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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04125927
Recruitment Status : Recruiting
First Posted : October 14, 2019
Last Update Posted : May 24, 2022
Sponsor:
Information provided by (Responsible Party):
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.


Condition or disease Intervention/treatment Phase
Cystinosis Drug: Mercaptamine Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : September 1, 2020
Estimated Primary Completion Date : December 31, 2022
Estimated Study Completion Date : August 3, 2023


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.




Primary Outcome Measures :
  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 Outcome Measures :
  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.



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Ages Eligible for Study:   6 Months to 2 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04125927


Locations
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France
Hôpital Necker-Enfants Malades Recruiting
Paris, France
Contact: Hôpital Necker Enfants Malades    0144494000    dominique.bremond@aphp.fr   
Sponsors and Collaborators
Recordati Rare Diseases
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Responsible Party: Recordati Rare Diseases
ClinicalTrials.gov Identifier: NCT04125927    
Other Study ID Numbers: CYT-C2-001
First Posted: October 14, 2019    Key Record Dates
Last Update Posted: May 24, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystinosis
Lysosomal Storage Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Cysteamine
Cystine Depleting Agents
Molecular Mechanisms of Pharmacological Action