Topics

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

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

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.

Study Design

Conditions

Cystinosis

Intervention

Mercaptamine

Status

Not yet recruiting

Source

Recordati Rare Diseases

Results (where available)

View Results

Links

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

Clinical Trials [20 Associated Clinical Trials listed on BioPortfolio]

Clinical and Biological Study of Sub-Pigmentation During Infantile Cystinosis

The purpose of this study is to determine the molecular mechanism of this disease and to research the relationship between cystinosis and skin phenotype.

Evaluation of Fanconi Syndrome and Cystinosis

OBJECTIVES: I. Classify renal tubular defects using clinical and biochemical findings in patients with Fanconi syndrome and cystinosis.

Establishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis

OBJECTIVES: I. Establish a computerized databank to monitor the progress of patients with cystinosis treated with cysteamine. II. Track and monitor all patients including renal transpla...

Biomarker for Patient With Cystinosis Disease or Highly Suspected for Cystinosis Disease

Development of a new MS-based biomarker for the early and sensitive diagnosis of Cystinosis disease from plasma.

Use of Cysteamine in the Treatment of Cystinosis

Cystinosis is an inherited disease resulting in poor growth and kidney failure. There is no known cure for cystinosis, although kidney transplantation may help the renal failure and prolo...

PubMed Articles [7 Associated PubMed Articles listed on BioPortfolio]

Visual and verbal learning and memory in cystinosis.

Cystinosis is a rare genetic lysosomal storage disorder characterized by the accumulation of cystine in lysosomes. Many organ systems are vulnerable to this cystine accumulation including the CNS. A p...

Central nervous system complications in adult cystinosis patients.

Little is known about the long-term progression of adult nephropathic cystinosis patients. Our objective was to study central nervous system complications in cystinosis patients in the era of early cy...

The novel aminoglycoside, ELX-02, permits CTNSW138X translational read-through and restores lysosomal cystine efflux in cystinosis.

Cystinosis is a rare disorder caused by recessive mutations of the CTNS gene. Current therapy decreases cystine accumulation, thus slowing organ deterioration without reversing renal Fanconi syndrome ...

Management of bone disease in cystinosis: Statement from an international conference.

Cystinosis is an autosomal recessive storage disease due to impaired transport of cystine out of lysosomes. Since the accumulation of intracellular cystine affects all organs and tissues, the manageme...

Vitamin D repletion ameliorates adipose tissue browning and muscle wasting in infantile nephropathic cystinosis-associated cachexia.

Ctns mice, a mouse model of infantile nephropathic cystinosis, exhibit hypermetabolism with adipose tissue browning and profound muscle wasting. Ctns mice are 25(OH)D and 1,25(OH) D insufficient. We i...

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.

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

Quick Search

Relevant Topic

Pediatrics
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 ...


Searches Linking to this Trial