Phenylketonuria Clinical Trial
Official title:
Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response
The investigators will investigate the effect of blood phenylalanine on Kuvan responsiveness
in the same patients with PKU when their blood phenylalanine concentrations are altered by
diet.
Lowering blood phenylalanine concentrations in Kuvan non-responsive patients with PKU will
increase the frequency of Kuvan responsiveness.
n/a
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT05099640 -
A Study of PTC923 in Participants With Phenylketonuria
|
Phase 3 | |
Completed |
NCT01924026 -
Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
|
N/A | |
Completed |
NCT01428258 -
Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria
|
N/A | |
Completed |
NCT00925054 -
Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
|
Phase 2 | |
Completed |
NCT00778206 -
PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
|
||
Recruiting |
NCT05948020 -
Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria
|
N/A | |
Recruiting |
NCT05781399 -
First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria
|
Phase 1 | |
Completed |
NCT02555579 -
Simplified Diet Approach in Phenylketonuria
|
N/A | |
Completed |
NCT03097250 -
MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria
|
||
Completed |
NCT01965912 -
Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
|
Phase 4 | |
Completed |
NCT01965691 -
Protein Requirements in Children With Phenylketonuria (PKU)
|
N/A | |
Completed |
NCT00688844 -
Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
|
N/A | |
Completed |
NCT00789568 -
A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
|
Phase 1 | |
Terminated |
NCT01465100 -
Liver Cell Transplant for Phenylketonuria
|
Phase 1/Phase 2 | |
Completed |
NCT01732471 -
Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
|
Phase 3 | |
Completed |
NCT04879277 -
Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria
|
N/A | |
Completed |
NCT02176603 -
Observational Study of Endothelial Dysfunction in Phenylketonuria
|
N/A | |
Terminated |
NCT01904708 -
Moderate Intensity Exercise and Phenylketonuria
|
N/A | |
Completed |
NCT01869972 -
Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
|
N/A | |
Completed |
NCT01819727 -
An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
|
Phase 3 |