Very Long-chain Acyl-CoA Dehydrogenase DeficiencyPhase 1/2NCT01494051
High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders
A Phase 1/2 study testing Diet counseling in people with Very Long-chain Acyl-CoA Dehydrogenase Deficiency and Trifunctional Protein Deficiency, run by Oregon Health and Science University.
Trial summary
The study also determines if eating a diet higher in protein alters body composition, energy balance and metabolic control among patients with a long-chain fatty acid oxidation disorder. Read more about this study
- Who is it for?
- Ages 7 to 45 years
- Conditions the study is for
- Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency, Carnitine Palmitoyltransferase 2 Deficiency
- What is being tested?
- Diet counseling
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- No sites listed yet
- Study dates
- Jan 2006to Jan 2010
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 7 to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- diagnosis of a long-chain fatty acid oxidation disorder including VLCAD, CPT2, LCHAD or TFP deficiency
- 7 years old or greater
- able to comply with diet guidelines
Who cannot join
- pregnant
- enrolled in another study that alters diet composition
- cannot complete treadmill exercise study
About this study
Official title: Fatty Acid Oxidation and Body Weight Regulation in Long-chain Fatty Acid Oxidation Disorders.
The study also determines if eating a diet higher in protein alters body composition, energy balance and metabolic control among patients with a long-chain fatty acid oxidation disorder.
Read the detailed description
At baseline, the amount of muscle and fat in the whole body and inside the liver and muscle will be measured. All subjects will complete a moderate treadmill exercise test and a test to determine how they use sugar (oral glucose tolerance test). The amount and type of energy burned by each subject will be measured. Subjects will be randomly assigned to follow either a high carbohydrate diet or a reduced carbohydrate, high protein diet for 4 months. At the end of 4 months, the tests will be repeated.
- Conditions
- Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency, Carnitine Palmitoyltransferase 2 Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
- Being tested
- Diet counseling
- Keywords
- fatty acid oxidation disorders
- Medical subject headings
- VLCAD deficiency, Trifunctional Protein Deficiency With Myopathy And Neuropathy, Carnitine palmitoyl transferase 2 deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Diet counselingBehavioural
Also called low fat diet, high carbohydrate diet, high protein diet
Subjects counseled how to follow either the high carbohydrate diet or the high protein diet for 4 months at home.
| Study group | Role | What they receive |
|---|---|---|
| High carbohydrate diet | Active comparator | Diet composition of 10% long-chain fatty acids, 20% medium-chain triglycerides, 12% protein and 68% carbohydrate is the current standard of care in long-chain fatty acid oxidation disorders. Receives: Behavioral: Diet counseling |
| High protein diet | Experimental | Diet composition of 10% long-chain fatty acids, 20% medium chain triglycerides, 25% protein and 45% carbohydrate is the comparison diet. Fat content is the same between treatments; only the carbohydrate to protein ratio varies. Receives: Behavioral: Diet counseling |
What is being measured
The primary outcome is the main question the study is designed to answer.
Energy Expenditure
does energy expenditure and substrate oxidation differ between subjects randomized to the high carbohydrate versus the high protein diet? We measured resting energy expenditure with indirect calorimetry and estimated substrate oxidation with indirect calorimetry results and urine urea nitrogen excretion. We also measured total energy expenditure with doubly labeled water.
Measured: change from baseline after 4 months of treatment
Secondary outcomes (1)
Body Composition
does body composition change more among subjects randomized to the high protein diet compared to the high carbohydrate diet? We measured body composition at baseline and again at the end of the study by DEXA - dual energy x-ray absorptiometry.
Measured: change from baseline to 4 months of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2011 (estimated)
- Study startJan 2006
- Primary completionSep 2009
- Study completionJan 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Very Long-chain Acyl-CoA Dehydrogenase DeficiencyStudies of the same condition, open and under way.
- More trials run by Oregon Health and Science UniversityThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 13 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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01494051
- Sponsor's protocol ID
- DK71869-2
- Status last verified
- Apr 2020
- First submitted
- 14 Dec 2011
- Last updated
- 29 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Fatty Acid Oxidation and Body Weight Regulation in Long-chain Fatty Acid Oxidation Disorders.
- ClinicalTrials.gov ID
- NCT01494051
- Acronym / sponsor ID
- DK71869-2
- Conditions
- Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency, Carnitine Palmitoyltransferase 2 Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
- Keywords
- fatty acid oxidation disorders
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Oregon Health and Science UniversityUniversity, hospital or other
- Study start
- Jan 2006
- Primary completion
- Sep 2009 (actual)
- Study completion
- Jan 2010 (actual)
- First posted
- 16 Dec 2011 (estimated)
- Results
- Posted 21 Apr 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study also determines if eating a diet higher in protein alters body composition, energy balance and metabolic control among patients with a long-chain fatty acid oxidation disorder.
Detailed description
At baseline, the amount of muscle and fat in the whole body and inside the liver and muscle will be measured. All subjects will complete a moderate treadmill exercise test and a test to determine how they use sugar (oral glucose tolerance test). The amount and type of energy burned by each subject will be measured. Subjects will be randomly assigned to follow either a high carbohydrate diet or a reduced carbohydrate, high protein diet for 4 months. At the end of 4 months, the tests will be repeated.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| High carbohydrate diet | Active comparator | Behavioral: Diet counselingDiet composition of 10% long-chain fatty acids, 20% medium-chain triglycerides, 12% protein and 68% carbohydrate is the current standard of care in long-chain fatty acid oxidation disorders. |
| High protein diet | Experimental | Behavioral: Diet counselingDiet composition of 10% long-chain fatty acids, 20% medium chain triglycerides, 25% protein and 45% carbohydrate is the comparison diet. Fat content is the same between treatments; only the carbohydrate to protein ratio varies. |
Who may qualify
- Ages
- 7 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- diagnosis of a long-chain fatty acid oxidation disorder including VLCAD, CPT2, LCHAD or TFP deficiency
- 7 years old or greater
- able to comply with diet guidelines
Key exclusion criteria (3 listed)
- pregnant
- enrolled in another study that alters diet composition
- cannot complete treadmill exercise study
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 |
|---|---|---|
| Diet counselingAlso: low fat diet, high carbohydrate diet, high protein dietSubjects counseled how to follow either the high carbohydrate diet or the high protein diet for 4 months at home. | Behavioural | High carbohydrate diet, High protein diet |
Primary outcome
Energy Expenditure
Time frame: change from baseline after 4 months of treatment
does energy expenditure and substrate oxidation differ between subjects randomized to the high carbohydrate versus the high protein diet? We measured resting energy expenditure with indirect calorimetry and estimated substrate oxidation with indirect calorimetry results and urine urea nitrogen excretion. We also measured total energy expenditure with doubly labeled water.
1 secondary outcome
Body Composition
Time frame: change from baseline to 4 months of treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Very Long-chain Acyl-CoA Dehydrogenase Deficiency
Enrolling by invitation
Investigation of the Treatment of Patients With Mitochondrial Defects by Assisted Reproductive Technology
Pearson Syndrome, Mitochondria DNA Deletion
RecruitingPhase 2
Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS)
Mitochondrial Diseases, Pearson Syndrome
Active, not recruitingPhase 3
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
RecruitingObservational study
Global Registry and Natural History Study for Mitochondrial Disorders
Mitochondrial Diseases, Kearns-Sayre Syndrome
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Diet counseling in people with Very Long-chain Acyl-CoA Dehydrogenase Deficiency and Trifunctional Protein Deficiency, run by Oregon Health and Science University.
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 3 inclusion and 3 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: finished, as of the sponsor's last update on 29 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Apr 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Energy Expenditure (time frame: change from baseline after 4 months of treatment). 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 29 Apr 2020.