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]

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] - SCOB2

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-002984-24-GB

Enrollment

Registered

2019-07-15

Start date

2019-10-25

Completion date

Unknown

Last updated

2019-12-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Corneal cystine crystal deposits in pediatric patients with nephropathic cystinosis from 6 months to less than 2 years old

Interventions

Trade Name: Cystadrops Product Name: Cystadrops Pharmaceutical Form: Eye drops, solution INN or Proposed INN: Mercaptamine hydrochloride CAS Number: 156-57-0 Current Sponsor code: Cystadrops Other des

Eligibility

Sex/Gender

All

Inclusion 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 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

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

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the safety profile of Cystadrops® over a 90-day period as measured by the incidence of: - Serious ocular Adverse Events (ocular SAEs) - Serious ADRs related to Cystadrops® - All AEs that required temporary discontinuation/withdrawal of treatment (IMP) or unscheduled/emergency ophthalmic visit(s) ;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 (Day 90): • Ocular Treatment Emergent SAEs, • SADRs related to Cystadrops® • All AEs that required temporary discontinuation/withdrawal of treatment (IMP) or unscheduled/emergency ophthalmic visit(s);Timepoint(s) of evaluation of this end point: Day 90.	—

Measure

Time frame

Main Objective: To assess the safety profile of Cystadrops® over a 90-day period as measured by the incidence of: - Serious ocular Adverse Events (ocular SAEs) - Serious ADRs related to Cystadrops® - All AEs that required temporary discontinuation/withdrawal of treatment (IMP) or unscheduled/emergency ophthalmic visit(s) ;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 (Day 90): • Ocular Treatment Emergent SAEs, • SADRs related to Cystadrops® • All AEs that required temporary discontinuation/withdrawal of treatment (IMP) or unscheduled/emergency ophthalmic visit(s);Timepoint(s) of evaluation of this end point: Day 90.

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Secondary

Measure	Time frame
Secondary end point(s): Change from Day 1 (Inclusion) to Day 90 (EOS) in CCCS, photophobia, and BCVA.;Timepoint(s) of evaluation of this end point: Day 90.	—

Countries

France, United Kingdom

Contacts

Public ContactSonia Davenne

ICON plc

sonia.davenne@iconplc.com07795850529

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026