Clinical Trials on PKU

Total 53 results

Nutricia Research

Completed

Feeding Patterns and Practices in Young Children With PKU During the First Two Years of Life (PKU-weaning)

PKU

United Kingdom
University of Guelph
McMaster University; Laval University

Not yet recruiting

Phe for Me? The Effects of L-Phe on PKU Carriers and Non-carriers (Phe for Me)

Autosomal Recessive Disorder (Genetic Carriers of PKU)

Canada
University of Guelph
McMaster University

Recruiting

PKU Carriers Trial (Pilot Study): Impact on Cognition, Mental Health, Blood Pressure and Metabolism

Autosomal Recessive Disorder (Genetic Carriers of PKU)

Canada
University of California, Berkeley
Procter and Gamble

Unknown

Safe Water Techniques in Kenya

Consumer Willingness to Pay for POU Products | Consumer Preferences and Use of POU Products

Kenya
Taipei City Hospital

Completed

The Relationship of Platelet Micro-RNA Expression and Platelet Reactivity in Patients Under Clopidogrel or Ticagrelor Treatment

miRNA | PRU

Taiwan
Taipei City Hospital

Completed

Platelet Reactivity (High On-Treatment Platelet Reactivity) as Guidance for APT (Antiplatelet Therapy) Adjustment After PCI (Percutaneous Coronary Intervention)

PRU(Platelet Reactivity Unit) | APT(Antiplatelet Therapy) | HOTPR(High on Treat Platelet Reactivity)

Taiwan
University of Patras

Completed

High on Treatment Platelet Reactivity Following Peripheral Endovascular Procedures (PRECLOP)

Influence of PRU Values on Primary Outcome

Greece
Nutricia UK Ltd

Completed

GMP Case Studies of Tolerance, Safety and Acceptability in PKU and TYR

PKU | Tyrosinemias

United Kingdom
AstraZeneca
IQVIA RDS (Shanghai) Co., Ltd.

Completed

Evaluate the Pharmacokinetics, Safety, and Tolerability of Nirsevimab in Healthy Chinese Adults (PK/ADA)

Evaluate PK Profile

China
BioMarin Pharmaceutical

Recruiting

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

Phenylketonuria (PKU)

United States
Taipei Veterans General Hospital, Taiwan
BioMarin Pharmaceutical

Unknown

BH4 Responsiveness in PAH Deficiency PKU Patients

PAH Deficiency | Pku Phenylketonuria
University of Southern California
BioMarin Pharmaceutical

Completed

Effects of Kuvan on Melatonin Secretion

Phenylketonuria (PKU)

United States
Stanford University
BioMarin Pharmaceutical

Withdrawn

The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU

Classical Phenylketonuria(PKU)
Vitaflo International, Ltd
Birmingham Women's and Children's NHS Foundation Trust

Recruiting

Evaluation of PKU UP (PKU UP)

PKU

United Kingdom
Vitaflo International, Ltd

Completed

Evaluation of PKU Sphere in Italy

Phenylketonurias | PKU

Italy
Homology Medicines, Inc

Terminated

Natural History Clinical Study in Adult PKU

Phenylketonurias | PKU

United States
Children's National Research Institute
Georgetown University

Withdrawn

Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria (PKU)

PKU

United States
Oregon Health and Science University

Terminated

Moderate Intensity Exercise and Phenylketonuria

Phenylketonuria | PKU

United States
BioMarin Pharmaceutical

Recruiting

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) (PALace)

Phenylketonuria (PKU)

United States, Germany, Italy
Nutricia Research

Completed

PANDA: PKU Amino Acid Evaluation (PANDA)

Phenylketonuria (PKU)

United Kingdom, Belgium
BioMarin Pharmaceutical

Active, not recruiting

AAV Gene Therapy Study for Subjects With PKU

Phenylketonuria (PKU)

United States, United Kingdom
BioMarin Pharmaceutical

Completed

A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults (165-901)

Phenylketonuria (PKU)

United States, Turkey, Canada, France, Germany, Italy, United Kingdom
BioMarin Pharmaceutical

Active, not recruiting

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria (PEGASUS)

Phenylketonuria (PKU)

United States, Germany
BioMarin Pharmaceutical

Completed

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

Phenylketonuria (PKU)

United States
BioMarin Pharmaceutical

Completed

Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU (Prism-2)

Phenylketonuria (PKU)

United States
University of British Columbia
Rare Disease Foundation, Vancouver, Canada

Completed

Simple Breath Test to Examine Phenylalanine Metabolism

Phenylketonuria (PKU)

Canada
University of Glasgow

Unknown

Body Composition, Energy Intake and Expenditure in People With Phenylketonuria

Phenylketonuria (PKU)

United Kingdom
University Hospital, Tours
Institut National de la Santé Et de la Recherche Médicale, France

Completed

Antioxidant Signature in Adult Patients With Phenylketonuria (StressOX-PCU)

PKU | Hyperphenylalaninemia

France
Dr. Linda Randolph
BioMarin Pharmaceutical

Terminated

A Pilot Study on Diurnal Variation

Phenylketonuria (PKU)

United States
University of Glasgow

Completed

Impact of Phenylketonuria-type Diet on Appetite, Appetite Hormones and Diet Induced Thermogenesis

Phenylketonuria (PKU)

United Kingdom
Jiangsu vcare pharmaceutical technology co., LTD

Completed

Pharmacokinetics and Pharmacodynamics of Vicagrel in Healthy Adult Subjects of Different CYP2C19

Healthy Subjects | PK/PD

China
University of Zurich

Completed

Phenylketonuria and Hyperphenylalaninemia Nutrition Study

Phenylketonuria (PKU) and Hyperphenylalaninemia

Switzerland
Dr. Schär AG / SPA

Completed

Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein Substitute

Phenylketonurias | Inborn Errors of Metabolism | Metabolic Disease | PKU

United Kingdom
Dr. Schär AG / SPA

Completed

Market Research - Acceptability Study for a New PKU Protein Substitute

Phenylketonurias | Inborn Errors of Metabolism | Metabolic Disease | PKU

United Kingdom
Hadassah Medical Organization

Recruiting

Adjustment of Aminoglycoside Dosing Based on Peak Serum Concentration and Bacterial Minimal Inhibitory Concentration

Aminoglycoside Dosing Based on PK/PD Characteristics

Israel
BioMarin Pharmaceutical

Completed

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency (KAMPER)

Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency

Sweden, France, Italy, Spain, Germany, Netherlands, Austria, Slovakia, Portugal
Astellas Pharma Europe B.V.
FibroGen

Completed

A Study to Evaluate the Dose-proportionality and Effects of FG-4592 in Healthy Young and Elderly Male and Female Subjects

Healthy Subjects | PK for FG-4592

Germany
MingSight Pharmaceuticals Pty Limited

Completed

A Multiple-Ascending Dose Study of MS-553 in Healthy Volunteers

Safety and PK in Healthy Volunteers

Australia
University Hospital, Tours
Institut National de la Santé Et de la Recherche Médicale, France

Completed

Study of a National Cohort of Adult Patients With Phenylketonuria (ECOPHEN)

PKU | Hyperphenylalaninemia

France
Drugs for Neglected Diseases
Sanofi

Completed

Fexinidazole (1200mg) Bioavailability Under Different Food Intake Conditions

PK in Healthy Volunteers

France
Neurocrine Biosciences

Completed

Study to Assess the Effect of Ketoconazole on the Pharmacokinetics of NBI-98854 in Healthy Subjects

Effect of Ketoconazole on the PK of NBI-98854 in Healthy Subjects

United States
Beijing Hospital

Completed

A Study Evaluating the Bioavailability of Oral Insulin (N11005)

PK, PD, and Safety

China
Vitaflo International, Ltd

Recruiting

Evaluation of the Express Plus Range (express plus)

Phenylketonurias | PKU | Maple Syrup Urine Disease | Homocystinuria | Glutaric Acidemia I | Hereditary Tyrosinemia

United Kingdom
Astellas Pharma Europe B.V.
FibroGen

Completed

A Study to Investigate the Exposure and Safety and Tolerability of a Single Dose of FG-4592 in Subjects With Moderately Diminished Liver Function Compared to Those With Normal Liver Function

Hepatic Insufficiency | Healthy Subjects | PK of FG-4592

Bulgaria
Egalet Ltd

Completed

Study to Assess the PK of Ketorolac Tromethamine Intranasal and to Assess the Effects of a Oxymetazoline Hydrochloride on the PK of Ketorolac Tromethamine

Assess Pharmacokinetics of Ketorolac Tromethamine Intranasal and Assess Effects of a Oxymetazoline Hydrochloride on the PK of Ketorolac Tromethamine

United Kingdom
Stanford University
University of Colorado, Denver

Completed

Pharmacokinetics of Ketamine in Infants and Children

The Pk of IV Ketamine in Children With Heart Disease

United States
The Seventh Affiliated Hospital of Sun Yat-sen...
Huizhou Municipal Central Hospital; Dongguan Hospital of Traditional Chinese... and other collaborators

Recruiting

Development and Validation of a Postoperative Re-fracture Risk Model for Osteoporotic Spinal Fractures

Osteoporosis Fracture | Compression Fracture | Osteoporosis Vertebral | Perform PVP / PKP Treatment

China
Neurocrine Biosciences

Completed

Safety, Tolerability, and Pharmacokinetics of a Single Dose of NBI-98854 in Subjects With Mild,Moderate, or Severe Hepatic Insufficiency

Safety, Tolerability, and PK of NBI-98854 in Hepatically Impaired Subjects

United States
Allecra

Completed

Pharmacokinetics of Cefepime and AAI101 in Subjects With Renal Insufficiency and Healthy Subjects

PK in Patients With Various Degrees of Renal Impairment

Czechia, Hungary
Pfizer's Upjohn has merged with Mylan to form Viatris...

Terminated

An Open Label Pharmacokinetic Study Of Gabapentin In Japanese Subjects With Renal Impairment Including Hemodialysis

PK Properties Of Gabapentin In Subjects With Impaired Renal Function

Japan

Clinical Trials on PKU

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Gambia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations