Pharmacodynamics of CNP During Growth Hormone Treatment

NCT01504802 | Completed

• 1 other identifier: NCC 167624
• Study Type: observational
• Target: 22
• Locations: 1 country
• Sites: 2

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 child's 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.

Conditions

Pituitary Dwarfism; Idiopathic Short Stature

Keywords

Growth hormone; C-type natriuretic peptide; amino-terminal propeptide of C-type natriuretic peptide; insulin-like growth factor-I; bone specific alkaline phosphatase; leptin; deoxypyridinoline; growth velocity; pharmacodynamic; biomarker

Outcome Measures

Primary Outcomes (1)

• Determine the time after starting rhGH that NTproCNP level reaches 95% of its peak level

  NTproCNP will be modeled over time for each individual subject and the time it reaches 95% of its peak value determined. This value will then be averaged for the cohort.

  One year

Secondary Outcomes (2)

• Correlate NTproCNP levels at the time it reaches 95% of its peak with six-month and one year growth velocity on rhGH treatment

  one year

• Compare NTproCNP levels with other biomarkers of growth (serum IGF-I, bone-specific alkaline phosphatase, and leptin, and urine deoxypyridinoline) during rhGH treatment

  One year

Study Arms (2)

Growth hormone deficient

Children with short stature, a peak growth hormone response on stimulation testing of less than 7 ng/ml, and no other identifiable cause of short stature

Idiopathic short stature

Children with short stature, a peak growth hormone response on stimulation testing of greater than or equal to 7 ng/ml, and no identifiable cause for the short stature

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Children with short stature

You may qualify if:

• Age greater than 3 years
• Prepubertal
• Height SD score less than -2.25
• Had a growth hormone stimulation test

You may not qualify if:

• History of any other disease or drug treatment that might interfere with linear growth, including amphetamine derivatives for treatment of ADD or ADHD
• Previous treatment with any growth-promoting medication, including growth hormone
• Any contraindication to growth hormone therapy
• Minor acute illness (upper respiratory infections, strep throat, gastroenteritis, urinary tract infection, etc.) less than one month prior to starting growth hormone
• Major acute illness (pneumonia, meningitis, pyelonephritis, any illness requiring hospitalization, etc.), any surgery, or bone fracture less than six months prior to starting growth hormone
• Weight less than 13 kg (NCC-J) or 15 kg (CHLA), due to blood volume being drawn.

Sponsors & Collaborators

• Nemours Children's Clinic: lead
• Children's Hospital Los Angeles: collaborator
• Novo Nordisk A/S: collaborator

Study Sites (2)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Nemours Children Clinic

Jacksonville, Florida, 32207, United States

Location

Related Links

• Nemours Children's Clinic
• Childrens Hospital Los Angeles

Biospecimen

Retention: SAMPLES WITHOUT DNA

Serum, plasma, and urine will be saved for potential future studies.

MeSH Terms

Conditions

Dwarfism, Pituitary

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases

Study Officials

• Robert Olney, MD

  Nemours Children's Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Physician/Researcher

Study Record Dates

• First Submitted: July 20, 2011
• First Posted: January 5, 2012
• Study Start: November 1, 2010
• Primary Completion: March 1, 2013
• Study Completion: September 1, 2014
• Last Updated: October 2, 2014

Record last verified: 2014-09

Locations

US (2)