Growth Hormone Deficiency Clinical Trial
Official title:
Consequence of Lifetime Isolated Growth Hormone Deficiency
Growth hormone (GH) deficiency (GHD) in adulthood has been associated with changes in body
composition (e.g. increased abdominal obesity, and reduced muscle mass), in organ functions
(e.g. reduced cardiac systolic function), in metabolic parameters linked to increased risk
of cardiovascular disease (e.g. increased serum total and LDL cholesterol, C reactive
protein, and plasma fibrinogen), and with reduced bone density. These observations have been
used to define the "adult GHD syndrome" and to advocate GH replacement therapy in GHD
adults. However, most of the studies have been performed in patients who have had
hypothalamic or pituitary diseases, and/or have undergone brain irradiation. Such patients
are often chronically sick, and commonly lack other pituitary hormones, whose replacement
therapies may not fully restore the physiological functions of the under-active glands.
Reliable data on the existence of the AGHD syndrome and its response to GH therapy can be
only obtained by studying patients that are otherwise healthy. However, isolated GH
deficiency (IGHD) is a rare disease. In addition, up to 50% of patients who have been
diagnosed with IGHD in childhood are no longer GH deficient as adults, making such study
difficult to perform due to the scarcity of patients population. We have identified a very
large homogeneous population of patients who have IGHD due to a homozygous mutation in the
GHRH-receptor (GHRHR) gene that resides in a rural area of Brazil. None of the adult dwarf
patients has ever been treated with hGH replacement. This population represents a unique
model to study the effect of isolated lifetime lack of GH. We propose studies of
physiological and metabolic parameters in subjects who are homozygous for this mutation and
compare them with normal subjects residing in the same community.
The primary goal of this proposal is to determine the consequences of life-long lack of GH
on body composition, muscle strength, cardiovascular status, cardiovascular risk factors,
thyroid status and bone density and metabolism, and to test which of these parameters are
reversed by a 6-month course of GH replacement therapy. In addition, we want to test the
hypothesis that heterozygosity for this GHRHR mutation causes a phenotype that may be
intermediate between the one present in homozygous normal subjects and in homozygous
affected GHD patients. This is relevant because inactivating mutations in the GHRHR are
being described with increasing frequency in populations of different genetic background,
suggesting that individuals with faulty single GHRHR alleles may be present in significant
numbers in the general population.
Status | Completed |
Enrollment | 400 |
Est. completion date | December 2009 |
Est. primary completion date | December 2009 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 18 Years to 80 Years |
Eligibility |
Inclusion Criteria: - Lifetime isolated and untreated growth hormone deficiency Exclusion Criteria: - Age below 18 years, pregnancy, diabetes |
Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
Brazil | Federal University of Sergipe | Aracaju | Sergipe |
Lead Sponsor | Collaborator |
---|---|
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Genentech, Inc. |
Brazil,
Alcântara MR, Salvatori R, Alcântara PR, Nóbrega LM, Campos VS, Oliveira EC, Oliveira MH, Souza AH, Aguiar-Oliveira MH. Thyroid morphology and function in adults with untreated isolated growth hormone deficiency. J Clin Endocrinol Metab. 2006 Mar;91(3):86 — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Body composition | after 6 months of GH and after wash out (6 and 12 months) | No | |
Primary | Lipid levels | after 6 months of GH and after 6 and 12 months of wash out | No | |
Primary | Heart function | after 6 months of GH and after 6 and 12 months of wash out | No |
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