Clinical Trials Logo
  1. Home
  2. Phenylketonurias
  3. NCT04452513
  4. Details
NCT04452513 Clinical Trial

A Prospective Clinical Study of Phenylketonuria (PKU)

Phenylketonurias Clinical Trial

Official title:
A Prospective Clinical Study of Phenylketonuria (PKU)

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT04452513
Other study ID # 307-902
Secondary ID
Status Completed
Phase
First received
Last updated
Start date October 11, 2019
Est. completion date June 2, 2022

Study information
Verified date September 2022
Source BioMarin Pharmaceutical
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

This is a study for adults and children ≥ 14 years old who have Phenylketonuria (PKU) with uncontrolled plasma Phe levels. No clinical intervention or study drug is provided by BioMarin in this study.

Recruitment information / eligibility
Status Completed
Enrollment 32
Est. completion date June 2, 2022
Est. primary completion date June 2, 2022
Accepts healthy volunteers No
Gender All
Age group 14 Years and older
Eligibility Inclusion Criteria: - Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency - Ability and willingness to maintain dietary protein intake consistent with baseline intake - Willingness and capable per investigator opinion to comply with study procedures and requirements - Plasma Phe Levels > 600umol/L Exclusion Criteria: - Clinically significant liver dysfunction or disease - Prior treatment with gene therapy - Any condition that, in the opinion of the investigator or Sponsor, would prevent the subject from fully complying with the requirements of the study

Study Design

Related Conditions & MeSH terms

Locations
Country Name City State
United States Emory University Hospital Atlanta Georgia
United States Children's Hosptial of Colorado Aurora Colorado
United States Boston Children's Hospital Boston Massachusetts
United States Ann & Robert H. Lurie Chilren's Hospital Chicago Illinois
United States University of Florida Gainesville Florida
United States University of Texas Health Science Center at Houston Houston Texas
United States Riley Hospital for Children Indianapolis Indiana
United States University of Kentucky Lexington Kentucky
United States Morristown Medical Center Morristown New Jersey
United States UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania
United States Oregon Health & Science University Portland Oregon
United States Clinic for Special Children Strasburg Pennsylvania
United States University of South Florida Tampa Florida
United States Medical College of Wisconsin Wauwatosa Wisconsin

Sponsors (1)
Lead Sponsor Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome
Type Measure Description Time frame Safety issue
Primary Change in Plasma Phe Levels through study completion, an average of 2 years
Secondary Change in dietary protein intake from medical and intact food through study completion, an average of 2 years
See also
  Status Clinical Trial Phase
Recruiting NCT05998109 - PheCheck Feasibility Study
Not yet recruiting NCT04433728 - Life With Phenylketonuria. Adult Neurological Outcome of PCU Screened Patients From 1971 to 2002.
Completed NCT01209819 - Bone Mineral Density in Adults With Hyperphenylalaninemia N/A
Active, not recruiting NCT05174559 - Additional Dietary Large Neutral Amino Acids (LNAA) for Improved Symptoms in Adult Classical Phenylktonuria (PKU) N/A
Not yet recruiting NCT04969809 - Comparison of Atherogenic Risk Factors and Efficacy of Nutritional Treatment Among Adult Phenylketonuria Patients N/A
Recruiting NCT04404530 - Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
Recruiting NCT05827536 - Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU. N/A
Active, not recruiting NCT05971563 - Amino Acid Kinetics of GMP-AA in Healthy Human Volunteers N/A
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Completed NCT04368624 - PKU Skin Stripping
Completed NCT05497050 - The Effect of Nursing Empowerment Program N/A
Completed NCT04943393 - Remote Neurocognitive and Psychological Assessment in PKU
Completed NCT05096988 - Evaluation of PKU Sphere Liquid N/A
Completed NCT00225615 - A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels Phase 3
Recruiting NCT05128149 - Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference?
Terminated NCT05229549 - Study to Evaluate the Effect on Nitrogen Retention of Two Different Posology Schemes of PKU GOLIKE PLUS 3-16 and Free AAs in Patients With Phenylketonuria N/A
Enrolling by invitation NCT05356377 - Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
Not yet recruiting NCT06332807 - AAV Gene Therapy Clinical Study in Adult Classic PKU Phase 1/Phase 2
Not yet recruiting NCT06337864 - Evaluating the Efficacy and Safety of Large Neutral Amino Acids in the Treatment of Classical Phenylketonuria N/A
Completed NCT02445521 - Testing of Four Home Phenylalanine Monitoring Prototype Devices N/A