Effect of Nutritional Formula Supplementation on Growth Rate of Growth Hormone (GH) Treated Children With Growth Hormone Deficiency (GHD) After the Second Year of Therapy

Effect of Nutritional Formula Supplementation on Growth Rate of Growth Hormone (GH) Treated Children With Growth Hormone Deficiency (GHD) After the Second Year of 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 Isolated Growth Hormone Deficiency (IGHD) after the 2nd year of GH treatment.

In this trial GH is a background treatment (non-investigational medicinal product. Participants may be treated with any of the commercial GH products approved in Israel for the indication of IGHD.

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 and tanner stage.

Participants in the intervention groups will be treated with the study formulas and participants in the control group will be treated with a placebo low caloric formulas (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: Growth Retardation; IGHD - Isolated Growth Hormone Deficiency
• 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, MD; Phone Number: 972-3-9253282; Email: mosheph@clalit.org.il
• Study Contact Backup: Name: Alona Hamou, Msc; Phone Number: 972-3-9253747; Email: alonah@clalit.org.il

Study Locations

• Israel
  • Petach-Tikva, Israel, 49202
    • Recruiting
    • Schnider Children's medical center
    • Contact: Moshe Phillip, Professor; Phone Number: 972-3-9253778; Email: mosheph@clalit.org.il
    • Sub-Investigator: Liat De Vries, Dr.
    • Sub-Investigator: Liora Lazar, Dr.
    • Sub-Investigator: Shlomit Shalitin, Dr.
    • Sub-Investigator: Judit Nir, Dr
    • Sub-Investigator: Sari Karpel, Dr
    • Sub-Investigator: Marie Muller, Dr
    • Sub-Investigator: Naama Fish, Dr
    • Sub-Investigator: Sharon Demol, Dr
    • Principal Investigator: Moshe Phillip, Prof
    • Sub-Investigator: Revital Nimri, Dr.
    • Sub-Investigator: Tal Oron, Dr.
    • Sub-Investigator: Ariel Tenennbaum, Dr.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 14 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• GH treatment for at least 24 months due to IGHD. Confirmed diagnosis of GHD as determined by at least one GH stimulation test, defined as a peak GH level of ≤ 10 ng/ml
• Boys: testicular volume < 8ml; Girls: breast ≤ Tanner 3
• weight SDS < 0
• BMI SDS < 50 precentile for age and gender
• Signing inform consent forms

Exclusion Criteria:

• Other indication for GH treatment.
• Bone age: boys>13.5, girls>11.5.
• Any known morbidity: chronic disease dysmorphic syndromes, bone diseases, organic brain diseases, neurological disease, past or current malignancy, chronic cardiac, renal or pulmonary problems, metabolic disorders.
• Any known gastrointestinal problem including absorption problems.
• Any chronic treatment with medication that might affect appetite, weight or growth (for example steroids) except from Ritalin use
• Any eating disorders and/or psychiatric disorder

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 mineral	Dietary supplement: Placebo; Low caloric formula (Powder added to water) without added vitamins and mineral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Weight Standard Deviation Score (SDS)	at 6 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Height-SDS	at 12 months
Weight SDS	at 12 months
BMI SDS	at 12 months

Other Outcome Measures

Outcome Measure	Time Frame
Body composition	at 12 months

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2017
• Primary Completion (Estimated): October 1, 2024
• Study Completion (Estimated): October 1, 2024

Study Registration Dates

• First Submitted: February 7, 2017
• First Submitted That Met QC Criteria: February 12, 2017
• First Posted (Actual): February 15, 2017

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: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Musculoskeletal Diseases; Hypothalamic Diseases; Bone Diseases; Bone Diseases, Endocrine; Pituitary Diseases; Dwarfism; Bone Diseases, Developmental; Hypopituitarism; Dwarfism, Pituitary; Endocrine System Diseases
• Other Study ID Numbers: rmc082116ctil

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