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

Cystinosis Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04125927
• Other study ID #: CYT-C2-001
• Status: Completed
• Phase: Phase 3
• Start date: September 1, 2020
• Est. completion date: December 23, 2022

Study information

• Verified date: February 2024
• Source: Recordati Rare Diseases
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 5
• Est. completion date: December 23, 2022
• Est. primary completion date: December 23, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 2 Years

Eligibility

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

Related Conditions & MeSH terms

• Cystinosis

Intervention

Drug:
• Mercaptamine
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.

Locations

Country	Name	City	State
Belgium	UZ Leuven	Leuven
France	Hôpital Necker-Enfants Malades	Paris
Germany	Klinik für Pädiatrische Nieren	Hannover
Italy	Bambin Gesù Hospital in Palidoro	Roma
United Kingdom	Great Ormond Street Hospital	London
United Kingdom	Manchester Royal Eye Hospital	Manchester

Sponsors (1)

Lead Sponsor	Collaborator
Recordati Rare Diseases

Countries where clinical trial is conducted

Belgium,  France,  Germany,  Italy,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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	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
Secondary	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
Secondary	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