI)
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
8455682
Citation
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. doi: 10.1056/NEJM199304223281604.
Results Reference
background
PubMed Identifier
932205
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. doi: 10.1172/JCI108448.
Results Reference
background

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New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis

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