Clinical trial NCT01314508: Increlex Treatment of Children With Chronic Liver Disease and Short Stature
Official title: Increlex Treatment of Children With Chronic Liver Disease and Short Stature
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: NA
Conditions: Growth Failure
Chronic Liver Disease
Keywords: pre-pubertal children
pre-transplant
not treated with growth hormone for at least 3 months
Lead sponsor: University of California, Los Angeles
Sponsor class: OTHER
First posted: 2011-03-14
Enrollment: 0
Sex: ALL
Minimum age (years): 4
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: Growth velocity is the primary outcome. Improved height SDS
timeFrame: One year of therapy
Secondary outcomes:
measure: Improved BMI
timeFrame: 12 months
measure: Improved quality of life
timeFrame: 12 months
Other outcomes:
Locations:
Study officials:
References: