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CompletedNCT00010426Updated Mar 25, 2015

Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis

An interventional study of cysteamine hydrochloride in Cystinosis, sponsored by FDA Office of Orphan Products Development. Completed. Open to participants aged 1 Year to 50 Years. Per ClinicalTrials.gov, last updated 2015-03-25.

Sponsored by FDA Office of Orphan Products Development · Not applicable, Interventional, and Treatment

Phase
Not applicable
Study type
Interventional
Enrollment
30
Ages
1 Year to 50 Years
Sex
All
01

Study summary

OBJECTIVES: I. Determine the proportion of patients with cystinosis who experience a serious adverse effect when treated with a new formulation of cysteamine hydrochloride for corneal cystine accumulation.

II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen.

Read the detailed description

PROTOCOL OUTLINE: This is a randomized, multicenter study Patients are randomized to receive the current formulation of cysteamine hydrochloride as drops in one eye and the new formulation of cysteamine hydrochloride as drops in the other eye.

Patients receive the two formulations of cysteamine hydrochloride in their assigned eyes every hour during waking hours daily for 6 months (safety study) or for 1 year (efficacy study).

02

Conditions studied

  • Cystinosis

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Keywords

  • cystinosis
  • rare disease
  • renal and genitourinary disorders
03

In context

Cystinosis

44 studies on the registry are indexed under Cystinosis; 14 are open to participants now.

This study's enrollment of 30 is close to the median of 30 across 24 interventional studies indexed under Cystinosis.

Browse Cystinosis studies →

Lead sponsor

FDA Office of Orphan Products Development is the lead sponsor of 31 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
1 Year to 50 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

  • Diagnosis of cystinosis with more than 2 nmole half-cystine/mg protein in leukocytes OR presence of corneal crystals consistent with cystinosis and distributed in corneal stroma observed by slit lamp biomicroscopy
  • Clinical history consistent with cystinosis
  • Safety study: History of adherence with current eye drop and follow-up schedule on protocol #86-El-0062 Any crystal density score, including zero, on photographs, that has been stable or improved over the past year
  • Efficacy study: Crystal density score at least 1.00 on photographs Concurrently on cysteamine for at least the past 6 months

--Prior/Concurrent Therapy--

  • No prior cysteamine drops (efficacy study)

--Patient Characteristics--

  • Age: 1 to 50 (safety study) 2 to 12 (efficacy study)
  • Other: Willingness and ability to tolerate corneal photographs
05

Study design

Phase
Not applicable
Primary purpose
Treatment
Enrollment
30 participants

Interventions

  • Drugcysteamine hydrochloride
06

Study locations

No study locations are listed for this record.

07

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Mar 25, 2015, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
08

Registry details

Key details

Study ID
NCT00010426
Lead sponsor
FDA Office of Orphan Products Development
Collaborators
Leadiant Biosciences, Inc.
First posted
Feb 2, 2001
Start date
Dec 1999
Completion
Feb 2001
Last update
Mar 25, 2015

Study contacts

Edward F. Lemanowicz
study chair · Leadiant Biosciences, Inc.
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Apr 2001. You cannot join it, but the record below documents what was studied.

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