Prader Willi Syndrome MedDRA version: 14.1 Level: LLT Classification code 10041331 Term: Somatotrophin System Organ Class: 10022891 - Investigations
Conditions
Interventions
Trade Name: GENOTROPIN (Somatropin) is a growth hormone treatment. It is an exact copy of the natural growth hormone that our bodies make. The main difference is that GENOTROPIN is man-made.
Product
Sponsors
Dutch growth research foundation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Young adults with PWS who have been treated with GH for several years in the original Cohort study or Transition study will be included. They will be treated with GH according to this new follow-up protocol from 3 months after the end of the Transition study or when the inclusion in the Transition study has been completed. Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 10 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: The study participation will be closed to patients who do not meet the above mentioned inclusion criteria and/or have: - non cooperative behavior - extremely low dietary intake of less than minimal required intake according to WHO - medication to reduce weight (fat)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - A manualized treatment for an optimal dosage GH in young adults with Prader Willi Syndrome - To assess the long term effects and safety of GH-treatment in a dose of 0.33mg/m2/day on: - body composition - carbohydrate metabolism - serum lipids - blood pressure - behavioral problems ;Secondary Objective: - To study the effects of long term GH-treatment on thyroid hormone levels, IGF-I and IGF binding proteins, adiponectin, leptin and ghrelin. - To study the compliance to the diet. ;Primary end point(s): - Weight, Waist-hip ratio - Blood pressure • DXA for body composition and bone density, radiation exposure 200 mSv per scan Laboratory assessments - ASAT, ALAT, Creatinin, - Fasting glucose and insulin, OGTT - Fasting lipid levels: trygliceride (TG), total cholesterol, LDL, HDL and Lipoprotein [a] - IGF-I, IGFBP-1, IGFBP-3 - T4, free T4, TSH, T3, Reverse T3 - Leptin, Adiponectin, Ghrelin ;Timepoint(s) of evaluation of this end point: At six months, 12 months and 24 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Nutritional intake during 7 days (via daily intake book) Fasting glucose and insulin, OGTT - Fasting lipid levels: trygliceride (TG), total cholesterol, LDL, HDL and Lipoprotein [a] - IGF-I, IGFBP-1, IGFBP-3 - T4, free T4, TSH, T3, Reverse T3 - Leptin, Adiponectin, Ghrelin ;Timepoint(s) of evaluation of this end point: At six months, 12 months and 24 months | — |
Countries
Netherlands
Contacts
Public ContactDutch growth research foundation
Dutch growth research foundation
Outcome results
None listed