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 A/S
Information provided by:
Karolinska University Hospital
ClinicalTrials.gov Identifier:
NCT00372125
First received: September 5, 2006
Last updated: November 25, 2009
Last verified: November 2009
  Purpose

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.


Condition Intervention
Prader-Willi Syndrome
Drug: Norditropin SimpleXx

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Crossover 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

Resource links provided by NLM:


Further study details as provided by Karolinska University Hospital:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 75
Study Start Date: April 2005
Estimated Study Completion Date: March 2010
Estimated Primary Completion Date: March 2010 (Final data collection date for primary outcome measure)
Intervention Details:
    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.
  Eligibility

Ages Eligible for Study:   18 Years to 50 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00372125

Locations
Denmark
Center for rare Diseases, Department of Pediatrics, Skejby University Hospital
Aarhus N, Denmark, 8200
Norway
Endokrinologisk seksjon, Med Avd, Rikshospitalet
Oslo, Norway
Sweden
Department of Endocrinology and Diabetology, Karolinska Hospital
Stockholm, Sweden, 171 76
Sponsors and Collaborators
Karolinska University Hospital
Novo Nordisk A/S
Investigators
Principal Investigator: Charlotte Hoybye, Dr. Department of Endocrinology and Diabetology, Karolinska Hospital
Study Chair: Jens S Christiansen, Professor Århus University Hospital, Denmark
  More Information

No publications provided by Karolinska University Hospital

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Charlotte Hoeybye, Karolinska University Hospital
ClinicalTrials.gov Identifier: NCT00372125     History of Changes
Other Study ID Numbers: CH1234
Study First Received: September 5, 2006
Last Updated: November 25, 2009
Health Authority: Sweden: Medical Products Agency
Norway: Norwegian Medicines Agency
Denmark: Danish Medicines Agency

Keywords provided by Karolinska University Hospital:
Prader-Willi syndrome
Growth hormone
Body composition

Additional relevant MeSH terms:
Prader-Willi Syndrome
Syndrome
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Disease
Pathologic Processes
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 19, 2014