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

NCT04125927 | Completed Phase 3

• 1 other identifier: CYT-C2-001
• Study Type: interventional
• Target: 5
• Locations: 5 countries
• Sites: 6

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.

Conditions

Cystinosis

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (3)

• Ophtalmologic assessments (Best Corrected Visual Acuity)

  90-day period

• Ophtalmologic assessments (Corneal Cystine Crystal Score)

  90-day period

• Ophtalmologic assessments (Photophobia clinician-assessed evaluation according to a validated scale)

  90-day period

Study Arms (1)

Open-label arm

EXPERIMENTAL

Drug: Mercaptamine

Interventions

Mercaptamine DRUG

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.

Open-label arm

Eligibility Criteria

• Age: 6 Months - 2 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• RECORDATI GROUP: lead

Study Sites (6)

UZ Leuven

Leuven, 3000, Belgium

Location

Hôpital Necker-Enfants Malades

Paris, France

Location

Klinik für Pädiatrische Nieren

Hanover, Germany

Location

Bambin Gesù Hospital in Palidoro

Roma, 00146, Italy

Location

Great Ormond Street Hospital

London, WC1N3JH, United Kingdom

Location

Manchester Royal Eye Hospital

Manchester, M139WL, United Kingdom

Location

MeSH Terms

Conditions

Cystinosis

Interventions

Cysteamine

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Mercaptoethylamines; Ethylamines; Amines; Organic Chemicals; Sulfhydryl Compounds; Sulfur Compounds

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 3, 2019
• First Posted: October 14, 2019
• Study Start: September 1, 2020
• Primary Completion: December 23, 2022
• Study Completion: December 23, 2022
• Last Updated: February 5, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

GB (2); BE (1); FR (1); DE (1); IT (1)