Increlex Treatment of Children With Chronic Liver Disease and Short Stature

Chronic Liver Disease Clinical Trial

Official title:

Increlex Treatment of Children With Chronic Liver Disease and Short Stature

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT01314508
• Other study ID #: 07-03-018
• Status: Withdrawn
• Phase: N/A
• First received: March 8, 2011
• Last updated: July 12, 2016
• Start date: June 2011

Study information

• Verified date: July 2016
• Source: University of California, Los Angeles
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. primary completion date: October 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 4 Years to 18 Years

Eligibility

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

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Chronic Liver Disease
• Dwarfism
• Failure to Thrive
• Growth Failure
• Liver Diseases

Intervention

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.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
University of California, Los Angeles

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Growth velocity is the primary outcome. Improved height SDS	Improved growth velocity with improved height standard deviation scores (SDS) is the primary expected result.	One year of therapy	No
Secondary	Improved BMI	An improved body mass index is a secondary expected result of this study.	12 months	No
Secondary	Improved quality of life	An improved quality of life as assessed by the Pediatric Quality of Life Inventory forms is another expected result.	12 months	No