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This study is an international, multi-center, randomized, double-blind, placebo-controlled, two-treatment, two-period cross-over study to evaluate the pharmacodynamics, safety, tolerability and pharmacokinetics of a single oral dose of CDX-6114 in patients with phenylketonuria (PKU).
CDX 6114, Matching Placebo
Linear Clinical Research Ltd
Published on BioPortfolio: 2019-09-16T03:26:45-0400
The purpose of this study is to assess the safety and tolerability of an oral solution of CDX-6114 when administered as a single dose in healthy volunteers
The purpose of this study is to assess the safety and tolerability of an oral solution of CDX-6114 when administered as a single dose in healthy volunteers.
The PICO-Study is a randomized, placebo-controlled, crossover, non-inferiority trial conducted to add evidence to the current European treatment guidelines for adult patients with phenylke...
This study will evaluate SYNB1618 (investigational product) in healthy volunteers (HV) and subjects diagnosed with phenylketonuria (PKU) for the possible treatment of PKU. Phenylketonuria ...
The trial used single-center, randomized, double-blind, placebo-controlled, single-dose ascending study. The trial planned to enroll fifty-six healthy volunteers. The subjects were alloca...
Phenylketonuria (PKU), which is caused by mutations in the phenylalanine hydroxylase (PAH) gene, is one of the most common inherited diseases of amino acid metabolism. Phenylketonuria is characterized...
The standard, lifelong therapy of phenylketonuria (PKU) is a natural protein-restricted diet complemented with phenylalanine (Phe)-free L-amino acid mixtures that provide the daily necessary micronutr...
Dietetic treatment of phenylketonuria (PKU) includes a low-phenylalanine (phe) diet that provides sufficient phe for maintenance and growth plus special phe-free formulas with amino acids to meet requ...
This cohort study aimed to determine the frequency of overweight and obesity in classical phenylketonuria children and to identify the possible influence of metabolic control on the BMI of the studied...
In the past, the accuracy of surface matching has been shown to be disappointing. We aimed to determine whether this had improved over the years by assessing application accuracy of current navigation...
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).
Misunderstanding among individuals, frequently research subjects, of scientific methods such as randomization and placebo controls.
An exchange of DNA between matching or similar sequences.
An effect usually, but not necessarily, beneficial that is attributable to an expectation that the regimen will have an effect, i.e., the effect is due to the power of suggestion.
Clinical Research Organization
Contract Research Organization (CRO) provide research services outsourced on a contract basis to the pharmaceutical, biotechnology, healthcare and medical device industries: biopharmaceutical development biologic assay development commercial...