Ignite Creation Date:
2024-05-06 @ 12:08 AM
Last Modification Date:
2024-10-26 @ 10:45 AM
Study NCT ID:
NCT01504802
Status:
COMPLETED
Last Update Posted:
2014-10-02
First Post:
2011-07-20
Brief Title:
Pharmacodynamics of CNP During Growth Hormone Treatment
Sponsor:
Nemours Childrens Clinic
Organization:
Nemours Childrens Clinic
Study Overview
Official Title:
Pharmacodynamics of C-Type Natriuretic Peptide During Growth Hormone Treatment in Children A Potential Biomarker of Efficacy
Status:
COMPLETED
Status Verified Date:
2014-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
It is now widespread practice to treat children with short stature with growth hormone However how an individual child will respond to growth hormone treatment is unpredictable and highly variable Some children will not respond to growth hormone treatment at all Currently the only way to determine how well growth hormone therapy is working is to wait until they have been treated for six months and to compare the pre-treatment growth velocity with the growth velocity on treatment It would be helpful to have a blood test that could be done shortly after starting growth hormone that could predict whether how well a child is responding to treatment Such a blood test would allow endocrinologists to adjust the growth hormone dose or possibly stop it altogether if it is not working long before the six months it currently takes
C-type natriuretic peptide CNP and its partner amino-terminal propeptide of CNP NTproCNP are proteins that play a critical role in regulating growth The investigators have previously shown that blood levels of these proteins increase in children being treated with growth hormone The investigators believe that a blood test for these proteins will be useful in predicting a childs response to growth hormone treatment
The purpose of this study is to determine when after starting growth hormone the blood levels of CNP and NTproCNP start to increase
C-type natriuretic peptide CNP and its partner amino-terminal propeptide of CNP NTproCNP are proteins that play a critical role in regulating growth The investigators have previously shown that blood levels of these proteins increase in children being treated with growth hormone The investigators believe that a blood test for these proteins will be useful in predicting a childs response to growth hormone treatment
The purpose of this study is to determine when after starting growth hormone the blood levels of CNP and NTproCNP start to increase
Detailed Description:
Treatment of children with short stature with recombinant human growth hormone is widespread practice However the growth response to growth hormone treatment is highly variable particularly for those children who do not have classic growth hormone deficiency The availability of a biomarker of efficacy that can be measured early in treatment would be beneficial
C-type natriuretic peptide CNP plays a critical role in linear growth CNP is produced in the growth plate and signals through a paracrine mechanism Its bioinactive amino-terminal propeptide NTproCNP is easily measurable in plasma and levels reflect rate of CNP biosynthesis Previous studies in lambs and children have shown that the plasma concentration of NTproCNP correlates with linear growth velocity and the investigators have also shown that levels are increased during growth hormone therapy The investigators have proposed that NTproCNP is a biomarker for linear growth and consider it the first growth plate function test Such a growth biomarker is likely to reflect efficacy of growth hormone therapy soon after starting growth hormone possibly as soon as a few days Before the clinical utility of this can be determined the investigators need to ascertain the pharmacodynamics of CNP and NTproCNP in response to growth hormone
The goal of this study is to describe the pharmacodynamics of the CNP response to the initiation of growth hormone in two sets of children with short stature those with growth hormone deficiency and those in whom normal growth hormone secretion idiopathic short stature and to compare these data to the pharmacodynamics of other peptides previously identified as potential biomarkers The investigators hypothesize that plasma NTproCNP levels will increase within four days of starting growth hormone therapy and that the response in children with growth hormone deficiency will be more prompt and greater than those with idiopathic short stature The investigators second hypothesis is that the increase in NTproCNP in response to growth hormone will correlate with the increase in growth velocity after six and twelve months of treatment
The study is a prospective observational study of children with growth hormone deficiency n10 and with idiopathic short stature n10 being started on rhGH therapy The study consists of frequent monitoring of analyte levels over one year of treatment
This is a two site study Nemours Childrens Clinic Jacksonville Florida and Childrens Hospital Los Angeles
C-type natriuretic peptide CNP plays a critical role in linear growth CNP is produced in the growth plate and signals through a paracrine mechanism Its bioinactive amino-terminal propeptide NTproCNP is easily measurable in plasma and levels reflect rate of CNP biosynthesis Previous studies in lambs and children have shown that the plasma concentration of NTproCNP correlates with linear growth velocity and the investigators have also shown that levels are increased during growth hormone therapy The investigators have proposed that NTproCNP is a biomarker for linear growth and consider it the first growth plate function test Such a growth biomarker is likely to reflect efficacy of growth hormone therapy soon after starting growth hormone possibly as soon as a few days Before the clinical utility of this can be determined the investigators need to ascertain the pharmacodynamics of CNP and NTproCNP in response to growth hormone
The goal of this study is to describe the pharmacodynamics of the CNP response to the initiation of growth hormone in two sets of children with short stature those with growth hormone deficiency and those in whom normal growth hormone secretion idiopathic short stature and to compare these data to the pharmacodynamics of other peptides previously identified as potential biomarkers The investigators hypothesize that plasma NTproCNP levels will increase within four days of starting growth hormone therapy and that the response in children with growth hormone deficiency will be more prompt and greater than those with idiopathic short stature The investigators second hypothesis is that the increase in NTproCNP in response to growth hormone will correlate with the increase in growth velocity after six and twelve months of treatment
The study is a prospective observational study of children with growth hormone deficiency n10 and with idiopathic short stature n10 being started on rhGH therapy The study consists of frequent monitoring of analyte levels over one year of treatment
This is a two site study Nemours Childrens Clinic Jacksonville Florida and Childrens Hospital Los Angeles
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None