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Nordic Study on the Effects of Growth Hormone (Norditropin SimpleXx) Treatment in Adults With Prader-Willi Syndrome

The recruitment status of this study is unknown because the information has not been verified recently.
Verified November 2009 by Karolinska University Hospital.
Recruitment status was  Active, not recruiting
Sponsor:
Collaborator:
Novo Nordisk
Information provided by:
Karolinska University Hospital
ClinicalTrials.gov Identifier:
NCT00372125
First received: September 5, 2006
Last updated: November 25, 2009
Last verified: November 2009
History of Changes

September 5, 2006
November 25, 2009
April 2005
March 2010   (final data collection date for primary outcome measure)
  • 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 ]
  • Muscle and fat mass measured by abdominal and mid-femoral computerized tomography (CT) scan [ 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 ]
  • Changes in body composition (lean body mass and fat mass) measured by dual energy X-ray absorptiometry (DXA)
  • Muscle and fat mass measured by abdominal and mid-femoral computerized tomography (CT) scan
  • Activity of daily living measured a.m. Guralnik
  • Quality of life estimated by questionnaires
Complete list of historical versions of study NCT00372125 on ClinicalTrials.gov Archive Site
  • 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 ]
  • Bone mineral density measured by DXA
  • Effects on forced expiratory volume (Peakflow)
  • Standard photography appearance according to visual analogue scale (VAS)
  • Effects on free and total IGF-I, IGF-binding protein (BP)-1 and 3
  • Effects on lipids (fasting triglycerides(TG), total, HDL and LDL cholesterol)
  • Effects on body composition measured with bioimpedance
  • Effects on haemoglobin (Hb), leucocyte and thrombocyte counts, FSH, LH, estradiol, Testosterone, inhibin B, TSH and Thyroxine
Not Provided
Not Provided
 
Nordic Study on the Effects of Growth Hormone (Norditropin SimpleXx) Treatment in Adults With Prader-Willi Syndrome
Nordic Study on the Effects of Growth Hormone (Norditropin SimpleXx) Treatment in Adults With Prader-Willi Syndrome

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.
Not Provided
Interventional
Not Provided
Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Prader-Willi Syndrome
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.
Not Provided
Jørgensen AP, Ueland T, Sode-Carlsen R, Schreiner T, Rabben KF, Farholt S, Høybye C, Christiansen JS, Bollerslev J. Two years of growth hormone treatment in adults with Prader-Willi syndrome do not improve the low BMD. J Clin Endocrinol Metab. 2013 Apr;98(4):E753-60. doi: 10.1210/jc.2012-3378. Epub 2013 Feb 22.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
75
March 2010
March 2010   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Genetically verified PWS diagnosis (by methylation and FISH test.)
  • Between 18 and 50 years old
  • Informed consent obtained before any trial-related activities. (Trial-related activities are any procedure that would not have been performed during normal management of the treatment of the subject.)

Exclusion Criteria:

  • Known or suspected allergy to GH preparation.
  • Previous participation in this trial.
  • GH treatment within the last 1 years
  • Malignancy or other serious diseases (ex severe cardiovascular diseases, severe infections)
  • Sexhormone treatment initiated within the last year
  • Pregnancy
  • Untreated respiratory impairment, untreated sleep apnoea or untreated respiratory infection.
Both
18 Years to 50 Years
No
Contact information is only displayed when the study is recruiting subjects
Denmark,   Norway,   Sweden
 
NCT00372125
CH1234
Yes
Charlotte Hoeybye, Karolinska University Hospital
Karolinska University Hospital
Novo Nordisk
Principal Investigator: Charlotte Hoybye, Dr. Department of Endocrinology and Diabetology, Karolinska Hospital
Study Chair: Jens S Christiansen, Professor Århus University Hospital, Denmark
Karolinska University Hospital
November 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP