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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00372125
Other study ID # CH1234
Secondary ID
Status Completed
Phase N/A
First received September 5, 2006
Last updated September 11, 2016
Start date April 2005
Est. completion date March 2010

Study information

Verified date September 2016
Source Karolinska University Hospital
Contact n/a
Is FDA regulated No
Health authority Sweden: Medical Products AgencyNorway: Norwegian Medicines AgencyDenmark: Danish Medicines Agency
Study type Interventional

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Completed
Enrollment 46
Est. completion date March 2010
Est. primary completion date March 2010
Accepts healthy volunteers No
Gender Both
Age group 18 Years to 50 Years
Eligibility 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.

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

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.

Locations

Country Name City State
Denmark Center for rare Diseases, Department of Pediatrics, Skejby University Hospital Aarhus N
Norway Endokrinologisk seksjon, Med Avd, Rikshospitalet Oslo
Sweden Department of Endocrinology and Diabetology, Karolinska Hospital Stockholm

Sponsors (2)

Lead Sponsor Collaborator
Karolinska University Hospital Novo Nordisk A/S

Countries where clinical trial is conducted

Denmark,  Norway,  Sweden, 

Outcome

Type Measure Description Time frame Safety issue
Primary Changes in body composition (lean body mass and fat mass) measured by dual energy X-ray absorptiometry (DXA) 36 months No
Secondary Bone mineral density measured by DXA 36 months No
Secondary Effects on forced expiratory volume (Peakflow) 36 months No
Secondary Standard photography appearance according to visual analogue scale (VAS) 36 months No
Secondary Effects on free and total IGF-I, IGF-binding protein (BP)-1 and 3 36 months No
Secondary Effects on lipids (fasting triglycerides(TG), total, HDL and LDL cholesterol) 36 months No
Secondary Effects on body composition measured with bioimpedance 36 months No
Secondary Effects on haemoglobin (Hb), leucocyte and thrombocyte counts, FSH, LH, estradiol, Testosterone, inhibin B, TSH and Thyroxine 36 months No
Secondary Muscle and fat mass measured by abdominal and mid-femoral computerized tomography 36 months No
Secondary Activity of daily living measured a.m. Guralnik 36 months No
Secondary Quality of life estimated by questionnaires 36 months No
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