Increlex Treatment of Children With Chronic Liver Disease and Short Stature

The recruitment status of this study is unknown because the information has not been verified recently.
Verified February 2011 by University of California, Los Angeles.
Recruitment status was  Not yet recruiting
Sponsor:
Information provided by:
University of California, Los Angeles
ClinicalTrials.gov Identifier:
NCT01314508
First received: March 8, 2011
Last updated: March 23, 2011
Last verified: February 2011

March 8, 2011
March 23, 2011
June 2011
June 2013   (final data collection date for primary outcome measure)
Growth velocity is the primary outcome. Improved height SDS [ Time Frame: One year of therapy ] [ Designated as safety issue: No ]
Improved growth velocity with improved height standard deviation scores (SDS) is the primary expected result.
Growth velocity is the primary outcome. Improved height SDS [ Time Frame: One year of therapy ] [ Designated as safety issue: No ]
Improved growth velocity with improved height standard deviation scores (SDS) is the primary exoected resykt.
Complete list of historical versions of study NCT01314508 on ClinicalTrials.gov Archive Site
  • Improved BMI [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    An improved body mass index is a secondary expected result of this study.
  • Improved quality of life [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    An improved quality of life as assessed by the Pediatric Quality of Life Inventory forms is another expected result.
Same as current
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Increlex Treatment of Children With Chronic Liver Disease and Short Stature
Increlex Treatment of Children With Chronic Liver Disease and Short Stature

A major consequence of chronic liver disease in childhood is growth failure. This is because a chemical essential for growth called growth factor is created in the liver. Lack of response to growth hormone in people with chronic liver disease is characterized by high levels of growth hormone and low levels of growth factors. This growth hormone resistance is reflected in a variety of factors including insulin resistance and low nutritional intake. Unfortunately, growth hormone therapy has no effect for children with liver disease. In addition, failure of normal growth or malnutrition makes liver disease even worse in children, and growth hormone therapy is not likely to reverse this. A lack of proper nutrition is associated with hospitalizations and frequent complications. Poor growth is a predictor of poor outcomes after liver transplantation. Thus the management of children with liver disease remains a challenge. Children who have successful orthotopic liver transplants (OLT) show much improvement in some aspects of growth, including skin fold thickness, mid-arm circumference, and normalization of growth factor levels. However, some studies have recently reported that the growth of 15-20% of children remains poor even after a liver transplant. This can be explained by persistent abnormalities in growth factors after transplant.

Growth factor was found to be a good tool for prognosis in patients with chronic liver disease. Studies showed that patients with liver cirrhosis and growth factor levels below normal values showed lower long-term survival rates compared with patients who had above normal values. This suggests that growth factor can be a good predictor of survival and early marker of poor liver function. In this case, aggressive feeding may modestly improve growth factor levels leading to improved growth but it is unlikely that effects will be optimal. The investigators propose that growth factor administration may have a positive effect that leads to better growth which is a major predictor of good outcome. To date, no reports study the use of growth factor in children with chronic liver disease. This study proposes to examine the effect of growth factor therapy in childhood chronic liver disease.

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Interventional
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Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Growth Failure
  • Chronic Liver Disease
Drug: Increlex
Increlex therapy will begin at 40 micrograms/kg/day twice a day. The dose will be escalated by 20 mcg twice a day every other week up to 100mcg/kg/week.
All patients will be treated with IGF-1 factors
Patients will serve as their own control.
Intervention: Drug: Increlex
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Not yet recruiting
20
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June 2013   (final data collection date for primary outcome measure)

Inclusion Criteria:

Pre liver transplant patients with:

  • Chronic liver disease
  • Short stature (< 5%)
  • Low IGF-1 (<-1SDS for age)
  • Chronologic age 4-18 and bone age < 14 for boys and < 12 for girls (pre-pubertal)

Exclusion Criteria:

  • Status post transplant
  • Evidence of malignancy
  • Diabetes mellitus
  • Participation in other clinical trials involving investigational products
  • Treatment with growth hormone within 3 months
  • Pregnancy
  • Significant abnormality in clinical results
  • Hypoglycemic at baseline
  • Allergic to benzyl alcohol
Both
4 Years to 18 Years
No
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NCT01314508
07-03-018
No
Anna Haddal, M.D., UCLA Department of Pediatrics, Division of Pediatric Endocrinology
University of California, Los Angeles
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Not Provided
University of California, Los Angeles
February 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP