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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Cystinosis

Intervention / Treatment
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* Drug: Mercaptamine


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 2 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Open-label arm<br> | Drug: Mercaptamine<br>* Patients will be treated with 1 drop in each eye 4 times a day, at the same dose and regimen than the one indicated in adults and children from 2 years of age.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Ocular Serious Adverse Events, Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP | To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs) | Over a 90-day period | 
| Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP | To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs) | Over a 90-day period | 
| All Adverse Events that required discontinuation/withdrawal of IMP | To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs) | Over a 90-day period | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Ophtalmologic assessments (Best Corrected Visual Acuity) | To assess the efficacy of Cystadrops® by measuring best corrected visual acuity (BCVA) of both eyes after 90 days of treatment with Cystadrops® when possible considering the age of the patients. | 90-day period | 
| Ophtalmologic assessments (Corneal Cystine Crystal Score) | To assess the efficacy of Cystadrops® by measuring Corneal cystine crystal score (CCCS), of both eyes after 90 days of treatment with Cystadrops® when possible considering the age of the patients. | 90-day period | 
| Ophtalmologic assessments (Photophobia clinician-assessed evaluation according to a validated scale) | To assess the efficacy of Cystadrops® by measuring photophobia of both eyes using a validated scale after 90 days of treatment with Cystadrops® when possible considering the age of the patients. | 90-day period |