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.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Type d'étude
Phase
- N'est pas applicable
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
La description
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
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Autre: All patients will be treated with IGF-1 factors
Patients will serve as their own control.
|
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.
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Growth velocity is the primary outcome. Improved height SDS
Délai: One year of therapy
|
Improved growth velocity with improved height standard deviation scores (SDS) is the primary expected result.
|
One year of therapy
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Improved BMI
Délai: 12 months
|
An improved body mass index is a secondary expected result of this study.
|
12 months
|
|
Improved quality of life
Délai: 12 months
|
An improved quality of life as assessed by the Pediatric Quality of Life Inventory forms is another expected result.
|
12 months
|
Collaborateurs et enquêteurs
Parrainer
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude
Achèvement primaire (Réel)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Estimation)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Estimation)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Mots clés
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- 07-03-018
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