Ignite Creation Date:
2024-05-05 @ 5:03 PM
Last Modification Date:
2024-10-26 @ 9:27 AM
Study NCT ID:
NCT00372125
Status:
COMPLETED
Last Update Posted:
2016-09-13
First Post:
2006-09-05
Brief Title:
Nordic Study on the Effects of Growth Hormone Norditropin SimpleXx Treatment in Adults With Prader-Willi Syndrome
Sponsor:
Karolinska University Hospital
Organization:
Karolinska University Hospital
Study Overview
Official Title:
Nordic Study on the Effects of Growth Hormone Norditropin SimpleXx Treatment in Adults With Prader-Willi Syndrome
Status:
COMPLETED
Status Verified Date:
2016-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The aim of this study is to study the effects of GH on body composition lipid and glucose metabolism physical performance and safety aspects in adults with PWSThe patients are randomized to either GH or placebo the first year of the study subsequently followed by two years of GH treatment the study is performed in Norway Sweden and Denmark
Detailed Description:
OBJECTIVES
Prader Willi syndrome PWS is a multi-symptomatic genetic disorder associated with abnormalities in the growth hormone GH-insulin-like-growth factor IGF-I axis and in the body composition GH treatment is a registered indication in children with PWS and improves growth rate and body composition One pilot study in adult patients with clinical PWS has shown beneficial effects on body composition without simultaneous significant side effects The aim of the present study is to evaluate the effects of GH treatment on body composition muscle function and quality of life in PWS adults
TRIAL DESIGN
The study will be an investigator initiated and investigator sponsored multinational and multi-centre trial including centres in Norway Sweden and Denmark Within each centre patients will be randomised double blind to one year treatment with daily injections of GH or placebo efficacy followed by a two year observation period on GH treatment safety
TRIAL POPULATION
Twenty patients from each centre are included in the study The patients need a genetically verified diagnosis and should be between 18 and 40 years old Patients are excluded if GH treatment has been given within the last two years if they have a malignancy or other serious diseases in particular severe respiratory diseases
ASSESSMENTS
Effect is evaluated primarily as changes in body composition activity of daily living and quality of life
SAFETY Before starting in the study all patients will be examined for tonsillary hypertrophy and sleep apnoea Oral Glucose Tolerance Tests will be performed regularly
TRIAL PRODUCTS
During the initial 4 weeks of the placebo-controlled study phase patients will be treated with sc injections of GH Norditropin Simplexx in the evening with doses of 03 mgday respectively 04 mgday if BW is below or above 100 kg Thereafter doses will be increased to 06 mgday 08 mgday and maintained fixed for 11 months During the following 24 months open phase doses will be individually titrated
Prader Willi syndrome PWS is a multi-symptomatic genetic disorder associated with abnormalities in the growth hormone GH-insulin-like-growth factor IGF-I axis and in the body composition GH treatment is a registered indication in children with PWS and improves growth rate and body composition One pilot study in adult patients with clinical PWS has shown beneficial effects on body composition without simultaneous significant side effects The aim of the present study is to evaluate the effects of GH treatment on body composition muscle function and quality of life in PWS adults
TRIAL DESIGN
The study will be an investigator initiated and investigator sponsored multinational and multi-centre trial including centres in Norway Sweden and Denmark Within each centre patients will be randomised double blind to one year treatment with daily injections of GH or placebo efficacy followed by a two year observation period on GH treatment safety
TRIAL POPULATION
Twenty patients from each centre are included in the study The patients need a genetically verified diagnosis and should be between 18 and 40 years old Patients are excluded if GH treatment has been given within the last two years if they have a malignancy or other serious diseases in particular severe respiratory diseases
ASSESSMENTS
Effect is evaluated primarily as changes in body composition activity of daily living and quality of life
SAFETY Before starting in the study all patients will be examined for tonsillary hypertrophy and sleep apnoea Oral Glucose Tolerance Tests will be performed regularly
TRIAL PRODUCTS
During the initial 4 weeks of the placebo-controlled study phase patients will be treated with sc injections of GH Norditropin Simplexx in the evening with doses of 03 mgday respectively 04 mgday if BW is below or above 100 kg Thereafter doses will be increased to 06 mgday 08 mgday and maintained fixed for 11 months During the following 24 months open phase doses will be individually titrated
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None