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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

46 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-04-30

Study Completion Date

2010-03-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Retrospective Observational Study on Efficacy and Safety of Norditropin® in Children With Prader-Willi Syndrome

NCT00705172

Genetic Disorder Prader-Willi Syndrome

COMPLETED

Investigating Efficacy and Safety of Once-weekly NNC0195-0092 Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency

NCT02616562

Growth Hormone Disorder Growth Hormone Deficiency in Children

COMPLETED PHASE2

Safety and Efficacy of Somatropin in Children With Growth Hormone Deficiency

NCT01502124

Growth Hormone Disorder Growth Hormone Deficiency in Children

COMPLETED PHASE3

Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)

NCT03972345

Growth Hormone Deficiency in Children Born Small for Gestational Age

ENROLLING_BY_INVITATION

A Trial Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of a Single Dose of Long-acting Growth Hormone (Somapacitan) Compared to Daily Dosing of Norditropin® SimpleXx® in Children With Growth Hormone Deficiency

NCT01973244

Growth Hormone Disorder Growth Hormone Deficiency in Children

COMPLETED PHASE1

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Prader-Willi Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Norditropin SimpleXx

0.3 mg/day or 0.4 mg/day if bodyweight was below or above 100 kg,for 4 weeks, 0.6 mg/day or 0.8 mg/day, for 11 months.

Group Type ACTIVE_COMPARATOR

Norditropin SimpleXx

Intervention Type DRUG

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.

Placebo

Placebo for 12 months

Group Type PLACEBO_COMPARATOR

Norditropin SimpleXx

Intervention Type DRUG

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.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

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.

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

50 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No