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  • A multicentre randomised double masked clinical trial of a new formulation of topical cysteamine for the treatment of corneal cystine crystals in cystinosis

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Scientific correspondence
A multicentre randomised double masked clinical trial of a new formulation of topical cysteamine for the treatment of corneal cystine crystals in cystinosis
  1. E T Tsilou1,
  2. D Thompson2,
  3. A S Lindblad2,
  4. G F Reed3,
  5. B Rubin1,
  6. W Gahl4,
  7. J Thoene5,
  8. M Del Monte6,
  9. J A Schneider7,
  10. D B Granet8,
  11. M I Kaiser-Kupfer1
  1. 1Ophthalmic Genetics and Visual Function Branch, National Eye Institute, National Institutes of Health, Bethesda, MD, USA
  2. 2The EMMES Corporation, USA
  3. 3Division of Epidemiology and Clinical Research, National Eye Institute, Bethesda, MD, USA
  4. 4Section on Human Biochemical Genetics, Heritable Disorders Branch, National Institute of Child Health and Human Development, Bethesda, MD, USA
  5. 5Hayward Genetic Center, Tulane University Health Science Center, New Orleans, LA, USA
  6. 6Department of Pediatric Ophthalmology, University of Michigan School of Medicine, Ann Arbor, MI, USA
  7. 7Department of Pediatrics, University of California, San Diego School of Medicine, La Jolla, CA, USA
  8. 8Departments of Pediatrics and Ophthalmology, University of California, San Diego School of Medicine, La Jolla, CA, USA
  1. Correspondence to: Ekaterini Tsilou, MD, Ophthalmic Genetics and Visual Function Branch, National Eye Institute, National Institutes of Health, 10 Center Drive, MSC-1860, Building 10, Room 10N226, Bethesda, MD 20892, USA; tsiloue{at}nei.nih.gov

Abstract

Aim: To evaluate the safety and efficacy of a new topical cysteamine formulation, stable at room temperature, for the treatment of corneal cystine crystals in cystinosis.

Methods: 20 study subjects were enrolled in the safety study and 16 in the efficacy study. Both studies were randomised and double blind. The primary outcome for the safety study was the occurrence of predefined serious adverse reactions over 6 months and for the efficacy study the reduction of corneal cystine crystal score (CCCS) by 1.00 or more units on photographs graded by a reading centre using a standardised protocol.

Results: No study subject developed any serious adverse reactions. In the efficacy study, 47% of eyes receiving the standard formulation experienced a reduction in the CCCS of ≥1.00 after 1 year, while 7% of eyes on the new formulation experienced such a decrease (p=0.04).

Conclusion: Although no serious adverse reactions were observed with either formulation, the new formulation was not as effective as the standard formulation.

  • cysteamine
  • cystinosis
  • corneal crystals

https://doi.org/10.1136/bjo.87.1.28

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