Increlex Treatment of Children With Chronic Liver Disease and Short Stature

NCT01314508 | Withdrawn

• 1 other identifier: 07-03-018
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Growth Failure; Chronic Liver Disease

Keywords

pre-pubertal children; pre-transplant; not treated with growth hormone for at least 3 months

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (2)

• Improved BMI

  12 months

• Improved quality of life

  12 months

Study Arms (1)

All patients will be treated with IGF-1 factors

OTHER

Patients will serve as their own control.

Drug: Increlex

Interventions

Increlex DRUG

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

Eligibility Criteria

• Age: 4 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• University of California, Los Angeles: lead

MeSH Terms

Conditions

Failure to Thrive

Interventions

mecasermin

Condition Hierarchy (Ancestors)

Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 8, 2011
• First Posted: March 14, 2011
• Study Start: June 1, 2011
• Primary Completion: October 1, 2011
• Last Updated: April 14, 2026

Record last verified: 2016-07