Clinical trial

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

Name

CYT-C2-001

Description

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.

Trial arms

Trial start

2020-09-01

Estimated PCD

2022-12-23

Trial end

2022-12-23

Status

Completed

Phase

Early phase I

Treatment

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.

Arms:

Open-label arm

Size

Primary endpoint

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

Over a 90-day period

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

Over a 90-day period

All Adverse Events that required discontinuation/withdrawal of IMP

Over a 90-day period

Eligibility 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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 5, 'type': 'ACTUAL'}}

Updated at

2024-02-05

1 organization

1 product

1 indication

Product

Mercaptamine

Indication

Cystinosis

Organization

Recordati Rare Diseases