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Completed

NCT Number: NCT04125927

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

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.

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Key information

Conditions

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

Age range

6 month–2 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

UZ Leuven, Leuven, Belgium

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

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.

Primary outcomes

  1. Ocular Serious Adverse Events, Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP

    Time frame: Over a 90-day period

    To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)

  2. Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP

    Time frame: Over a 90-day period

    To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)

  3. All Adverse Events that required discontinuation/withdrawal of IMP

    Time frame: Over a 90-day period

    To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)

Secondary outcomes

  1. Ophtalmologic assessments (Best Corrected Visual Acuity)

    Time frame: 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.

  2. Ophtalmologic assessments (Corneal Cystine Crystal Score)

    Time frame: 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.

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

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

RECORDATI GROUP

Industry

Registry information

Official study 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

Acronym: SCOB2

Important dates

Study start
2020
Primary completion
2022
Study completion
2022
First posted
Oct 14, 2019
Registry last updated
Feb 5, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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