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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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IGHD - Isolated Growth Hormone Deficiency, Growth Retardation

Intervention / Treatment
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* Dietary Supplement: Nutritional supplementation standardized formula
* Dietary Supplement: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 14 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Nutritional Standardized Supplementation Formula<br>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<br>* 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<br>|
| Placebo Comparator: Placebo<br>Low caloric formula (Powder added to water) without added vitamins and mineral | Dietary Supplement: Placebo<br>* Low caloric formula (Powder added to water) without added vitamins and mineral<br>|

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

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