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

Prader-Willi Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00372125
• Other study ID #: CH1234
• 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

• Prader-Willi Syndrome
• Syndrome

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