Brief Summary

This trial is conducted globally. The aim of this trial is to investigate the effect of liraglutide for weight management in paediatric subjects with Prader-Willi Syndrome.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for phase_3

Timeline

Completed

Started Nov 2015

Longer than P75 for phase_3

Geographic Reach

8 countries

32 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

5 years study duration

First Submitted

Initial submission to the registry

August 17, 2015

Completed

1 day until next milestone

First Posted

Study publicly available on registry

August 18, 2015

Completed

3 months until next milestone

Study Start

First participant enrolled

November 9, 2015

Completed

5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 4, 2020

Completed

15 days until next milestone

Study Completion

Last participant's last visit for all outcomes

November 19, 2020

Completed

1.2 years until next milestone

Results Posted

Study results publicly available

February 3, 2022

Completed

Last Updated

July 6, 2023

Status Verified

July 1, 2023

Enrollment Period

5 years

First QC Date

August 17, 2015

Results QC Date

October 27, 2021

Last Update Submit

July 5, 2023

Conditions

Metabolism and Nutrition Disorder Obesity

Outcome Measures

Primary Outcomes (2)

Change in Body Mass Index (BMI) Standard Deviation Score (SDS) From Baseline to 16 Weeks
Change in BMI SDS from baseline to week 16 is presented. BMI SDS also called Z-scores, was calculated using the following formula: Z=\[(y / M)\^L - 1\] / S\*L; where L, M and S are median (M), Box-cox power (L) and variation coefficient (S) of children/adolescents', y= individual BMI. BMI provided for each sex and age. For each participant, a standard deviation score Z (SDS) was calculated based on age and sex referring to the values L, M and S. Possible values range from -3 to +3, a negative score being beneficial.
Week 0, Week 16
Change in Body Mass Index (BMI) Standard Deviation Score (SDS) From Baseline to 52 Weeks
Change in BMI SDS from baseline to week 52 is presented. BMI SDS also called Z-scores, was calculated using the following formula: Z=\[(y / M)\^L - 1\] / S\*L; where L, M and S are median (M), Box-cox power (L) and variation coefficient (S) of children/adolescents', y= individual BMI. BMI provided for each sex and age. For each participant, a standard deviation score Z (SDS) was calculated based on age and sex referring to the values L, M and S. Possible values range from -3 to +3, a negative score being beneficial.
Week 0, Week 52

Secondary Outcomes (104)

Percentage of Participants Achieving ≥ 5% Reduction in Baseline BMI at Week 16
At week 16
Percentage of Participants Achieving ≥ 5% Reduction in Baseline BMI at Week 52
At week 52
Percentage of Participants Achieving ≥ 10% Reduction in Baseline BMI at Week 16
At week 16
Percentage of Participants Achieving ≥ 10% Reduction in Baseline BMI at Week 52
At week 52
Percentage of Participants With no Increase in BMI SDS at Week 16
At week 16
+99 more secondary outcomes

Study Arms (2)

Liraglutide

EXPERIMENTAL

Drug: liraglutide

Placebo

PLACEBO COMPARATOR

Drug: placebo

Interventions

liraglutideDRUG

Injected s.c./ subcutaneously (under the skin) This trial consists of a part A and a part B. Part A of the trial is conducted in obese adolescents with PWS. Part B of the trial is conducted in obese children with PWS. Entry into part A and part B of the trial will be sequential.

Liraglutide

placeboDRUG

Placebo

Eligibility Criteria

Age6 Years - 18 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial
Confirmed diagnosis of PWS (Prader-Willi Syndrome) (by genetic testing)
Male or female, age at the time of signing informed consent: - Part A: above or equal to 12 years and less than 18 years
Tanner stage 2-5 pubertal development for part A, and Tanner stage 1 for part B
BMI (body mass index) corresponding to equal or above 30 kg/m\^2 for adults by international cut-off points1 and equal or above the 95th percentile for age and sex (for diagnosis of obesity)
Stable body weight during the previous 90 days before screening ( below 10 kg self-reported weight change)
Testing has been performed to evaluate for adrenal insufficiency and documented in medical record

You may not qualify if:

Type 1 diabetes mellitus (T1DM)
Type 2 diabetes mellitus (T2DM)
Calcitonin equal or above 50 ng/L
No change in treatment plan with growth hormone (GH) from randomisation to the end of the open-label period patients on growth hormone to stay on, patients off GH to stay off during this period. Adjustments in doses of growth hormone will be permitted)
Family or personal history of Multiple Endocrine Neoplasia Type 2 (MEN2) or Medullary Thyroids Carcinoma (MTC)
History of pancreatitis (acute or chronic)
Treatment with any medication prescribed for weight loss within 90 days before screening (e.g. orlistat, zonisamide, topiramate/phentermine, lorcaserin, phentermine, bupropion/naltrexone,liraglutide, metformin)
Untreated adrenal insufficiency
Suggestive history of, or significant risk of gastroparesis (e.g. marked abdominal bloating post meal, history of vomiting, severe constipation), as judged by the Investigator

Contact the study team to confirm eligibility.