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Clinical Studies with CYSTARAN — an FDA-approved Ophthalmic Treatment for Corneal Crystals in Cystinosis1

Corneal slit-lamp photographs of patients treated with CYSTARAN2

Age: 5 years 9 months at start of treatment
Age: 25 years 4 months at start of treatment

In a prospective study, 67% of eyes showed corneal cystine crystal score (CCCS) reductions of ≥1 unit1,3

Study Design: Multicenter, randomized, double-blind efficacy trial of CYSTARAN in 15 treatment-naïve patients with a baseline CCCS
(0 units [clear at the center] to 3 units [highest crystal density]) of at least 1.25. The primary end point was the estimated proportion of eyes with a CCCS reduction ≥1 relative to baseline (where baseline CCCS was ≥1) anytime during the treatment period and at Months 3, 6, 9, and 12. Slit-lamp photography was used to assess CCCS changes from baseline.3

In the combined analysis of 3 clinical studies, sustained CCCS reductions and improvement in ocular complications1,3

  • Overall, 31% of eyes treated with CYSTARAN had a CCCS response
    —The greatest response—32%—was seen in eyes with CCCS ≥1.00 unit at baseline

Study Design: In the Combined Analysis of Patients Treated With Ophthalmic Cysteamine study, 247 patients who may have been concurrently receiving oral cysteamine were stratified into 2 modified intent-to-treat (mITT) arms (n=161). Primary end point was reduction of CCCS in eyes with high (≥1) CCCS at baseline and lack of increase in CCCS in eyes with low (<1) CCCS at baseline. End points were based on photo-rated CCCS (slit-lamp photography in conjunction with a photography-based scoring system) to quantify and document corneal cystine crystal accumulation over time.1,3

References: 1. CYSTARAN [prescribing information]. Gaithersburg, MD: Leadiant Biosciences, Inc.; 2020. 2. Gahl WA, Kuehl EM, Iwata F, Lindblad A, Kaiser-Kupfer MI. Corneal crystals in nephropathic cystinosis: natural history and treatment with cysteamine eyedrops. Mol Genet Metab. 2000;71:100-120.
3. Data on file. Leadiant Biosciences, Inc.


CYSTARAN (CYSTEAMINE OPHTHALMIC SOLUTION) 0.44% STERILE is a cystine-depleting agent indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis.

Important Safety Information:

To minimize contaminating the dropper tip and solution, care should be taken not to touch the eyelids or surrounding areas with the dropper tip of the bottle. Keep bottle tightly closed when not in use.

There have been reports of benign intracranial hypertension (or pseudotumor cerebri) associated with oral cysteamine treatment that has resolved with the addition of diuretic therapy. There have also been reports associated with ophthalmic use of cysteamine; however, all of these patients were on concurrent oral cysteamine.

CYSTARAN contains benzalkonium chloride, which may be absorbed by soft contact lenses. Contact lenses should be removed prior to application of solution and may be reinserted 15 minutes following its administration.

CYSTARAN is for topical ophthalmic use only.

The most frequently reported ocular adverse reactions occurring in ≥ 10% of patients were sensitivity to light, redness, and eye pain/irritation, headache and visual field defects.