Short StaturePhase 2NCT00102258
Role of Nutrition and Hormones in Boys With Disordered Growth
A Phase 2 study testing Growth Hormone Therapy in people with Short Stature and Constitutional Delay of Growth and Development, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at 1 site in United States.
Trial summary
This study will determine whether adding more calories to the diet helps boys with growth problems grow better while being treated with Nutropin, a growth hormone that is used to help children grow taller. The Food and Drug Administration has approved Nutropin for use in children who are very short. Read more about this study
- Who is it for?
- Ages 7 to 10 years, men only
- Conditions the study is for
- Short Stature, Constitutional Delay of Growth and Development, Dwarfism, Pituitary
- What is being tested?
- Growth Hormone Therapy
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Jan 2005to 2 Oct 2007
- Phase
- Phase 2
- 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 7 to 10 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Age 7-10 years, genital Tanner 1, significant short stature (current height less than 2.25SD below the mean for age), body mass index (BMI) less than 25th percentile for age, weight-for-height less than 25th percentile, bone age less than 10 years and with delay greater than 1 year below chronological age, minimum of 6 months of documented height measurements performed in the pediatric endocrinology clinic using a stadiometer to permit accurate calculation of baseline growth velocity at time of enrollment, otherwise normal physical exam, expressed desire for medical intervention to promote growth, and assessment by a pediatric endocrinologist that the boy qualifies for GH treatment according to current FDA guidelines for the use of GH in children with idiopathic short stature (i.e., children who are determined not to have growth hormone deficiency but have height less than -2.25SD below the mean for age).
Who cannot join
- History of major/chronic illness, anosmia/hyposmia, any clinically significant abnormalities on biochemical testing. GH deficiency would be excluded prior to enrollment based on anthropometric and GH stimulation test data. Endocrinopathies (except stable thyroid replacement), skeletal dysplasias, participation in highly competitive endurance sports activities, diagnosis of ADHD with use of stimulant medication in the previous 3 months will also be exclusion criteria.
Trial locations and contacts
1 site in 1 country.
United States1 site
Nemours Children's Clinic
Jacksonville, Florida, 32207
About this study
Official title: Nutrients and Hormones: Effects in Boys With Disordered Growth - Pilot Study
This study will determine whether adding more calories to the diet helps boys with growth problems grow better while being treated with Nutropin, a growth hormone that is used to help children grow taller. The Food and Drug Administration has approved Nutropin for use in children who are very short. This study will examine whether giving nutritional supplements in addition to Nutropin can help children grow better than with Nutropin alone.
Boys between 7 and 10 years of age who are very short and below average in weight, but are otherwise healthy may be eligible for this study. Candidates must qualify for Nutropin treatments to boost their growth. Boys will be recruited for the study from the Nemours Children's Clinic in Jacksonville, FL, and from the National Institutes of Health in Bethesda, MD.
Read more
Participants are randomly assigned to one of two treatment groups. One group is observed for 6 months and then receives a Nutropin injection every day for 12 months. The second group drinks 8 ounces of a high-calorie beverage called Pediasure every day for 6 months and then receives Nutropin plus Pediasure every day for 12 months. In addition to treatment, participants undergo the following tests and procedures at the schedule indicated: Baseline, 3, 6, 9, 12, 15 and 18 months
- Clinical examination
- Height measurement
- Body composition assessment: Skin-fold thickness calipers are used in four places on the body to estimate body fat
- Bioelectric impedance: A small amount of electrical current is used to calculate the percentage of body fat.
Baseline, 6, 12, and 18 months
- Blood tests
- Bone age x-ray: x-ray of the left hand to measure growth potential
- DEXA (dual energy x-ray absorptiometry) scan: x-ray scan to measure body fat, muscle, and bone mineral content. The subject lies on a flat table during the scan.
Baseline, 6, and 12 months
- Record of dietary intake: Parents are asked to write down everything the child eats and drinks for 3 days. Using this record, a dietitian calculates the daily caloric intake.
- Total energy expenditure: This test determines how much energy the child uses. For the test, the child drinks water labeled with harmless isotopes (heavy oxygen and heavy hydrogen). For the next 10 days he collects urine in plastic tubes at home. At the end of the 10 days, the parents bring the urine to the clinic for analysis to determine how fast the labeled water leaves the body. This information is used to calculate how much energy the child expends each day.
Participants' weight is measured at 2 and 4 weeks, and then monthly for the remainder of the 18-month study.
Read the detailed description
We propose a pilot study to examine the role of nutrition and its interaction with growth hormone (GH) therapy in boys with very short stature who also have delayed bone age characteristic of constitutional delay of growth and maturation (CDGM). Recent studies have suggested that insufficient caloric intake may contribute to the pathophysiology of CDGM. This prompted us to consider whether nutritional intervention may benefit children with severe short stature who also have CDGM.
To investigate this further, we would like to enroll a total of 20 boys (10-15 boys in Jacksonville, Florida and 5-10 boys at the NIH, Bethesda, Maryland), ages 7-10 years, who are otherwise healthy, but have significant short stature (height SDS less than -2.25 = the current FDA approved indication for the use of GH in idiopathic short stature), delayed bone age (greater than 12 months below chronologic age), and low BMI and weight-for-height (less than 25th percentile). Ten boys will be randomized to receive observation for 6 months, followed by GH therapy for 12 months. Ten boys will be randomized to receive daily liquid nutritional supplementation for 6 months, followed by combined GH therapy and nutritional supplementation for 12 months. For all subjects, weight gain will be monitored at 2 weeks, 4 weeks, and then monthly. Height gain and body composition (using skin-fold thickness calipers and bioelectric impedance analysis) will be assessed every 3 months. Total energy expenditure, three-day dietary history, bone age, body composition/bone mineral density (using dual energy X-ray absorptiometry), and laboratory studies (IGF1, IGFBP3, pre-albumin, transferrin, ghrelin, fasting insulin, glucose, and lipid profile) will be obtained at baseline and then every 6 months.
We hypothesize that nutritional supplementation alone can lead to small improvements in linear growth and lean body mass accrual, but when combined with GH therapy, can augment the anabolic actions of GH, thereby resulting in even greater improvements in linear growth velocity and lean body mass accrual than GH alone. If the data from this study confirm this trend, we would then like to proceed with a larger study, appropriately powered based on the results of this pilot.
- Being tested
- Growth Hormone Therapy
- Keywords
- Short Stature, Constitutional Delay of Growth and Development, Body Composition, Energy Expenditure, Dietary Supplements, Constitutional Delay of Growth, CDGM
- Medical subject headings
- Dwarfism, Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Growth Hormone TherapyDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2005 (estimated)
- Study start19 Jan 2005
- Study completion2 Oct 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Short StatureStudies 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 2
- Primary purpose
- Treatment
- Enrolment
- 20 participants
Sponsor and investigators
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00102258
- Sponsor's protocol ID
- 050077
- Other IDs
- 05-CH-0077
- Status last verified
- 2 Oct 2007
- First submitted
- 25 Jan 2005
- Last updated
- 2 Jul 2017
- Results posted
- No
Publications
Leschek EW, Rose SR, Yanovski JA, Troendle JF, Quigley CA, Chipman JJ, Crowe BJ, Ross JL, Cassorla FG, Blum WF, Cutler GB Jr, Baron J; National Institute of Child Health and Human Development-Eli Lilly & Co. Growth Hormone Collaborative Group. Effect of growth hormone treatment on adult height in peripubertal children with idiopathic short stature: a randomized, double-blind, placebo-controlled trial. J Clin Endocrinol Metab. 2004 Jul;89(7):3140-8. doi: 10.1210/jc.2003-031457.
PubMed 15240584Han JC, Damaso L, Welch S, Balagopal P, Hossain J, Mauras N. Effects of growth hormone and nutritional therapy in boys with constitutional growth delay: a randomized controlled trial. J Pediatr. 2011 Mar;158(3):427-32. doi: 10.1016/j.jpeds.2010.09.006. Epub 2010 Oct 18.
PubMed 20961566
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nutrients and Hormones: Effects in Boys With Disordered Growth - Pilot Study
- ClinicalTrials.gov ID
- NCT00102258
- Acronym / sponsor ID
- 050077
- Keywords
- Short Stature, Constitutional Delay of Growth and Development, Body Composition, Energy Expenditure, Dietary Supplements, Constitutional Delay of Growth, CDGM
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)US National Institutes of Health
- Study start
- 19 Jan 2005
- Study completion
- 2 Oct 2007
- First posted
- 26 Jan 2005 (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.
This study will determine whether adding more calories to the diet helps boys with growth problems grow better while being treated with Nutropin, a growth hormone that is used to help children grow taller. The Food and Drug Administration has approved Nutropin for use in children who are very short. This study will examine whether giving nutritional supplements in addition to Nutropin can help children grow better than with Nutropin alone.
Boys between 7 and 10 years of age who are very short and below average in weight, but are otherwise healthy may be eligible for this study. Candidates must qualify for Nutropin treatments to boost their growth. Boys will be recruited for the study from the Nemours Children's Clinic in Jacksonville, FL, and from the National Institutes of Health in Bethesda, MD.
Participants are randomly assigned to one of two treatment groups. One group is observed for 6 months and then receives a Nutropin injection every day for 12 months. The second group drinks 8 ounces of a high-calorie beverage called Pediasure every day for 6 months and then receives Nutropin plus Pediasure every day for 12 months. In addition to treatment, participants undergo the following tests and procedures at the schedule indicated: Baseline, 3, 6, 9, 12, 15 and 18 months
- Clinical examination
- Height measurement
- Body composition assessment: Skin-fold thickness calipers are used in four places on the body to estimate body fat
- Bioelectric impedance: A small amount of electrical current is used to calculate the percentage of body fat.
Baseline, 6, 12, and 18 months
- Blood tests
- Bone age x-ray: x-ray of the left hand to measure growth potential
- DEXA (dual energy x-ray absorptiometry) scan: x-ray scan to measure body fat, muscle, and bone mineral content. The subject lies on a flat table during the scan.
Baseline, 6, and 12 months
- Record of dietary intake: Parents are asked to write down everything the child eats and drinks for 3 days. Using this record, a dietitian calculates the daily caloric intake.
- Total energy expenditure: This test determines how much energy the child uses. For the test, the child drinks water labeled with harmless isotopes (heavy oxygen and heavy hydrogen). For the next 10 days he collects urine in plastic tubes at home. At the end of the 10 days, the parents bring the urine to the clinic for analysis to determine how fast the labeled water leaves the body. This information is used to calculate how much energy the child expends each day.
Participants' weight is measured at 2 and 4 weeks, and then monthly for the remainder of the 18-month study.
Detailed description
We propose a pilot study to examine the role of nutrition and its interaction with growth hormone (GH) therapy in boys with very short stature who also have delayed bone age characteristic of constitutional delay of growth and maturation (CDGM). Recent studies have suggested that insufficient caloric intake may contribute to the pathophysiology of CDGM. This prompted us to consider whether nutritional intervention may benefit children with severe short stature who also have CDGM.
To investigate this further, we would like to enroll a total of 20 boys (10-15 boys in Jacksonville, Florida and 5-10 boys at the NIH, Bethesda, Maryland), ages 7-10 years, who are otherwise healthy, but have significant short stature (height SDS less than -2.25 = the current FDA approved indication for the use of GH in idiopathic short stature), delayed bone age (greater than 12 months below chronologic age), and low BMI and weight-for-height (less than 25th percentile). Ten boys will be randomized to receive observation for 6 months, followed by GH therapy for 12 months. Ten boys will be randomized to receive daily liquid nutritional supplementation for 6 months, followed by combined GH therapy and nutritional supplementation for 12 months. For all subjects, weight gain will be monitored at 2 weeks, 4 weeks, and then monthly. Height gain and body composition (using skin-fold thickness calipers and bioelectric impedance analysis) will be assessed every 3 months. Total energy expenditure, three-day dietary history, bone age, body composition/bone mineral density (using dual energy X-ray absorptiometry), and laboratory studies (IGF1, IGFBP3, pre-albumin, transferrin, ghrelin, fasting insulin, glucose, and lipid profile) will be obtained at baseline and then every 6 months.
We hypothesize that nutritional supplementation alone can lead to small improvements in linear growth and lean body mass accrual, but when combined with GH therapy, can augment the anabolic actions of GH, thereby resulting in even greater improvements in linear growth velocity and lean body mass accrual than GH alone. If the data from this study confirm this trend, we would then like to proceed with a larger study, appropriately powered based on the results of this pilot.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 7 to 10 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Age 7-10 years, genital Tanner 1, significant short stature (current height less than 2.25SD below the mean for age), body mass index (BMI) less than 25th percentile for age, weight-for-height less than 25th percentile, bone age less than 10 years and with delay greater than 1 year below chronological age, minimum of 6 months of documented height measurements performed in the pediatric endocrinology clinic using a stadiometer to permit accurate calculation of baseline growth velocity at time of enrollment, otherwise normal physical exam, expressed desire for medical intervention to promote growth, and assessment by a pediatric endocrinologist that the boy qualifies for GH treatment according to current FDA guidelines for the use of GH in children with idiopathic short stature (i.e., children who are determined not to have growth hormone deficiency but have height less than -2.25SD below the mean for age).
Key exclusion criteria (1 listed)
- History of major/chronic illness, anosmia/hyposmia, any clinically significant abnormalities on biochemical testing. GH deficiency would be excluded prior to enrollment based on anthropometric and GH stimulation test data. Endocrinopathies (except stable thyroid replacement), skeletal dysplasias, participation in highly competitive endurance sports activities, diagnosis of ADHD with use of stimulant medication in the previous 3 months will also be exclusion criteria.
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 |
|---|---|---|
| Growth Hormone Therapy | Drug | – |
Primary outcome
Not reported in the source record.
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 2 Jul 2017.
- Background: PubMed 15240584Leschek EW, Rose SR, Yanovski JA, Troendle JF, Quigley CA, Chipman JJ, Crowe BJ, Ross JL, Cassorla FG, Blum WF, Cutler GB Jr, Baron J; National Institute of Child Health and Human Development-Eli Lilly & Co. Growth Hormone Collaborative Group. Effect of growth hormone treatment on adult height in peripubertal children with idiopathic short stature: a randomized, double-blind, placebo-controlled trial. J Clin Endocrinol Metab. 2004 Jul;89(7):3140-8. doi: 10.1210/jc.2003-031457.
- Derived from this study: PubMed 20961566Han JC, Damaso L, Welch S, Balagopal P, Hossain J, Mauras N. Effects of growth hormone and nutritional therapy in boys with constitutional growth delay: a randomized controlled trial. J Pediatr. 2011 Mar;158(3):427-32. doi: 10.1016/j.jpeds.2010.09.006. Epub 2010 Oct 18.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Growth Hormone Therapy in people with Short Stature and Constitutional Delay of Growth and Development, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 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 1 inclusion and 1 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 2 Jul 2017. 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.
What does this phase mean?
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?
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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00102258, which also lists 2 linked publications.
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 2 Jul 2017.