Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2) (NCT04125927) | Clinical Trial Compass
CompletedPhase 3
Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)
Belgium, France, Germany5 participantsStarted 2020-09-01
Plain-language summary
Cystadrops® is currently indicated in adults and children from 2 years of age diagnosed with cystinosis with corneal crystal accumulation observed.
However administration of Cystadrops® in patients below 2 years old could be of value for these patients and prevent the crystal deposit. It is the reason why as part of the Cystadrops® pediatric investigational plan (PIP), RECORDATI Rare Diseases committed to conduct a clinical study to assess Cystadrops® safety and efficacy in the pediatric population from 6 months to less than 2 years old.
Who can participate
Age range
6 Months – 2 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Patient aged from 6 months to less than 2 years old
. Cystinosis diagnosed patients confirmed by the physician and with presence of corneal cystine crystal deposits assessed during ophthalmic examination
. 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)
. Parents/ legally acceptable representatives who are willing to comply with regular visits and ophthalmic exams
Exclusion criteria
. Contraindications to any of the Cystadrops® components
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Ocular Serious Adverse Events, Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP
Timeframe: Over a 90-day period
2
Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP
Timeframe: Over a 90-day period
3
All Adverse Events that required discontinuation/withdrawal of IMP