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Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period

2014-08-27 03:15:24 | BioPortfolio

Summary

The primary objective of the study is to evaluate the proportion of responders [≥30% reduction from baseline in blood phenylalanine (Phe) level] to 20 mg/kg/day sapropterin dihydrochloride treatment at several time points during 28 days.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care

Conditions

Phenylketonuria

Intervention

Kuvan® (Sapropterin dihydrochloride)

Location

Department of Paediatric Research, Division of Paediatrics, Oslo University Hospital, Rikshospitalet
Oslo
Norway

Status

Recruiting

Source

Merck KGaA

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:15:24-0400

Clinical Trials [76 Associated Clinical Trials listed on BioPortfolio]

A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects

Sapropterin dihydrochloride (subsequently referred to as sapropterin) (Kuvan®) was approved by the FDA for the treatment of hyperphenylalaninemia in 2007. Preclinical and clinical studies...

The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)

The objective of the study is to determine whether Kuvan™ (sapropterin) improves the strength of the functional connectivity between brain regions in individuals with PKU.

Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

This double-blind, placebo-controlled, randomized study is designed to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects w...

PKUDOS: PKU Demographic, Outcomes, and Safety Registry

The objective of this study is to evaluate the safety of long-term treatment with Kuvan.

Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU

This multicenter, open label study is designed to evaluate the safety of Kuvan® and its effect on neurocognitive function, blood Phe concentration, and growth in children with PKU who are...

PubMed Articles [42 Associated PubMed Articles listed on BioPortfolio]

The impact of phenylketonuria on PKU patients' quality of life: Using of the phenylketonuria-quality of life (PKU-QOL) questionnaires.

Phenylketonuria (PKU) has a very high prevalence throughout the world. Nowadays, number of studies about impact of this metabolic disease on patients increasing. The aim of our study is to examine PKU...

Antifungal Activity of Octenidine Dihydrochloride and Ultraviolet-C light against Multidrug-Resistant Candida auris.

Outbreaks due to multidrug-resistant Candida auris have emerged as a large threat to modern medicine. Since skin colonization and environmental contamination have been identified as a precursor for ou...

Pegvaliase: a novel treatment option for adults with phenylketonuria.

In May 2018, the United States Food and Drug Administration approved pegvaliase-pqpz (Palynziq*), the first enzyme substitution therapy for the treatment of phenylketonuria (PKU). This article provide...

Characteristics of PAH gene variants among 113 phenylketonuria patients from Henan Province.

To explore the characteristics of PAH gene variants among 113 phenylketonuria patients from Henan Province.

Molecular-genetic causes for the high frequency of phenylketonuria in the population from the North Caucasus.

Phenylketonuria is an inherited disease caused by mutations in the phenylalanine hydroxylase gene PAH. Different PAH pathogenic variants occur in different ethnic groups with various frequencies and t...

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 anticholesteremic agent that inhibits sterol biosynthesis in animals.

A piperazine derivative, PHOSPHODIESTERASE 5 INHIBITOR and VASODILATOR AGENT that is used as a UROLOGICAL AGENT in the treatment of ERECTILE DYSFUNCTION.

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.

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