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

Phase 3

Completed

• Conditions: Cystinosis
• Interventions: Drug: Mercaptamine

• Registration Number: NCT04125927
• Lead Sponsor: Recordati Rare Diseases

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-10-03
• Study First Submitted QC: 2019-10-11
• Study First Posted: 2019-10-14
• Study Start: 2020-09-01
• Primary Completion: 2022-12-23
• Study Completion: 2022-12-23
• Status Verified: 2024-02
• Last Update Submitted: 2024-02-02
• Last Update Posted: 2024-02-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 5

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open-label arm	Mercaptamine	-

Primary Outcome Measures

Name	Time	Method
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)
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)

Secondary Outcome Measures

Name	Time	Method
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 (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 (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.

Trial Locations

Locations (6)

UZ Leuven; 🇧🇪 Leuven, Belgium

Hôpital Necker-Enfants Malades; 🇫🇷 Paris, France

Great Ormond Street Hospital; 🇬🇧 London, United Kingdom

Klinik für Pädiatrische Nieren; 🇩🇪 Hannover, Germany

Manchester Royal Eye Hospital; 🇬🇧 Manchester, United Kingdom

Bambin Gesù Hospital in Palidoro; 🇮🇹 Roma, Italy