Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 February 2024
• Main ID: NCT04125927
• Date of registration: 03/10/2019
• Prospective Registration: Yes
• Primary sponsor: Recordati Rare Diseases
• Public title: Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2) SCOB2
• Scientific title: 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
• Date of first enrolment: September 1, 2020
• Target sample size: 5
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT04125927
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Belgium, France, Germany, Italy, United Kingdom

Key inclusion & exclusion criteria

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

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



Age minimum: 6 Months
Age maximum: 2 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cystinosis

Intervention(s)

Drug: Mercaptamine

Primary Outcome(s)

Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP [Time Frame: Over a 90-day period]

Ocular Serious Adverse Events, Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP [Time Frame: Over a 90-day period]

All Adverse Events that required discontinuation/withdrawal of IMP [Time Frame: Over a 90-day period]

Secondary Outcome(s)

Ophtalmologic assessments (Best Corrected Visual Acuity) [Time Frame: 90-day period]

Ophtalmologic assessments (Corneal Cystine Crystal Score) [Time Frame: 90-day period]

Ophtalmologic assessments (Photophobia clinician-assessed evaluation according to a validated scale) [Time Frame: 90-day period]

Secondary ID(s)

CYT-C2-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors