Cystadrops® is currently indicated in adults and children from 2 years of age diagnosed with cystinosis with corneal crystal accumulation observed. However administration of Cystadrops® in patients below 2 years old could be of value for these patients and prevent the crystal deposit. It is the reason why as part of the Cystadrops® pediatric investigational plan (PIP), RECORDATI Rare Diseases committed to conduct a clinical study to assess Cystadrops® safety and efficacy in the pediatric population from 6 months to less than 2 years old.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
5
Patients will be treated with 1 drop in each eye 4 times a day, at the same dose and regimen than the one indicated in adults and children from 2 years of age.
UZ Leuven
Leuven, Belgium
Hôpital Necker-Enfants Malades
Paris, France
Klinik für Pädiatrische Nieren
Hanover, Germany
Bambin Gesù Hospital in Palidoro
Roma, Italy
Ocular Serious Adverse Events, Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP
To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)
Time frame: Over a 90-day period
Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP
To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)
Time frame: Over a 90-day period
All Adverse Events that required discontinuation/withdrawal of IMP
To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)
Time frame: Over a 90-day period
Ophtalmologic assessments (Best Corrected Visual Acuity)
To assess the efficacy of Cystadrops® by measuring best corrected visual acuity (BCVA) of both eyes after 90 days of treatment with Cystadrops® when possible considering the age of the patients.
Time frame: 90-day period
Ophtalmologic assessments (Corneal Cystine Crystal Score)
To assess the efficacy of Cystadrops® by measuring Corneal cystine crystal score (CCCS), of both eyes after 90 days of treatment with Cystadrops® when possible considering the age of the patients.
Time frame: 90-day period
Ophtalmologic assessments (Photophobia clinician-assessed evaluation according to a validated scale)
To assess the efficacy of Cystadrops® by measuring photophobia of both eyes using a validated scale after 90 days of treatment with Cystadrops® when possible considering the age of the patients.
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Great Ormond Street Hospital
London, United Kingdom
Manchester Royal Eye Hospital
Manchester, United Kingdom
Time frame: 90-day period