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

Primary Purpose

Cystinosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Mercaptamine
Sponsored by
Recordati Rare Diseases
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystinosis

Eligibility Criteria

6 Months - 2 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • UZ Leuven
  • Hôpital Necker-Enfants Malades
  • Klinik für Pädiatrische Nieren
  • Bambin Gesù Hospital in Palidoro
  • Great Ormond Street Hospital
  • Manchester Royal Eye Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Open-label arm

Arm Description

Outcomes

Primary Outcome Measures

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)
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)
All Adverse Events that required discontinuation/withdrawal of IMP
To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)

Secondary Outcome Measures

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.
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.
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.

Full Information

First Posted
October 3, 2019
Last Updated
April 24, 2023
Sponsor
Recordati Rare Diseases
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1. Study Identification

Unique Protocol Identification Number
NCT04125927
Brief Title
Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)
Acronym
SCOB2
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
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Completed
Study Start Date
September 1, 2020 (Actual)
Primary Completion Date
December 23, 2022 (Actual)
Study Completion Date
December 23, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Recordati Rare Diseases

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystinosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Open-label arm
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Mercaptamine
Intervention Description
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 Measure Information:
Title
Ocular Serious Adverse Events, Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP
Description
To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)
Time Frame
Over a 90-day period
Title
Serious Adverse Reactions related to Cystadrops, all Adverse Events that required discontinuation/withdrawal of IMP
Description
To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)
Time Frame
Over a 90-day period
Title
All Adverse Events that required discontinuation/withdrawal of IMP
Description
To assess the safety profile of Cystadrops® over a 90-day period (ocular SAEs, SADRs)
Time Frame
Over a 90-day period
Secondary Outcome Measure Information:
Title
Ophtalmologic assessments (Best Corrected Visual Acuity)
Description
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.
Time Frame
90-day period
Title
Ophtalmologic assessments (Corneal Cystine Crystal Score)
Description
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.
Time Frame
90-day period
Title
Ophtalmologic assessments (Photophobia clinician-assessed evaluation according to a validated scale)
Description
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.
Time Frame
90-day period

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
UZ Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Hôpital Necker-Enfants Malades
City
Paris
Country
France
Facility Name
Klinik für Pädiatrische Nieren
City
Hannover
Country
Germany
Facility Name
Bambin Gesù Hospital in Palidoro
City
Roma
ZIP/Postal Code
00146
Country
Italy
Facility Name
Great Ormond Street Hospital
City
London
ZIP/Postal Code
WC1N3JH
Country
United Kingdom
Facility Name
Manchester Royal Eye Hospital
City
Manchester
ZIP/Postal Code
M139WL
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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