Clinical trial NCT04125927: Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystinosis
Lead sponsor: Recordati Rare Diseases
Sponsor class: INDUSTRY
First posted: 2019-10-14
Enrollment: 5
Sex: ALL
Minimum age (years): 6
Maximum age (years): 2
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility 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

Primary outcomes:
measure: 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
measure: Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP
timeFrame: Over a 90-day period
measure: All Adverse Events that required discontinuation/withdrawal of IMP
timeFrame: Over a 90-day period
Secondary outcomes:
measure: Ophtalmologic assessments (Best Corrected Visual Acuity)
timeFrame: 90-day period
measure: Ophtalmologic assessments (Corneal Cystine Crystal Score)
timeFrame: 90-day period
measure: Ophtalmologic assessments (Photophobia clinician-assessed evaluation according to a validated scale)
timeFrame: 90-day period
Other outcomes:
Locations:
city: Leuven
country: Belgium
facility: UZ Leuven
city: Paris
country: France
facility: Hôpital Necker-Enfants Malades
city: Hannover
country: Germany
facility: Klinik für Pädiatrische Nieren
city: Roma
country: Italy
facility: Bambin Gesù Hospital in Palidoro
city: London
country: United Kingdom
facility: Great Ormond Street Hospital
city: Manchester
country: United Kingdom
facility: Manchester Royal Eye Hospital
Study officials:
References: