Open-label, Single-arm, Multicenter Study to Assess the Safety of, Cystadrops® in Pediatric Cystinosis Patients,from 6 Months to Less Than 2 Years Old [SCOB2 Study]

Phase 1

• Conditions: Corneal cystine crystal deposits in pediatric patients withnephropathic cystinosis from 6 months to less than 2 years old; Therapeutic area: Diseases [C] - Eye Diseases [C11]

• Registration Number: EUCTR2018-002984-24-GB
• Lead Sponsor: RECORDATI Rare Diseases SAR

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-07-15
• Last Update Posted: 16 December 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

1.Patient aged from 6 months to less than 2 years old
2.Cystinosis diagnosed patients confirmed by the physician and with presence of corneal cystine crystal deposits assessed during ophthalmic examination
3.Evidence of a signed and dated informed consent document indicating that parents/ legally acceptable representatives had been informed of all pertinent aspects of the study (if required by regulation)
4.Parents/ legally acceptable representatives who are willing to comply with regular visits and ophthalmic exams

Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

1.Contraindications to any of the Cystadrops® components
2.Participation in another ophthalmic investigational study or intent to participate during the course of the study
3.Any medical condition that would, in the opinion of the Investigator, interfere with the evaluation of the study objectives

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the safety profile of Cystadrops® over a 90-day period as measured by the incidence of:<br>-Serious ocular Adverse Events (ocular SAEs)<br>-Serious ADRs related to Cystadrops®<br>-All AEs that required temporary discontinuation/withdrawal of treatment (IMP) or unscheduled/emergency ophthalmic visit(s)<br>;Secondary Objective: To assess the efficacy of Cystadrops® by measuring ophthalmologic assessments (CCCS, photophobia and BCVA) after 90 days of treatment with Cystadrops® when possible considering the age of the patients.;Primary end point(s): Occurrence of the following safety criteria between signature of the ICF and the EOS visit<br>(Day 90):<br>• Ocular Treatment Emergent SAEs,<br>• SADRs related to Cystadrops®<br>• All AEs that required temporary discontinuation/withdrawal of treatment (IMP) or<br>unscheduled/emergency ophthalmic visit(s);Timepoint(s) of evaluation of this end point: Day 90.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Change from Day 1 (Inclusion) to Day 90 (EOS) in CCCS,<br>photophobia, and BCVA.;Timepoint(s) of evaluation of this end point: Day 90.