Cystic FibrosisNCT01172301
Leucine-enriched Essential Amino Acid Intake to Optimize Protein Anabolism in Children With Cystic Fibrosis
A study testing Essential amino acid intake + Leucine vs total AA supplement in people with Cystic Fibrosis, run by Texas A&M University at 1 site in United States.
Trial summary
Malnutrition, including muscle wasting commonly occurs in children with cystic fibrosis (CF), negatively influencing their quality of life and survival. At the time of a diagnosis of CF, severe protein deficits can already be present. Read more about this study
- Who is it for?
- Ages 10 to 21 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Essential amino acid intake + Leucine vs total AA supplement
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2008to Feb 2013
- 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 10 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects who already have a diagnosis of CF based on universal diagnostic criteria.
- Age 14 to 21 years at the time of enrollment
- Under routine medical control at the CF center of ACH
- Admitted to the ACH for treatment of pulmonary exacerbation of CF disease.
- Improvement in lung function (FEV1) at the time of enrollment back to baseline values (as determined in the clinically stable pre-hospital period)
- Central or peripheral venous line in place
- No planned major changes or interventions in the treatment and care of the pediatric subject on Day -2 and -1 before discharge from the hospital.
Who cannot join
- Established diagnosis of Diabetes Mellitus
- Presence of fever within the last 3 days
- Unstable metabolic diseases including liver (cirrhosis) or renal disease
- Chronic respiratory failure with cor pulmonale
- Use of long-term oral corticosteroids or short course of oral corticosteroids in the preceding month before enrollment
- Any other condition according to the principle investigator or study physician would interfere with collecting study samples
- Failure to give assent / informed consent
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205
About this study
Malnutrition, including muscle wasting commonly occurs in children with cystic fibrosis (CF), negatively influencing their quality of life and survival. At the time of a diagnosis of CF, severe protein deficits can already be present. It is important to get CF children fed adequately to prevent that their condition becomes worse or that recovery takes longer. Oral supplementation trials showed that gains in lean body mass are difficult to achieve in CF unless specific metabolic abnormalities are targeted. However, the specific needs for certain food components are not clear yet in children that are ill. Therefore, more information is necessary on the need for protein and certain amino acids in children with CF. Previous studies support the concept of essential amino acids (EAA) as an anabolic stimulus in the young and elderly and in insulin resistant states. Until yet no information is present on the anabolic effects of EAA in CF.
It is therefore our hypothesis that a high-leucine essential amino acids mixture specifically designed to stimulate protein anabolism will target the metabolic alterations of pediatric subjects with CF. In the present proposal, the acute metabolic effects of this high leucine essential amino acids mixture will be examined in pediatric subjects with CF and compared to that of a regular balanced total mixture of essential and non-essential amino acids. The principal endpoints will be the extent of stimulation of whole body protein synthesis as this is the principal mechanism by which either amino acid or protein intake causes muscle anabolism, and the reduction in endogenous protein breakdown. Both endpoints will be assessed by isotope methodology which is thought to be the reference method.
Read the detailed description
In this study, we will test the following hypothesis: A high-leucine essential amino acid mixture (dose of 6.7 g) will stimulate protein anabolism to a greater extent than a standard balanced mixture of total (essential and non-essential) amino acids in CF pediatric subjects. The principal endpoints will be the extent of stimulation of protein synthesis rate and the reduction in endogenous protein breakdown. The current project will provide information that will enable us to better understand the underlying metabolic mechanisms that regulate protein metabolism in pediatric subjects with CF.
- Conditions
- Cystic Fibrosis
- Being tested
- Essential amino acid intake + Leucine vs total AA supplement
- Keywords
- CF, protein metabolism, essential amino acid intake
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Essential amino acid intake + Leucine vs total AA supplementDietary supplement
Also called 7 g EAA + 40% LEU
7 g as bolus
| Study group | Role | What they receive |
|---|---|---|
| Oral EAA vs total AA supplement | Experimental | Not described Receives: Dietary Supplement: Essential amino acid intake + Leucine vs total AA supplement |
What is being measured
The primary outcome is the main question the study is designed to answer.
Net whole body protein synthesis rate
Acute change from postabsorptive state after intake of essential amino acid + LEU vs total amino acid supplement
Measured: Up to 2 years
Secondary outcomes (9)
Whole body collagen breakdown rate
Acute change from postabsorptive state after intake of essential amino acid + LEU vs total amino acid supplement
Measured: Up to 2 years
Urea turnover rate
Acute change from postabsorptive state after intake of essential amino acid + LEU vs total amino acid supplement
Measured: Up to 2 years
Arginine turnover rate
Measured in postabsorptive state
Measured: Up to 2 years
Liver protein synthesis rate
Acute change from postabsorptive state after intake of essential amino acid + LEU vs total amino acid supplement
Measured: Up to 2 years
Resting Energy expenditure
Measured in postabsorptive state
Measured: Up to 2 years
Insulin kinetics
Acute change from postabsorptive state after intake of essential amino acid + LEU vs total amino acid supplement
Measured: Up to 2 years
Amino acid kinetics
Acute change from postabsorptive state after intake of essential amino acid + LEU vs total amino acid supplement
Measured: Up to 2 years
Glucose kinetics
Acute change from postabsorptive state after intake of essential amino acid + LEU vs total amino acid supplement
Measured: Up to 2 years
Fat-free mass
Characterization of subjects
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2010 (estimated)
- Study startJul 2008
- Primary completionDec 2012
- Study completionFeb 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies 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
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Texas A&M University
- Sponsor type
- University, hospital or other
- Collaborators
- Arkansas Children's Hospital Research Institute
- Responsible party
- Principal investigator, Marielle PKJ Engelen, PhD, Texas A&M University
- Investigators
- Nicolaas EP Deutz, MD, PhD, Principal investigator, University of Arkansas
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01172301
- Sponsor's protocol ID
- 104738
- Status last verified
- Aug 2015
- First submitted
- 27 Jul 2010
- Last updated
- 11 Aug 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Engelen MP, Com G, Deutz NE. Increased whole body hydroxyproline production as assessed by a new stable isotope technique is associated with hip and spine bone mineral loss in cystic fibrosis. Clin Nutr. 2014 Dec;33(6):1117-21. doi: 10.1016/j.clnu.2013.12.008. Epub 2013 Dec 29.
PubMed 24423745
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Leucine-enriched Essential Amino Acid Intake to Optimize Protein Anabolism in Children With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT01172301
- Acronym / sponsor ID
- 104738
- Conditions
- Cystic Fibrosis
- Keywords
- CF, protein metabolism, essential amino acid intake
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Texas A&M UniversityUniversity, hospital or other
- Collaborators
- Arkansas Children's Hospital Research Institute
- Study start
- Jul 2008
- Primary completion
- Dec 2012 (actual)
- Study completion
- Feb 2013 (actual)
- First posted
- 29 Jul 2010 (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.
Malnutrition, including muscle wasting commonly occurs in children with cystic fibrosis (CF), negatively influencing their quality of life and survival. At the time of a diagnosis of CF, severe protein deficits can already be present. It is important to get CF children fed adequately to prevent that their condition becomes worse or that recovery takes longer. Oral supplementation trials showed that gains in lean body mass are difficult to achieve in CF unless specific metabolic abnormalities are targeted. However, the specific needs for certain food components are not clear yet in children that are ill. Therefore, more information is necessary on the need for protein and certain amino acids in children with CF. Previous studies support the concept of essential amino acids (EAA) as an anabolic stimulus in the young and elderly and in insulin resistant states. Until yet no information is present on the anabolic effects of EAA in CF.
It is therefore our hypothesis that a high-leucine essential amino acids mixture specifically designed to stimulate protein anabolism will target the metabolic alterations of pediatric subjects with CF. In the present proposal, the acute metabolic effects of this high leucine essential amino acids mixture will be examined in pediatric subjects with CF and compared to that of a regular balanced total mixture of essential and non-essential amino acids. The principal endpoints will be the extent of stimulation of whole body protein synthesis as this is the principal mechanism by which either amino acid or protein intake causes muscle anabolism, and the reduction in endogenous protein breakdown. Both endpoints will be assessed by isotope methodology which is thought to be the reference method.
Detailed description
In this study, we will test the following hypothesis: A high-leucine essential amino acid mixture (dose of 6.7 g) will stimulate protein anabolism to a greater extent than a standard balanced mixture of total (essential and non-essential) amino acids in CF pediatric subjects. The principal endpoints will be the extent of stimulation of protein synthesis rate and the reduction in endogenous protein breakdown. The current project will provide information that will enable us to better understand the underlying metabolic mechanisms that regulate protein metabolism in pediatric subjects with CF.
Study design
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind (participant, investigator)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Oral EAA vs total AA supplement | Experimental | Dietary Supplement: Essential amino acid intake + Leucine vs total AA supplement |
Who may qualify
- Ages
- 10 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Subjects who already have a diagnosis of CF based on universal diagnostic criteria.
- Age 14 to 21 years at the time of enrollment
- Under routine medical control at the CF center of ACH
- Admitted to the ACH for treatment of pulmonary exacerbation of CF disease.
- Improvement in lung function (FEV1) at the time of enrollment back to baseline values (as determined in the clinically stable pre-hospital period)
Key exclusion criteria (7 listed)
- Established diagnosis of Diabetes Mellitus
- Presence of fever within the last 3 days
- Unstable metabolic diseases including liver (cirrhosis) or renal disease
- Chronic respiratory failure with cor pulmonale
- Use of long-term oral corticosteroids or short course of oral corticosteroids in the preceding month before enrollment
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 |
|---|---|---|
| Essential amino acid intake + Leucine vs total AA supplementAlso: 7 g EAA + 40% LEU7 g as bolus | Dietary supplement | Oral EAA vs total AA supplement |
Primary outcome
Net whole body protein synthesis rate
Time frame: Up to 2 years
Acute change from postabsorptive state after intake of essential amino acid + LEU vs total amino acid supplement
9 secondary outcomes
Whole body collagen breakdown rate
Time frame: Up to 2 years
Urea turnover rate
Time frame: Up to 2 years
Arginine turnover rate
Time frame: Up to 2 years
Liver protein synthesis rate
Time frame: Up to 2 years
Resting Energy expenditure
Time frame: Up to 2 years
Insulin kinetics
Time frame: Up to 2 years
Amino acid kinetics
Time frame: Up to 2 years
Glucose kinetics
Time frame: Up to 2 years
Fat-free mass
Time frame: Up to 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2015.
- Derived from this study: PubMed 24423745Engelen MP, Com G, Deutz NE. Increased whole body hydroxyproline production as assessed by a new stable isotope technique is associated with hip and spine bone mineral loss in cystic fibrosis. Clin Nutr. 2014 Dec;33(6):1117-21. doi: 10.1016/j.clnu.2013.12.008. Epub 2013 Dec 29.
Related trials for Cystic Fibrosis
RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete
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.
Nicolaas EP Deutz, MD, PhD principal investigator
University of Arkansas
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Essential amino acid intake + Leucine vs total AA supplement in people with Cystic Fibrosis, run by Texas A&M 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 7 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: finished, as of the sponsor's last update on 11 Aug 2015. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Net whole body protein synthesis rate (time frame: Up to 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01172301, which also lists 1 linked publication.
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 11 Aug 2015.