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

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

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.

Study Overview

• Status: Completed
• Conditions: Cystinosis
• Intervention / Treatment: Drug: Mercaptamine

• Study Type: Interventional
• Enrollment (Actual): 5
• Phase: Phase 3

Contacts and Locations

Study Locations

• Belgium
  • Leuven, Belgium, 3000
    • UZ Leuven
• France
  • Paris, France
    • Hôpital Necker-Enfants Malades
• Germany
  • Hannover, Germany
    • Klinik für Pädiatrische Nieren
• Italy
  • Roma, Italy, 00146
    • Bambin Gesù Hospital in Palidoro
• United Kingdom
  • London, United Kingdom, WC1N3JH
    • Great Ormond Street Hospital
  • Manchester, United Kingdom, M139WL
    • Manchester Royal Eye Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 2 years (Child)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Open-label arm	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.

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Recordati Rare Diseases

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2020
• Primary Completion (Actual): December 23, 2022
• Study Completion (Actual): December 23, 2022

Study Registration Dates

• First Submitted: October 3, 2019
• First Submitted That Met QC Criteria: October 11, 2019
• First Posted (Actual): October 14, 2019

Study Record Updates

• Last Update Posted (Estimated): February 5, 2024
• Last Update Submitted That Met QC Criteria: February 2, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Cystinosis; Molecular Mechanisms of Pharmacological Action; Cystine Depleting Agents; Cysteamine
• Other Study ID Numbers: CYT-C2-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No