PMID- 28426870
OWN - NLM
STAT- MEDLINE
DCOM- 20170719
LR  - 20180118
IS  - 1552-5783 (Electronic)
IS  - 0146-0404 (Linking)
VI  - 58
IP  - 4
DP  - 2017 Apr 1
TI  - A New Viscous Cysteamine Eye Drops Treatment for Ophthalmic Cystinosis: An 
      Open-Label Randomized Comparative Phase III Pivotal Study.
PG  - 2275-2283
LID - 10.1167/iovs.16-21080 [doi]
AB  - PURPOSE: The purpose of this study was to evaluate the efficacy of new viscous 
      cysteamine hydrochloride (CH) eye drops (vCH 0.55%) compared with standard CH 
      0.10% drops treatment. METHODS: This was an open-label, phase III, randomized, 
      two-arm multicenter trial conducted at two centers in France. Cystinosis patients 
      >/=2 years old were randomized 1:1 to receive eye drops, four times per day for 90 
      days in both eyes. We compared the superiority in reducing corneal cystine 
      crystal density as assessed by in vivo confocal microscopy (IVCM). We also 
      evaluated photophobia, corneal cystine crystal scores (CCCSs), and cystine 
      crystal depth measured by optical coherence tomography. Safety objectives were to 
      assess adverse events (AEs), local adverse drug reactions, and ocular safety 
      parameters. RESULTS: We included 15 patients with vCH 0.55% and 16 patients with 
      CH 0.10% drops for 90 days. The mean absolute change in IVCM total score at day 
      90 in the vCH 0.55% drops group (-4.6 +/- 3.1) was significantly greater than and 
      superior to the mean absolute change in the CH 0.10% drops group (-0.46 +/- 3.38; P 
      < 0.0001). Photophobia, CCCS, and corneal cystine crystal depth were 
      significantly more improved in the vCH 0.55% drops group than in the CH 0.10% 
      group. The most frequent local adverse drug reactions in both groups were 
      stinging, burning, redness, and blurred vision. CONCLUSIONS: vCH 0.55% was 
      effective in reducing corneal cystine crystal density and superior to treatment 
      with CH 0.10% drops, which offer advantages over hospital pharmacy formulations 
      and is a more preferable and convenient treatment option.
FAU - Liang, Hong
AU  - Liang H
AD  - Department of Ophthalmology III, Department Hospitalo-Universitaire View 
      Maintain, INSERM-DHOS, and Center of Clinical Investigations 1423, Quinze-Vingts 
      National Ophthalmology Hospital, Institut de la Vision, Universite 
      Pierre-et-Marie-Curie University Paris 06, Paris, France.
FAU - Labbe, Antoine
AU  - Labbe A
AD  - Department of Ophthalmology III, Department Hospitalo-Universitaire View 
      Maintain, INSERM-DHOS, and Center of Clinical Investigations 1423, Quinze-Vingts 
      National Ophthalmology Hospital, Institut de la Vision, Universite 
      Pierre-et-Marie-Curie University Paris 06, Paris, France 2Department of 
      Ophthalmology, Ambroise Pare Hospital, APHP, University of Versailles 
      Saint-Quentin-en-Yvelines, Versailles, France.
FAU - Le Mouhaer, Jeannie
AU  - Le Mouhaer J
AD  - Orphan Europe, Medical Affairs Department, Puteaux, France.
FAU - Plisson, Celine
AU  - Plisson C
AD  - Orphan Europe, Medical Affairs Department, Puteaux, France.
FAU - Baudouin, Christophe
AU  - Baudouin C
AD  - Department of Ophthalmology III, Department Hospitalo-Universitaire View 
      Maintain, INSERM-DHOS, and Center of Clinical Investigations 1423, Quinze-Vingts 
      National Ophthalmology Hospital, Institut de la Vision, Universite 
      Pierre-et-Marie-Curie University Paris 06, Paris, France 2Department of 
      Ophthalmology, Ambroise Pare Hospital, APHP, University of Versailles 
      Saint-Quentin-en-Yvelines, Versailles, France.
LA  - eng
PT  - Clinical Trial, Phase III
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
PL  - United States
TA  - Invest Ophthalmol Vis Sci
JT  - Investigative ophthalmology & visual science
JID - 7703701
RN  - 0 (Cystine Depleting Agents)
RN  - 0 (Ophthalmic Solutions)
RN  - 48TCX9A1VT (Cystine)
RN  - 5UX2SD1KE2 (Cysteamine)
SB  - IM
MH  - Adolescent
MH  - Cornea/*metabolism/pathology
MH  - Corneal Diseases/diagnosis/*drug therapy/metabolism
MH  - Cysteamine/*administration & dosage
MH  - Cystine/drug effects/*metabolism
MH  - Cystine Depleting Agents/administration & dosage
MH  - Cystinosis/diagnosis/*drug therapy/metabolism
MH  - Dose-Response Relationship, Drug
MH  - Female
MH  - Follow-Up Studies
MH  - Humans
MH  - Male
MH  - Microscopy, Confocal
MH  - Ophthalmic Solutions
MH  - Retrospective Studies
MH  - Tomography, Optical Coherence
MH  - Treatment Outcome
MH  - Visual Acuity
MH  - Young Adult
EDAT- 2017/04/21 06:00
MHDA- 2017/07/20 06:00
CRDT- 2017/04/21 06:00
PHST- 2017/04/21 06:00 [entrez]
PHST- 2017/04/21 06:00 [pubmed]
PHST- 2017/07/20 06:00 [medline]
AID - 2621983 [pii]
AID - 10.1167/iovs.16-21080 [doi]
PST - ppublish
SO  - Invest Ophthalmol Vis Sci. 2017 Apr 1;58(4):2275-2283. doi: 
      10.1167/iovs.16-21080.