Nordic Study on the Effects of Growth Hormone (Norditropin SimpleXx) Treatment in Adults With Prader-Willi Syndrome
|Study Design:||Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
|Official Title:||Nordic Study on the Effects of Growth Hormone (Norditropin SimpleXx) Treatment in Adults With Prader-Willi Syndrome|
- Changes in body composition (lean body mass and fat mass) measured by dual energy X-ray absorptiometry (DXA) [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Bone mineral density measured by DXA [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Effects on forced expiratory volume (Peakflow) [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Standard photography appearance according to visual analogue scale (VAS) [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Effects on free and total IGF-I, IGF-binding protein (BP)-1 and 3 [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Effects on lipids (fasting triglycerides(TG), total, HDL and LDL cholesterol) [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Effects on body composition measured with bioimpedance [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Effects on haemoglobin (Hb), leucocyte and thrombocyte counts, FSH, LH, estradiol, Testosterone, inhibin B, TSH and Thyroxine [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Muscle and fat mass measured by abdominal and mid-femoral computerized tomography [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Activity of daily living measured a.m. Guralnik [ Time Frame: 36 months ] [ Designated as safety issue: No ]
- Quality of life estimated by questionnaires [ Time Frame: 36 months ] [ Designated as safety issue: No ]
|Study Start Date:||April 2005|
|Study Completion Date:||March 2010|
|Primary Completion Date:||March 2010 (Final data collection date for primary outcome measure)|
Active Comparator: Norditropin SimpleXx
0.3 mg/day or 0.4 mg/day if bodyweight was below or above 100 kg,for 4 weeks, 0.6 mg/day or 0.8 mg/day, for 11 months.
Drug: Norditropin SimpleXx
0.3-0.4 mg/day placebo or GH for 4 weeks. Thereafter 0.6-0.8 mg/day placebo or GH for 11 months. During the following 24 months open phase doses will be individually titrated.
Placebo Comparator: Placebo
Placebo for 12 months
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.
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).
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.
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.
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 0.3 mg/day respectively 0.4 mg/day if BW is below or above 100 kg. Thereafter doses will be increased to 0.6 mg/day (0.8 mg/day) and maintained fixed for 11 months. During the following 24 months open phase doses will be individually titrated.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00372125
|Center for rare Diseases, Department of Pediatrics, Skejby University Hospital|
|Aarhus N, Denmark, 8200|
|Endokrinologisk seksjon, Med Avd, Rikshospitalet|
|Department of Endocrinology and Diabetology, Karolinska Hospital|
|Stockholm, Sweden, 171 76|
|Principal Investigator:||Charlotte Hoybye, Dr.||Department of Endocrinology and Diabetology, Karolinska Hospital|
|Study Chair:||Jens S Christiansen, Professor||Århus University Hospital, Denmark|