For US Residents Only

Prescribing Information


Home > For Healthcare Professionals > Clinical Studies With CYSTARAN

Clinical Studies with CYSTARAN — the ONLY 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.; 2012. 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 is a cystine-depleting agent indicated for the treatment of corneal cystine crystal accumulation in patients with cystinosis.

Important Safety Information:

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