CystinosisPhase 3SCOB2NCT04125927
Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)
A Phase 3 study testing Mercaptamine in people with Cystinosis, run by RECORDATI GROUP at 6 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 months to 2 years
- Conditions the study is for
- Cystinosis
- What is being tested?
- Mercaptamine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 6 sitesin 5 countries
- Study dates
- 1 Sep 2020to 23 Dec 2022
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 2 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
6 sites in 5 countries.
6 sites in 5 countries
United Kingdom2 sites
Great Ormond Street Hospital
London, WC1N3JH
Manchester Royal Eye Hospital
Manchester, M139WL
About this study
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
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
- Being tested
- Mercaptamine
- Medical subject headings
- Cystinosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MercaptamineDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Open-label arm | Experimental | Not described Receives: Drug: Mercaptamine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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)
Measured: 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)
Measured: Over a 90-day period
Secondary outcomes (3)
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.
Measured: 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.
Measured: 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.
Measured: 90-day period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2019
- Study start1 Sep 2020
- Primary completion23 Dec 2022
- Study completion23 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CystinosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- RECORDATI GROUP
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04125927
- Sponsor's protocol ID
- CYT-C2-001
- Acronym
- SCOB2
- Status last verified
- Feb 2024
- First submitted
- 3 Oct 2019
- Last updated
- 5 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04125927
- Acronym / sponsor ID
- SCOB2 / CYT-C2-001
- Conditions
- Cystinosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- RECORDATI GROUPIndustry (pharma, biotech, devices)
- Study start
- 1 Sep 2020 (actual)
- Primary completion
- 23 Dec 2022 (actual)
- Study completion
- 23 Dec 2022 (actual)
- First posted
- 14 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open-label arm | Experimental | Drug: Mercaptamine |
Who may qualify
- Ages
- 6 months to 2 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MercaptaminePatients 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. | Drug | Open-label arm |
Primary outcome
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)
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)
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 secondary outcomes
Ophtalmologic assessments (Best Corrected Visual Acuity)
Time frame: 90-day period
Ophtalmologic assessments (Corneal Cystine Crystal Score)
Time frame: 90-day period
Ophtalmologic assessments (Photophobia clinician-assessed evaluation according to a validated scale)
Time frame: 90-day period
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mercaptamine in people with Cystinosis, run by RECORDATI GROUP at 6 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United Kingdom, Belgium, France, Germany, Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2024.