Nutritional Stimulation of Growth in Children With Short Stature

Short stature is a frequent reason for referral to a pediatric endocrinology clinic. Short stature is especially prevalent among those with failure to thrive (whose weight is significantly below the average weight of his/her peers). The growth hormone has limited efficacy for medical treatment of short stature when the cause of short stature is not growth hormone deficiency. This study will investigate the effect of 6 months of nutritional supplement (essential amino acids) compared to placebo in the linear growth of short children who have not yet reached puberty.

Study Overview

• Status: Active, not recruiting
• Conditions: Growth Failure; Nutritional Stunting
• Intervention / Treatment: Dietary supplement: Essential Amino Acid (EAA) group; Dietary supplement: Placebo

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Emir Tas

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 9 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Ages 3 to 11 years (inclusive).
• Pre-pubertal status (i.e. Tanner stage 1 for breast development for girls and testicular enlargement for boys and pubic hair for both sexes).

Exclusion criteria:

• Ages younger than 3 years or older than 11 years.
• Medical history of a neurologic, endocrinologic, genetic, or metabolic problem known to have a direct effect on height growth. This includes, but is not limited to, children with a Growth hormone deficiency, Down syndrome, Turner syndrome, Russel-Silver syndrome, Prader-Willi syndrome, Pseudohypoparathyroidism, chronic kidney disease, malabsorptive syndromes, cancer survivors, etc.
• Currently being treated or previously treated with Growth hormone, or history of oral steroid treatment within the last 3 months.
• Being in puberty.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Treatment Group; This is a double-blind study. Participants and the investigators will be blinded to the intervention.; Children in the treatment (intervention) arm will receive essential amino acids (EAA) twice a day.; Children 3 to 5 years of age will be asked to take either 8.5 g (1 serving) of EAA supplement or placebo at breakfast and at bedtime.; Children 6 to 11 years of age will be assigned to take either 17 g (2 servings) of EAA supplement or placebo at breakfast and at bedtime.; EAA and placebo dissolve easily in any liquid beverage and can be taken on empty versus full stomach.	Dietary supplement: Essential Amino Acid (EAA) group; Eligible children will be supplemented with Essential Amino Acids (EAA) for 6 months twice a day. Caution will be exercised to match the groups for age and sex.
Placebo Comparator: Placebo; This is a double-blind study. Participants and the investigators will be blinded to the intervention.; Children in the placebo arm will receive placebo twice a day. EAA and placebo supplements will look and taste alike. The same flavoring ingredients (stevia blend, citric acid, malic acid, natural flavors, tartaric acid, fruit and vegetable juice for color) will be used in both products at the same amount.; Children 3 to 5 years of age will be asked to take either 8.5 g (1 serving) of EAA supplement or placebo at breakfast and at bedtime.; Children 6 to 11 years of age will be assigned to take either 17 g (2 servings) of EAA supplement or placebo at breakfast and at bedtime.; EAA and placebo dissolve easily in any liquid beverage and can be taken on empty versus full stomach.	Dietary supplement: Placebo; Eligible children will be supplemented with a placebo for 6 months twice a day. Caution will be exercised to match the groups for age and sex.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Linear Growth	Growth velocity (cm/year) will be calculated and compared between arms using the height measurements taken before and after 6 months of Essential Amino Acids vs Placebo supplementation.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Body Composition	Lean body mass will be measured using Dual X-Ray Absorptimetry (DXA) scan and compared between arms using the measurements taken before and after 6 months of Essential Amino Acids vs Placebo supplementation.	6 months
Body Composition	Body fat mass will be measured using Dual X-Ray Absorptimetry (DXA) scan and compared between arms using the measurements taken before and after 6 months of Essential Amino Acids vs Placebo supplementation.	6 months
Body Composition	Visceral fat mass will be measured using Dual X-Ray Absorptimetry (DXA) scan and compared between arms using the measurements taken before and after 6 months of Essential Amino Acids vs Placebo supplementation.	6 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Serum growth factor concentrations	Serum concentration of Insulin-Like Growth-Factor (IGF)-I will be measured and compared between arms before and after 6 months of Essential Amino Acids vs Placebo supplementation.	6 months
Serum growth factor concentrations	Serum concentration of Insulin-Like Growth-Factor Binding-Protein (IGF BP)-III will be measured and compared between arms before and after 6 months of Essential Amino Acids vs Placebo supplementation.	6 months
Serum amino acid concentrations	Serum amino acid concentrations will be measured and compared between arms before and after 6 months of Essential Amino Acids vs Placebo supplementation.	6 months

Collaborators and Investigators

• Sponsor: Arkansas Children's Hospital Research Institute
• Investigators: Principal Investigator: Emir Tas, MD, Arkansas Children's Research Insitute

Study record dates

Study Major Dates

• Study Start (Actual): September 9, 2020
• Primary Completion (Actual): March 15, 2022
• Study Completion (Anticipated): December 31, 2022

Study Registration Dates

• First Submitted: January 7, 2020
• First Submitted That Met QC Criteria: January 9, 2020
• First Posted (Actual): January 13, 2020

Study Record Updates

• Last Update Posted (Actual): June 2, 2022
• Last Update Submitted That Met QC Criteria: June 1, 2022
• Last Verified: June 1, 2022

More Information

Terms related to this study

• Keywords: short stature; essential amino acids
• Additional Relevant MeSH Terms: Endocrine System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Bone Diseases; Bone Diseases, Developmental; Failure to Thrive; Dwarfism
• Other Study ID Numbers: 239581

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No