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

Phase 1

• Conditions: ephropatic Cystinosis patients with corneal cystine crystal deposits; MedDRA version: 20.0 Level: LLT Classification code 10071112 Term: Nephropathic cystinosis System Organ Class: 100000004850; Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

• Registration Number: EUCTR2018-002984-24-FR
• Lead Sponsor: Recordati Rare Diseases

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-08-19
• Last Update Posted: 10 December 2019

Recruitment & Eligibility

• Status: A
• Sex: Not specified
• Target Recruitment: 5

Inclusion Criteria

Patients must meet all inclusion criteria to be eligible for study participation.
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
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients will not be eligible for study participation if they meet any of the exclusion criteria, or will be discontinued at the discretion of the Investigator in consultation with the medical monitor if they develop any of the exclusion criteria during the study.
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