Phenylketonuria Clinical Trial
Official title:
A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects With Phenylketonuria
This double-blind, placebo-controlled, randomized study is designed to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU.
Phenylketonuria (PKU) results from deficient phenylalanine hydroxylase (PAH) activity and
leads to toxic phenylalanine (Phe) accumulation in patients with PKU causing mental
retardation, microcephaly, delayed speech, seizures, psychiatric symptoms and behavioral
abnormalities. Although for most PKU patients early initiation of dietary treatment prevents
severe complications, discontinuation of dietary restrictions at an early age is associated
with poor cognitive development and neuropsychiatric disorders are present even in
early-treated and well controlled PKU patients.
This study, PKU-016, will be conducted in PKU patients to evaluate the therapeutic effects
of sapropterin dihydrochloride on the symptoms of attention deficit hyperactivity disorder
(ADHD), depression, and anxiety.
;
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
| 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 |
NCT00789568 -
A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
|
Phase 1 | |
| Completed |
NCT00688844 -
Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
|
N/A | |
| 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 |
NCT01819727 -
An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
|
Phase 3 | |
| Completed |
NCT01869972 -
Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
|
N/A |