The Effect of Nutritional Formula Supplementation on Linear Growth of Growth Hormone (GH) Treated Prepubertal Children With Idiopathic Short Stature (ISS) After 2 Years From the Beginning of GH-therapy

The Effect of Nutritional Formula Supplementation on Linear Growth of GH Treated Prepubertal Children With Idiopathic Short Stature (ISS) After 2 Years From the Beginning of GH-therapy: a Randomized, Double- Blind, Placebo-controlled Trial

The proposed study is a double blind, randomized, placebo controlled study. The aim of the study is to evaluate the effect of combined growth hormone (GH) treatment &nutritional formula supplementation versus GH & placebo on growth parameters in 64 children with Idiopathic Short Stature (ISS) after the second year of GH treatment.

Participants will be randomly assigned either to the intervention group or the placebo control group. Randomization for the two study groups will be made in a ratio of 1:1. Both participants and study team will be blinded to the type of treatment that each patient will receive during the study. The randomization will be done according to gender.

Participants in the intervention groups will be treated with the study formula and participants in the control group will be treated with a placebo low caloric formula (powder added to water). The study will continue for 6 months of intervention versus active placebo, with additional 6 months (an extension period), in which participants at both groups, the intervention and the placebo, will be offered to continue their participation in the study with the active study supplement.

Study Overview

• Status: Recruiting
• Conditions: ISS- Idiopathic Short Stature
• Intervention / Treatment: Dietary supplement: Nutritional supplementation standardized formula; Dietary supplement: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 64
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Moshe Phillip, Prof.; Phone Number: 972-3-9253282; Email: mosheph@clalit.org.il
• Study Contact Backup: Name: Alonah Hamou, MSc; Phone Number: 972-39253747; Email: alonah@clalit.org.il

Study Locations

• Israel
  • Petah tikva, Israel
    • Recruiting
    • Schneider Children's Medical Center
    • Contact: Moshe Phillip, Prof; Phone Number: 972-3-9253778; Email: mosheph@clalit.org.il
    • Sub-Investigator: Naama Fish, Dr
    • Sub-Investigator: Liora Lazar, Prof
    • Sub-Investigator: Sharon Demol, Dr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 10 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. GH treatment for at least 24 months due to idiopathic short stature (ISS)
2. Age 5-10 years inclusive.
3. Tanner stage 1 (gonadarche).
4. BMI <85 percentile for age and gender.

Exclusion Criteria:

1. SGA/ IUGR
2. Diagnosis of GH deficiency
3. Any known morbidity: chronic disease dysmorphic syndromes, bone diseases, organic brain diseases, neurological disease, past or current malignancy, chronic cardial, renal or pulmonary problems, metabolic disorders.
4. Any known gastrointestinal problem including absorption problems.
5. Any chronic treatment with medication that might affect appetite, weight or growth (for example SSRI's, steroids).
6. Any eating disorders and/or psychiatric disorder
7. Milk or other food allergies

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
Experimental: Nutritional standardized supplementation formula.; Powder added to water, containing about 25% of recommended daily recommended intake (DRI) for calories, high protein (25% of calories) and multivitamin and mineral (25%-100% of DRI for recommended daily allowance (RDA) or adequate intake.	Dietary supplement: Nutritional supplementation standardized formula; Powder added to water, containing about 25% of recommended Daily Recommended Intake (DRI) for calories, high protein (25% of calories) and multivitamin and mineral (25%-100% of DRI for recommended daily allowance (RDA) or adequate intake
Placebo Comparator: Placebo; Low caloric formula (Powder added to water) without added vitamins and minerals	Dietary supplement: Placebo; Low caloric formula (Powder added to water) without added vitamins and minerals

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
weight standard deviation score	at 6 months
height standard deviation score (SDS)	at 6 moths

Secondary Outcome Measures

Outcome Measure	Time Frame
BMI SDS	at 6 months
Lean body mass	at 6 months
muscle mass	at 6 months
Fat mass	at 6 months
Fat mass percentage	at 6 months

Other Outcome Measures

Outcome Measure	Time Frame
Weight SDS	at 12 months
BMI SDS	at 12 months
height SDS	at 12 months
Lean body mass	at 12 months
muscle mass	at 12 months
fat mass	at 12 months
fat mass percentage	at 12 months

Collaborators and Investigators

• Sponsor: Rabin Medical Center
• Investigators: Principal Investigator: Moshe Phillip, Prof, Schnieder Children's Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): August 1, 2021
• Primary Completion (Estimated): July 1, 2025
• Study Completion (Estimated): October 1, 2025

Study Registration Dates

• First Submitted: July 4, 2021
• First Submitted That Met QC Criteria: July 13, 2021
• First Posted (Actual): July 15, 2021

Study Record Updates

• Last Update Posted (Actual): March 26, 2024
• Last Update Submitted That Met QC Criteria: March 25, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Endocrine System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Bone Diseases; Bone Diseases, Developmental; Dwarfism
• Other Study ID Numbers: rmc26021ctil

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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