Nordic Study on the Effects of Growth Hormone (Norditropin SimpleXx) Treatment in Adults With Prader-Willi Syndrome
Condition: Prader-Willi Syndrome
MeSH terms: Prader-Willi Syndrome; Syndrome
Study type: Interventional

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 PWS.The 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: 
      OBJECTIVE(S):

      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 PRODUCT(S):

      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.
    

Intervention name(s): Norditropin SimpleXx
Intervention type(s): Drug
Intervention description(s): 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.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment
Keywords: Prader-Willi syndrome; Growth hormone; Body composition