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

2019-10-18 11:21:36 | BioPortfolio


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






Not yet recruiting


Recordati Rare Diseases

Results (where available)

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Published on BioPortfolio: 2019-10-18T11:21:36-0400

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PubMed Articles [7 Associated PubMed Articles listed on BioPortfolio]

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Medical and Biotech [MESH] Definitions

A metabolic disease characterized by the defective transport of CYSTINE across the lysosomal membrane due to mutation of a membrane protein cystinosin. This results in cystine accumulation and crystallization in the cells causing widespread tissue damage. In the KIDNEY, nephropathic cystinosis is a common cause of RENAL FANCONI SYNDROME.

A radiation-protective agent that oxidizes in air to form CYSTAMINE. It can be given intravenously or orally to treat radiation sickness. The bitartrate has been used for the oral treatment of nephropathic cystinosis.

Compounds and drugs that react with CYSTINE and convert it into a compound that can be more easily metabolized or intracellularly transported. Drugs in this class have been used to treat CYSTINOSIS.

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Pediatrics is the general medicine of childhood. Because of the developmental processes (psychological and physical) of childhood, the involvement of parents, and the social management of conditions at home and at school, pediatrics is a specialty. With ...

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