Free Fatty Acids, Body Weight, and Growth Hormones Secretion in Children

NCT ID: NCT01237041

Last Updated: 2018-12-17

Study Results

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

37 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-07-01

Study Completion Date

2017-12-13

Brief Summary

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Background:

\- Overweight and obese children and adults often have lower levels of growth hormone in the blood. Regulation of growth hormone may be tied to weight and free fatty acids in the blood. Current tests of growth hormone (such as those used when evaluating the heights of children who are markedly shorter than other children of comparable age) may be affected by other factors, including obesity. Researchers are interested in evaluating the levels of growth hormone and free fatty acids in the blood of children between 7 and 14 years of age who weigh more than children of a comparable age, or who are shorter than other children of a comparable age and have been recommended for growth hormone testing as part of an evaluation for their height.

Objectives:

\- To determine the effect of changes in free fatty acids in the blood on changes in growth hormone secretion in overweight or shorter children and young adolescents.

Eligibility:

\- Children and adolescents between 7 and 14 years of age who weigh more than or are shorter than other children of a comparable age and do not have any medical illnesses.

Design:

* Participants will have two study visits, one of which will be a half day screening visit in the outpatient clinic and one of which will require 2 nights as an inpatient at the National Institutes of Health Clinical Center.
* Participants should not eat or drink anything except water after 10 PM the night before or on the morning of the screening visit.
* At the screening visit, participants will have a physical examination and medical history, provide blood and urine samples, have an oral glucose tolerance test (to check blood sugar levels), and have an x-ray of the left hand to check bone age.
* The inpatient study visit will involve a physical examination and medical history, a full x-ray scan to study body fat and muscle, frequent blood tests throughout the visit, and various medications to stimulate growth hormone production and lower levels of free fatty acids in the blood.

Detailed Description

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Obese children and adults display lower spontaneous and stimulated growth hormone (GH) secretion. It is presumed that dysregulation of some of the factors normally involved in controlling GH secretion underlies the hyposomatotropinemia of obesity, given that GH production usually normalizes after weight loss. Free fatty acids (FFA) are one factor thought to be involved in regulation of GH secretion. Niacin is a nicotinic acid derivative that inhibits lipolysis and lowers circulating FFA concentrations. Nicotinic acid derivatives have been used in several adult studies examining GH secretion. Specifically in obese adults, inhibition of lipolysis has been found to increase spontaneous and stimulated GH production, presumably due to direct effects of FFA on hypothalamic GH-regulating neurons. Thus far no pediatric studies have examined the effects of niacin on GH secretion, and there is only one small pediatric study of normal weight prepubertal children growing at the 5th-10th percentile in height has tested the effects of lipolytic inhibition by acipimox (a related medication also derived from nicotinic acid) on GH secretion. There are no data in obese children demonstrating the effects of inhibition of lipolysis on GH secretion.

We propose to investigate one of the mechanisms through which high adiposity alters GH secretion in children by testing the effects of inhibiting lipolysis. First we will conduct dose establishing studies to determine the appropriate dose of niacin needed to suppress FFA concentrations in children. We will then conduct the main study, designed as a pilot randomized, double-blind placebo controlled trial of niacin administration, to assess its effects on stimulated GH secretion. We hypothesize that in overweight children niacin will lead to a fall in free fatty acid concentrations and consequently a rise in stimulated GH secretion. We further hypothesize that the overweight subjects will demonstrate stimulated GH secretion profiles with niacin similar to those of control subjects who receive placebo. We expect this pilot study may help improve how diagnostic testing is carried out for growth hormone deficiency in children.

Conditions

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Obesity Short Stature Growth Hormone Deficiency

Keywords

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Child Body Fat Niacin Growth Hormone Obesity Overweight Growth Hormones Short Stature Children

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

First, we conducted dose-establishing studies using 250mg every 2 hours x 3 doses (2 subjects), 500mg every two hours x 3 doses (5 subjects), and 500mg hourly x 4 doses (5 subjects). This dose-establishing study was followed by the randomized crossover trial using 500mg hourly x 4 doses in conjunction with growth hormone stimulation testing. The RCT was terminated early because of inability to obtain necessary study medications.
Primary Study Purpose

DIAGNOSTIC

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Masking only during the two arms of the randomized trial portion of the study, not during the dose-finding arms.

Study Groups

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Niacin First

Subjects receive niacin 500mg hourly for 4 hours on day 1 (at 7:30am, 8:30am, 9:30am, and 10:30am) then cross over to receive placebo hourly for 4 hours on day 2 at (7:30am, 8:30am, 9:30am, and 10:30am).

Group Type EXPERIMENTAL

Niacin First

Intervention Type DRUG

Niacin 500 mg po four times on one of the inpatient days, then placebo on another inpatient day

Placebo First

Intervention Type DRUG

Placebo po four times on one of the inpatient days, then Niacin 500 mg po four times on another inpatient day

Placebo First

Subjects receive placebo hourly for 4 hours on day 1 (at 7:30am, 8:30am, 9:30am, and 10:30am) then cross over to receive niacin hourly for 4 hours on day 2 (at 7:30am, 8:30am, 9:30am, and 10:30am).

Group Type EXPERIMENTAL

Niacin First

Intervention Type DRUG

Niacin 500 mg po four times on one of the inpatient days, then placebo on another inpatient day

Placebo First

Intervention Type DRUG

Placebo po four times on one of the inpatient days, then Niacin 500 mg po four times on another inpatient day

Dose-Establishing Study 1 Niacin 250mg

Subjects received Niacin 250 mg every 2 hours for 3 doses (at 6am, 8am, and 10am).

Group Type EXPERIMENTAL

Dose-Establishing Study 1 Niacin 250mg

Intervention Type DRUG

Niacin 250 mg po three times at 2 hour intervals on an inpatient day

Dose-Establishing Study 1 Niacin 500mg

Subjects received Niacin 500 mg every 2 hours for 3 doses (at 6am, 8am, and 10am).

Group Type EXPERIMENTAL

Dose-Establishing Study 1 Niacin 500mg

Intervention Type DRUG

Niacin 500 mg po three times at 2 hour intervals on an inpatient day

Dose-Establishing Study 2 Niacin 500mg

Subjects received Niacin 500 mg hourly for 4 doses (administered at 7:30am, 8:30am, 9:30am, and 10:30am).

Group Type EXPERIMENTAL

Dose-Establishing Study 2 Niacin 500mg

Intervention Type DRUG

Open-Label Niacin 500mg four times at 1 hour intervals on an inpatient day

Interventions

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Niacin First

Niacin 500 mg po four times on one of the inpatient days, then placebo on another inpatient day

Intervention Type DRUG

Placebo First

Placebo po four times on one of the inpatient days, then Niacin 500 mg po four times on another inpatient day

Intervention Type DRUG

Dose-Establishing Study 1 Niacin 250mg

Niacin 250 mg po three times at 2 hour intervals on an inpatient day

Intervention Type DRUG

Dose-Establishing Study 1 Niacin 500mg

Niacin 500 mg po three times at 2 hour intervals on an inpatient day

Intervention Type DRUG

Dose-Establishing Study 2 Niacin 500mg

Open-Label Niacin 500mg four times at 1 hour intervals on an inpatient day

Intervention Type DRUG

Other Intervention Names

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RCT Niacin First RCT Placebo First Open-Label Niacin 250mg x 3 doses Open-Label Niacin 500mg x 3 doses Open-Label Niacin 500mg x four doses

Eligibility Criteria

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Inclusion Criteria

Subjects will qualify for the overweight group for the dose-establishing studies 1 and 2 and main study) if they meet the following criteria:

1. Good general health.
2. Age greater than or equal to 7 and less than 15 years.
3. Tanner stage I, II or III for the breast among girls and testes less than10 mL for boys based upon an examination by a trained physician or nurse practitioner.
4. Weight \> 30 kg.
5. Fasting plasma glucose \< 100 mg/dL, 2 hour post-dextrose glucose \< 140 mg/dL, and HgbA1C less than or equal to 6.4%.
6. Females who are age 10 or greater must have a negative pregnancy test.
7. Body mass index greater than or eqaul to 95th percentile determined by Centers for Disease Control age and sex specific data (given that most pathology of obesity does not usually emerge until children cross the 95th percentile).
8. No evidence of growth failure as defined as height \> 5th percentile.

Subjects will qualify for the non-overweight control group (for the main study only) if they meet the following criteria:

1. Recommended by a pediatric endocrinologist to undergo GH stimulation testing to establish the diagnosis of GH-deficiency.
2. Good general health.
3. Age greater than or equal to 7 and less than15 years.
4. Tanner stage I, II or III for the breast among girls and testes less than 10 mL for boys based upon an examination by a trained physician or nurse practitioner.
5. Weight \> 30 kg.
6. Fasting plasma glucose \< 100 mg/dL, 2 hour post-dextrose glucose \< 140 mg/dL, and HgbA1C less than or equal to 6.4%.
7. Females who are age 10 or greater must have a negative pregnancy test.
8. Height \< 5th percentile.
9. BMI between the 5th and 85th percentiles determined by Centers for Disease Control age and sex specific data.
10. Birth weight and length not consistent with small for gestational age (SGA) criteria or a history of intrauterine growth restriction (IUGR) based on recall history.

Exclusion Criteria

Subjects will be excluded if they have any of the following:

1. Baseline creatinine greater than or equal to 1.0 mg/dl.
2. Significant cardiac or pulmonary disease likely to or resulting in hypoxia or decreased perfusion.
3. Hepatic disease with elevated liver function tests (ALT or AST)greater than or equal to 1.5 the upper limits of normal.
4. Pregnancy.
5. Evidence for impaired glucose tolerance or Type 2 diabetes, including fasting plasma glucose greater than or equal to 100 mg/dL, 2 hour post-dextrose glucose greater than or equal to 140 mg/dL, or HgbA1C \> 6.4%.
6. Presence of other endocrinologic disorders leading to obesity (e.g. Cushing Syndrome).
7. Any disorder that is known to affect GH secretion (e.g. untreated hypothyroidism) or use of any medication known to affect GH levels (including glucocorticoids and GH itself).
8. Any other disorder that is known to affect stature including skeletal dysplasias.
9. Recent use (within two years) of anorexiant medications, stimulant medications, or other medications felt to impact growth.
10. Individuals who have, or whose parent or guardians have, current substance abuse or a psychiatric disorder or other condition that, in the opinion of the investigators, would impede competence or compliance or possibly hinder completion of the study.
11. Individuals receiving medical treatment other than diet for hypertension or dyslipidemia.
12. Individuals with evidence of precocious puberty as defined as palpable breast tissue noted in females before the age of 7, testicular size greater than or equal to 4cc in males before the age of 9, or bone age advancement more than 2 SD for chronologic age.
13. Individuals receiving androgen or estrogen hormone therapy.
Minimum Eligible Age

7 Years

Maximum Eligible Age

14 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NIH

Sponsor Role lead

Responsible Party

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Jack Yanovski, M.D.

Chief, Section on Growth and Obesity

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Jack A Yanovski, M.D.

Role: PRINCIPAL_INVESTIGATOR

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Locations

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National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, United States

Site Status

Countries

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United States

References

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Tauber M, Moulin P, Pienkowski C, Jouret B, Rochiccioli P. Growth hormone (GH) retesting and auxological data in 131 GH-deficient patients after completion of treatment. J Clin Endocrinol Metab. 1997 Feb;82(2):352-6. doi: 10.1210/jcem.82.2.3726.

Reference Type BACKGROUND
PMID: 9024217 (View on PubMed)

Cohen P, Rogol AD, Deal CL, Saenger P, Reiter EO, Ross JL, Chernausek SD, Savage MO, Wit JM; 2007 ISS Consensus Workshop participants. Consensus statement on the diagnosis and treatment of children with idiopathic short stature: a summary of the Growth Hormone Research Society, the Lawson Wilkins Pediatric Endocrine Society, and the European Society for Paediatric Endocrinology Workshop. J Clin Endocrinol Metab. 2008 Nov;93(11):4210-7. doi: 10.1210/jc.2008-0509. Epub 2008 Sep 9.

Reference Type BACKGROUND
PMID: 18782877 (View on PubMed)

Zucchini S, Pirazzoli P, Baronio F, Gennari M, Bal MO, Balsamo A, Gualandi S, Cicognani A. Effect on adult height of pubertal growth hormone retesting and withdrawal of therapy in patients with previously diagnosed growth hormone deficiency. J Clin Endocrinol Metab. 2006 Nov;91(11):4271-6. doi: 10.1210/jc.2006-0383. Epub 2006 Aug 15.

Reference Type BACKGROUND
PMID: 16912138 (View on PubMed)

Galescu OA, Crocker MK, Altschul AM, Marwitz SE, Brady SM, Yanovski JA. A pilot study of the effects of niacin administration on free fatty acid and growth hormone concentrations in children with obesity. Pediatr Obes. 2018 Jan;13(1):30-37. doi: 10.1111/ijpo.12184. Epub 2016 Sep 21.

Reference Type BACKGROUND
PMID: 27654882 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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11-CH-0004

Identifier Type: OTHER

Identifier Source: secondary_id

110004

Identifier Type: -

Identifier Source: org_study_id