PhenylketonuriaNCT01904708
Moderate Intensity Exercise and Phenylketonuria
A study testing Acute Moderate Intensity Exercise in people with Phenylketonuria and PKU, run by Oregon Health and Science University at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 14 to 17 years, men only
- Conditions the study is for
- Phenylketonuria, PKU
- What is being tested?
- Acute Moderate Intensity Exercise
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- May 2013to Jun 2014
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 17 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Oregon Health and Science University
Portland, Oregon, 97239
About this study
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.
Read the detailed description
The indicator amino acid oxidation technique utilizes a carbon labeled isotope (L-[13C]) tracer that is ingested orally and is measured in expired breath. This method is based on the theory that if one essential amino acid is deficient, all other amino acids will be oxidized until a break-point is reached and at that time all amino acids will be incorporated into muscle protein synthesis. Using a randomized crossover design the investigators plan to investigate the effects of an acute bout of moderate intensity treadmill exercise versus sedentary activities on plasma amino acids in four subjects with PKU. The long-term goal of this novel research is to determine if exercise could be used as an adjunctive therapy to improve the management of plasma phenylalanine levels and promote a normal, healthy quality of life among patients with PKU.
Patient Recruitment: Four post-pubertal participants ranging from 14-17 years of age with classical phenylketonuria (PKU), treated at Oregon Health \& Science University, will participate in this pilot study. Participants will be recruited during their routine clinic visit at Doernbecher Children's Hospital Metabolic clinic.
Study Design: This randomized crossover clinical trial will compare the effects of moderate intensity exercise and sedentary activities on amino acid oxidation and plasma amino acid pools using the indicator amino acid oxidation technique. Each participant will be studied on two separate occasions over a one-month period.
- Conditions
- Phenylketonuria, PKU
- Being tested
- Acute Moderate Intensity Exercise
- Keywords
- Phenylketonuria, PKU, Exercise, Muscle Protein Synthesis, Plasma Phenylalanine concentration
- Medical subject headings
- Phenylketonurias, Motor Activity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Acute Moderate Intensity ExerciseOther
Moderate intensity exercise by walking on a treadmill at 75% of max heart rate.
| Study group | Role | What they receive |
|---|---|---|
| Sedentary Visit | No intervention | Subjects will do quiet, sedentary activities for 8 hours. Hourly blood draws and breath sampling will be collected. Bolus C13-Lysine will be given at hour 4. |
| Exercise Visit | Active comparator | Subjects will do quiet, sedentary activities until hour 5. Blood and breath samples will be collected. At hour 4, subject will consume a bolus of C13-Lysine and immediately walk on a treadmill at a moderate intensity (exercise at 75% of max heart rate) for 45 minutes. Receives: Other: Acute Moderate Intensity Exercise |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: up to 8 hours
Secondary outcomes (1)
Breath Enrichment of C13-Lysine during sedentary and exercise
Compare the AUC total breath enrichment of C13-Lysine during the sedentary and exercise arms.
Measured: Hours 1-8 of the study day from each arm.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2013 (estimated)
- Study startMay 2013
- Primary completionJun 2014
- Study completionJun 2014
Why it stopped: Current design not able to measure the outcomes anticipated.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oregon Health and Science University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Melanie B Gillingham, Oregon Health and Science University
- Investigators
- Melanie Gillingham, PhD, RD, Principal investigator, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01904708
- Sponsor's protocol ID
- 9202
- Status last verified
- Oct 2014
- First submitted
- 31 May 2013
- Last updated
- 16 Oct 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effects of an Acute Bout of Moderate Intensity Exercise on Plasma Amino Acids in Subjects With Phenylketonuria
- ClinicalTrials.gov ID
- NCT01904708
- Acronym / sponsor ID
- 9202
- Conditions
- Phenylketonuria, PKU
- Keywords
- Phenylketonuria, PKU, Exercise, Muscle Protein Synthesis, Plasma Phenylalanine concentration
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Current design not able to measure the outcomes anticipated.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Oregon Health and Science UniversityUniversity, hospital or other
- Study start
- May 2013
- Primary completion
- Jun 2014 (actual)
- Study completion
- Jun 2014 (actual)
- First posted
- 22 Jul 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The indicator amino acid oxidation technique utilizes a carbon labeled isotope (L-[13C]) tracer that is ingested orally and is measured in expired breath. This method is based on the theory that if one essential amino acid is deficient, all other amino acids will be oxidized until a break-point is reached and at that time all amino acids will be incorporated into muscle protein synthesis. Using a randomized crossover design the investigators plan to investigate the effects of an acute bout of moderate intensity treadmill exercise versus sedentary activities on plasma amino acids in four subjects with PKU. The long-term goal of this novel research is to determine if exercise could be used as an adjunctive therapy to improve the management of plasma phenylalanine levels and promote a normal, healthy quality of life among patients with PKU.
Patient Recruitment: Four post-pubertal participants ranging from 14-17 years of age with classical phenylketonuria (PKU), treated at Oregon Health \& Science University, will participate in this pilot study. Participants will be recruited during their routine clinic visit at Doernbecher Children's Hospital Metabolic clinic.
Study Design: This randomized crossover clinical trial will compare the effects of moderate intensity exercise and sedentary activities on amino acid oxidation and plasma amino acid pools using the indicator amino acid oxidation technique. Each participant will be studied on two separate occasions over a one-month period.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Sedentary Visit | No intervention | –Subjects will do quiet, sedentary activities for 8 hours. Hourly blood draws and breath sampling will be collected. Bolus C13-Lysine will be given at hour 4. |
| Exercise Visit | Active comparator | Other: Acute Moderate Intensity ExerciseSubjects will do quiet, sedentary activities until hour 5. Blood and breath samples will be collected. At hour 4, subject will consume a bolus of C13-Lysine and immediately walk on a treadmill at a moderate intensity (exercise at 75% of max heart rate) for 45 minutes. |
Who may qualify
- Ages
- 14 to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Acute Moderate Intensity ExerciseModerate intensity exercise by walking on a treadmill at 75% of max heart rate. | Other | Exercise Visit |
Primary outcome
Compare plasma phenylalanine concentration of final blood draw during exercise versus sedentary visits.
Time frame: up to 8 hours
Compare the final blood sample for phenylalanine concentration from each arm of the study.
1 secondary outcome
Breath Enrichment of C13-Lysine during sedentary and exercise
Time frame: Hours 1-8 of the study day from each arm.
Results and safety
The study was stopped early (reason given: Current design not able to measure the outcomes anticipated.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2014.
Related trials for Phenylketonuria
RecruitingPhase 1
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
Phenylketonuria
RecruitingPhase 2
A Study of MZE782 in Adults With PKU
Phenylketonuria (PKU)
RecruitingPhase 3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Phenylketonuria
Recruiting
Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers
Carrier of Phenylketonuria, Healthy
RecruitingPhase 3
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
Phenylketonuria
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Melanie Gillingham, PhD, RD principal investigator
Oregon Health and Science University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Acute Moderate Intensity Exercise in people with Phenylketonuria and PKU, run by Oregon Health and Science University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 16 Oct 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Current design not able to measure the outcomes anticipated.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Compare plasma phenylalanine concentration of final blood draw during exercise versus sedentary visits. (time frame: up to 8 hours). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2014.