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Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

2014-07-24 14:18:29 | BioPortfolio

Summary

The purpose of this study is to assess the safety and tolerability of injections of rAvPAL-PEG in subjects with PKU.

Study Design

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Phenylketonuria

Intervention

rAvPAL-PEG

Location

Children's Memorial Hospital
Chicago
Illinois
United States
60614

Status

Completed

Source

BioMarin Pharmaceutical

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-07-24T14:18:29-0400

Clinical Trials [32 Associated Clinical Trials listed on BioPortfolio]

Long-Term Extension of a Dose Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With Phenylketonuria (PKU) (PAL-003)

This study is an extension of the dose-finding study (Study PAL-002). Administration of rAvPAL-PEG will be continued to assess whether long-term dosing of rAvPAL-PEG is safe and can mainta...

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

The purpose of this study is to evaluate whether weekly injections of phenylalanine ammonia lyase (rAvPAL-PEG) can reduce blood phenylalanine concentrations in PKU subjects and whether rep...

Behavioral Effects of Kuvan in Children With Mild Phenylketonuria

The purpose of this study is to determine whether improvements in behavior occur in children with phenylketonuria (PKU) who are taking Kuvan.

Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria

This study will evaluate SYNB1618 (investigational product) in healthy volunteers (HV) and subjects diagnosed with phenylketonuria (PKU) for the possible treatment of PKU. Phenylketonuria ...

A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults

The 165-901 study is designed to identify the appropriate tools for use in future interventional studies on the neurocognitive effects of pegvaliase on adults with PKU.

PubMed Articles [22 Associated PubMed Articles listed on BioPortfolio]

Metabolomic Markers of Essential Fatty Acids, Carnitine, and Cholesterol Metabolism in Adults and Adolescents with Phenylketonuria.

Individuals with phenylketonuria (PKU) have a risk of cognitive impairment and inflammation. Many follow a low-phenylalanine (low-Phe) diet devoid of animal protein in combination with medical foods (...

Non-invasive prenatal testing of pregnancies at risk for phenylketonuria.

Phenylketonuria (PKU) is a common metabolic disorder caused predominately by mutations in the phenylalanine hydroxylase (PAH) gene. The aim of the study was to design and validate the performance of a...

Clinical and electrophysiological findings in patients with phenylketonuria and epilepsy: Reflex features.

Phenylketonuria (PKU) is the most common form of amino acid metabolism disorders with autosomal recessive inheritance. The brain damage can be prevented by early diagnosis and a phenylalanine-restrict...

Maternal Phenylketonuria syndrome: Studies in mice suggest a potential approach to a continuing problem.

Untreated Phenylketonuria (PKU), one of the most common human genetic disorders, usually results in mental retardation. While a protein-restricted artificial diet can prevent retardation, dietary comp...

Aptamer-based assay for monitoring genetic disorder phenylketonuria (PKU).

The genetic disorder phenylketonuria (PKU) is the inability to metabolize phenylalanine because of a lack of the enzyme phenylalanine hydroxylase. Phenylalanine is used to biochemically form proteins,...

Medical and Biotech [MESH] Definitions

A condition occurring in untreated or partially treated females with PHENYLKETONURIA when they become pregnant. This may result in damages to the FETUS, including MICROCEPHALY; MENTAL RETARDATION; congenital heart disease; FETAL GROWTH RETARDATION; and CRANIOFACIAL ABNORMALITIES. (From Am J Med Genet 1997 Mar 3;69(1):89-95)

A group of autosomal recessive disorders marked by a deficiency of the hepatic enzyme PHENYLALANINE HYDROXYLASE or less frequently by reduced activity of DIHYDROPTERIDINE REDUCTASE (i.e., atypical phenylketonuria). Classical phenylketonuria is caused by a severe deficiency of phenylalanine hydroxylase and presents in infancy with developmental delay; SEIZURES; skin HYPOPIGMENTATION; ECZEMA; and demyelination in the central nervous system. (From Adams et al., Principles of Neurology, 6th ed, p952).

An enzyme that catalyzes the reduction of 6,7-dihydropteridine to 5,6,7,8-tetrahydropteridine in the presence of NADP+. Defects in the enzyme are a cause of PHENYLKETONURIA II. Formerly listed as EC 1.6.99.7.

An enzyme of the oxidoreductase class that catalyzes the formation of L-TYROSINE, dihydrobiopterin, and water from L-PHENYLALANINE, tetrahydrobiopterin, and oxygen. Deficiency of this enzyme may cause PHENYLKETONURIAS and PHENYLKETONURIA, MATERNAL. EC 1.14.16.1.

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