Phenylketonuria Clinical Trial
Official title:
The Effects of an Acute Bout of Moderate Intensity Exercise on Plasma Amino Acids in Subjects With Phenylketonuria
Phenylketonuria (PKU), an inherited genetic disorder, can cause irreversible brain damage,
declined executive function, and autistic tendencies unless a phenylalanine (Phe) restricted
diet is consistently maintained throughout life. Promoting anabolism, the uptake of free
amino acids from the extracellular space, is a key component to maintaining plasma
phenylalanine concentrations within treatment range among patients with PKU. Exercise
promotes muscle protein synthesis and anabolism, but the effect on blood phenylalanine
concentrations in patients with PKU has not been reported.
Our objective is to assess the impact of an acute bout of moderate intensity exercise on
protein oxidation and plasma amino acid concentrations, as a potential adjunctive therapy
for patients with PKU.
The investigators hypothesize that moderate intensity exercise decreases amino acid
oxidation, increases muscle protein synthesis, and promotes tissue uptake of essential amino
acids, thereby lowering plasma phenylalanine concentrations in patients with
Phenylketonuria.
Status | Terminated |
Enrollment | 2 |
Est. completion date | June 2014 |
Est. primary completion date | June 2014 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 14 Years to 17 Years |
Eligibility |
Inclusion Criteria: - plasma phenylalanine concentration of >1200 micromols at diagnosis - treated by dietary phenylalanine restriction from early infancy - willing to participate in this study - stable BMI of 20-25 for the past 3 months - currently in average to good physical fitness condition - physically active 3-4 days per week Exclusion Criteria: - previously or are currently taking approved or experimental pharmacologic treatments (i.e.: Biopterin (BH4), sapropterin dihydrochloride (Kuvan), Large Neutral Amino Acids (LNAA), pegylated phenylalanine ammonia lyase (PEG-PAL)) - recent history of weight loss - endocrine disorder - pregnant - anemic - involved in any other study or research protocol - participation in a current strength training or performance training regimen |
Allocation: Randomized, Endpoint Classification: Pharmacokinetics/Dynamics Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Basic Science
Country | Name | City | State |
---|---|---|---|
United States | Oregon Health and Science University | Portland | Oregon |
Lead Sponsor | Collaborator |
---|---|
Oregon Health and Science University |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Compare plasma phenylalanine concentration of final blood draw during exercise versus sedentary visits. | Compare the final blood sample for phenylalanine concentration from each arm of the study. | up to 8 hours | No |
Secondary | Breath Enrichment of C13-Lysine during sedentary and exercise | Compare the AUC total breath enrichment of C13-Lysine during the sedentary and exercise arms. | Hours 1-8 of the study day from each arm. | No |
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 | |
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 | |
Completed |
NCT01560286 -
A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
|
Phase 2 |