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 range6 Months – 2 Years

SexALL

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
✕. Participation in another ophthalmic investigational study or intent to participate during the course of the study
✕. Any medical condition that would, in the opinion of the Investigator, interfere with the evaluation of the study objectives

What they're measuring

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

Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP

Timeframe: Over a 90-day period

All Adverse Events that required discontinuation/withdrawal of IMP

Timeframe: Over a 90-day period

Trial details

NCT IDNCT04125927

SponsorRECORDATI GROUP

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-12-23

View on ClinicalTrials.gov More Cystinosis trials

Plain-language summary

Who can participate

Age range6 Months – 2 Years

SexALL

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
✕. Participation in another ophthalmic investigational study or intent to participate during the course of the study
✕. Any medical condition that would, in the opinion of the Investigator, interfere with the evaluation of the study objectives

What they're measuring

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

Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP

Timeframe: Over a 90-day period

All Adverse Events that required discontinuation/withdrawal of IMP

Timeframe: Over a 90-day period