Cystinosis
Conditions
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.
Interventions
DRUGMercaptamine
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.
Sponsors
RECORDATI GROUP
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Months to 2 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Ocular Serious Adverse Events, Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP | Over a 90-day period | To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs) |
| Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP | Over a 90-day period | To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs) |
| All Adverse Events that required discontinuation/withdrawal of IMP | Over a 90-day period | To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Ophtalmologic assessments (Best Corrected Visual Acuity) | 90-day period | 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. |
| Ophtalmologic assessments (Corneal Cystine Crystal Score) | 90-day period | 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. |
| Ophtalmologic assessments (Photophobia clinician-assessed evaluation according to a validated scale) | 90-day period | 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. |
Countries
Belgium, France, Germany, Italy, United Kingdom
Outcome results
None listed