Nephropathic cystinotic patients with cystine crystals corneal deposits MedDRA version: 9.1 Level: SOC Classification code 10010331 Term: Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: CYSTADROPS
Pharmaceutical Form: Eye drops*
INN or Proposed INN: cysteamine hydrochloride
CAS Number: 156-57-0
Other descriptive name: mercaptamine, mercamine, 2-aminoethanethiol
Concentr
Sponsors
Orphan Europe SARL
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Treated with reference Cysteamine eye drops at least for 1 month at an usual regimen dose of at least 3 instillations per day Able to comply with a regular dose regimen for 6 months Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Patients with uncontrolled hepatic disorder, cardiovascular disease, neurologic disease, or cancer, - Laboratory tests out of normal range according to the reference laboratory values. Deviations may be accepted if the investigator considers that they are not clinically significant for the conduct of study, - Patients with history or presence of alcohol abuse or drug addiction, - Pregnant or breast-feeding women, - Women of child-bearing potential without effective contraception (oral pill or IUCD), - Patients likely to be non-compliant to the study procedure or for whom a long-term follow-up seems to be difficult to achieve.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To compare safety of Cystadrops versus a recommended dose regimen reference treatment;Secondary Objective: - to compare efficacy of Cystadrops versus a recommended dose regimen reference treatment - to find the lowest efficient dose according to an empiric adaptive dose regimen algorithm - to evaluate the latest developments of measurement methods for corneal topography and cystine crystals deposit assessment. ;Primary end point(s): 1-Safety The safety will be based on the assessment and the recording of the adverse events, physical examinations, laboratory parameters and ocular findings including: oTransitional ocular symptoms evaluated by patient and/or parents: - Redness - Blurring - Irritation - Itching - Burning - Discomfort - Pain o Ocular tonometry o Eye fundus examination o Anterior segment topography (Optical Coherence Tomography, slit-scanning) 2- Efficacy o Clinical o Photophobia: 0-5 points scale o Pain: Visual Analogic Scale o Visual acuity (ETDRS chart, contrast scale) o Imaging: corneal crystals deposits measurement o HRT confocal biomicroscopy (Heidelberg Retina Tomograph) o Anterior segment topography (Optical Coherence Tomography) o Slit-lamp CCCS assessment (Cystinosis Corneal Crystal Score) 3- Compliance o Vials weight o Diary card | — |
Countries
France
Outcome results
None listed