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Trial · Interventional

CompletedPhase 3Results postedRegistry data

Effect of Liraglutide for Weight Management in Paediatric Subjects With Prader-Willi Syndrome

NCTNCT02527200 (opens ClinicalTrials.gov in a new tab)Other idNN8022-4179Other id2014-004415-37Other idU1111-1162-7884Other idNL54145.078.15
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 3
Enrollment
56 participants (actual)
Sponsor
Novo Nordisk A/S · Industry
Design
Randomized · Double masking
Countries
8 countries

United States, Australia, Canada, France, Italy, Netherlands, New Zealand, Turkey (Türkiye)

Start
9 Nov 2015
Primary completion
4 Nov 2020
Completion
19 Nov 2020
First posted
18 Aug 2015 (estimated)
Last update posted
6 Jul 2023
Conditions
Metabolism and Nutrition Disorder · Obesity

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2015201620172018201920202021
  1. Ph 3

    Effect of Liraglutide for Weight Management in Paediatric Subjects With Prader-Willi Syndrome

    NCT02527200

    9 Nov 2015 to 19 Nov 2020

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • liraglutideDrug
  • placeboDrug

Arms2

  • Experimental

    Liraglutide

  • Placebo comparator

    Placebo

Primary outcomes2

Outcomes the sponsor listed as primary.

  1. Change in Body Mass Index (BMI) Standard Deviation Score (SDS) From Baseline to 16 Weeks

    Time frame · Week 0, Week 16

    Registry description

    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.

  2. Change in Body Mass Index (BMI) Standard Deviation Score (SDS) From Baseline to 52 Weeks

    Time frame · Week 0, Week 52

    Registry description

    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.

Secondary outcomes(20)

  1. Percentage of Participants Achieving ≥ 5% Reduction in Baseline BMI at Week 16

    Time frame · At week 16

    Registry description

    Percentage of participants achieving more than or equal to (≥) 5% reduction in their baseline (week 0) BMI at week 16 is presented. In below table, 'Yes' infers percentage of participants who achieved ≥ 5% reduction in their baseline (week 0) BMI at week 16 and 'No' infers percentage of participants who did not achieve ≥ 5% reduction in their baseline (week 0) BMI at week 16.

  2. Percentage of Participants Achieving ≥ 5% Reduction in Baseline BMI at Week 52

    Time frame · At week 52

    Registry description

    Percentage of participants achieving more than or equal to (≥) 5% reduction in their baseline (week 0) BMI at week 52 is presented. In below table, 'Yes' infers percentage of participants who achieved ≥ 5% reduction in their baseline (week 0) BMI at week 52 and 'No' infers percentage of participants who did not achieve ≥ 5% reduction in their baseline (week 0) BMI at week 52.

  3. Percentage of Participants Achieving ≥ 10% Reduction in Baseline BMI at Week 16

    Time frame · At week 16

    Registry description

    Percentage of participants achieving more than or equal to (≥) 10% reduction in their baseline (week 0) BMI at week 16 is presented. In below table, 'Yes' infers percentage of participants who achieved ≥ 10% reduction in their baseline (week 0) BMI at week 16 and 'No' infers percentage of participants who did not achieve ≥ 10% reduction in their baseline (week 0) BMI at week 16.

  4. Percentage of Participants Achieving ≥ 10% Reduction in Baseline BMI at Week 52

    Time frame · At week 52

    Registry description

    Percentage of participants achieving more than or equal to (≥) 10% reduction in their baseline (week 0) BMI at week 52 is presented. In below table, 'Yes' infers percentage of participants who achieved ≥ 10% reduction in their baseline (week 0) BMI at week 52 and 'No' infers percentage of participants who did not achieve ≥ 10% reduction in their baseline (week 0) BMI at week 52.

  5. Percentage of Participants With no Increase in BMI SDS at Week 16

    Time frame · At week 16

    Registry description

    Percentage of participants with no increase in BMI SDS at week 16 is presented. In below table, 'Yes' infers percentage of participants with no increase in BMI SDS at week 16 and 'No' infers percentage of participants with increase in BMI SDS at week 16.

  6. Percentage of Participants With no Increase in BMI SDS at Week 52

    Time frame · At week 52

    Registry description

    Percentage of participants with no increase in BMI SDS at week 52 is presented. In below table, 'Yes' infers percentage of participants with no increase in BMI SDS at week 52 and 'No' infers percentage of participants with increase in BMI SDS at week 52.

  7. Change in BMI From Baseline at Week 16

    Time frame · Week 0, week 16

    Registry description

    Change in body mass index (BMI) from baseline to week 16 is presented.

  8. Change in BMI From Baseline at Week 52

    Time frame · Week 0, week 52

    Registry description

    Change in body mass index (BMI) from baseline to week 52 is presented.

  9. Change in Body Weight (Kilogram (kg)) From Baseline at Week 16

    Time frame · Week 0, week 16

    Registry description

    Change in body weight (kg) from baseline to week 16 is presented.

  10. Change in Body Weight (kg) From Baseline at Week 52

    Time frame · Week 0, week 52

    Registry description

    Change in body weight (kg) from baseline to week 52 is presented.

  11. Change in Body Weight (Pounds (lb)) From Baseline at Week 16

    Time frame · Week 0, week 16

    Registry description

    Change in body weight (lb) from baseline to week 16 is presented.

  12. Change in Body Weight (lb) From Baseline at Week 52

    Time frame · Week 0, week 52

    Registry description

    Change in body weight (lb) from baseline to week 52 is presented.

  13. Change in Body Weight (Percentage [%]) From Baseline at Week 16

    Time frame · Week 0, week 16

    Registry description

    Change in body weight (%) from baseline to week 16 is presented.

  14. Change in Body Weight (%) From Baseline at Week 52

    Time frame · Week 0, week 52

    Registry description

    Change in body weight (%) from baseline to week 52 is presented.

  15. Change in Waist Circumference From Baseline at Week 16

    Time frame · Week 0, week 16

    Registry description

    Change in waist circumference from baseline to week 16 is presented.

  16. Change in Waist Circumference From Baseline at Week 52

    Time frame · Week 0, week 52

    Registry description

    Change in waist circumference from baseline to week 52 is presented.

  17. Change in Waist-to-hip Circumference Ratio From Baseline at Week 16

    Time frame · Week 0, week 16

    Registry description

    Change in waist-to-hip circumference ratio from baseline to week 16 is presented.

  18. Change in Waist-to-hip Circumference Ratio From Baseline at Week 52

    Time frame · Week 0, week 52

    Registry description

    Change in waist-to-hip circumference ratio from baseline to week 52 is presented.

  19. Change in Hyperphagia Score: Total Score and Hyperphagic Behaviour, Drive and Severity Score From Baseline at Week 16

    Time frame · Week 0, week 16

    Registry description

    Change in hyperphagia score (hyperphagia total score and hyperphagic behaviour, drive and severity score respectively), from baseline to week 16 is presented. The Hyperphagia Questionnaire (HQ)- Total Score. It contains 11 questions which are categorised into 3 subscales; Hyperphagic Behaviour, Hyperphagic Drive, and Hyperphagia Severity. The subscales are summed together to compute the Total Score. The Total Score ranges from 11-55, with higher scores indicating more hyperphagia and a worse outcome. The HQ-Behaviour Factor Score ranges from 5-25, with higher scores indicating more hyperphagic behaviours and a worse outcome. The HQ- Drive Factor Score ranges from 4-20, with higher scores indicating higher hyperphagic drive and a worse outcome. The HQ- Severity Factor Score ranges from 2-10, with higher scores indicating higher severity.

  20. Change in Hyperphagia Score: Total Score and Hyperphagic Behaviour, Drive and Severity Score From Baseline at Week 52

    Time frame · Week 0, week 52

    Registry description

    Change in hyperphagia score (hyperphagia total score and hyperphagic behaviour, drive and severity score respectively), from baseline to week 52 is presented.The Hyperphagia Questionnaire (HQ)- Total Score. It contains 11 questions which are categorised into 3 subscales; Hyperphagic Behaviour, Hyperphagic Drive, and Hyperphagia Severity. The subscales are summed together to compute the Total Score. The Total Score ranges from 11-55, with higher scores indicating more hyperphagia and a worse outcome. The HQ-Behaviour Factor Score ranges from 5-25, with higher scores indicating more hyperphagic behaviours and a worse outcome. The HQ- Drive Factor Score ranges from 4-20, with higher scores indicating higher hyperphagic drive and a worse outcome. The HQ- Severity Factor Score ranges from 2-10, with higher scores indicating higher severity.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· Week 0, Week 16

Change in Body Mass Index (BMI) Standard Deviation Score (SDS) From Baseline to 16 Weeks

GroupValue (SDS score)Spread
Part A: Liraglutide-0.180.21
Part A: Placebo-0.180.23
Part B: Liraglutide-0.500.65
Part B: Placebo-0.480.39
Part A+B: Liraglutide-0.330.49
Part A+B: Placebo-0.290.32
Primary outcomeMean· Week 0, Week 52

Change in Body Mass Index (BMI) Standard Deviation Score (SDS) From Baseline to 52 Weeks

GroupValue (SDS score)Spread
Part A: Liraglutide-0.270.37
Part A: Placebo-0.130.25
Part B: Liraglutide-0.791.21
Part B: Placebo-0.710.68
Part A+B: Liraglutide-0.500.88
Part A+B: Placebo-0.360.53
Secondary and other outcome results(6)
Secondary outcomeNumber· At week 16

Percentage of Participants Achieving ≥ 5% Reduction in Baseline BMI at Week 16

GroupValue (Percentage of participants)
Part A: Liraglutide27.8
Part A: Placebo8.3
Part B: Liraglutide37.5
Part B: Placebo57.1
Part A+B: Liraglutide32.4
Part A+B: Placebo26.3
Secondary outcomeNumber· At week 52

Percentage of Participants Achieving ≥ 5% Reduction in Baseline BMI at Week 52

GroupValue (Percentage of participants)
Part A: Liraglutide29.4
Part A: Placebo18.2
Part B: Liraglutide35.7
Part B: Placebo42.9
Part A+B: Liraglutide32.3
Part A+B: Placebo27.8
Secondary outcomeNumber· At week 16

Percentage of Participants Achieving ≥ 10% Reduction in Baseline BMI at Week 16

GroupValue (Percentage of participants)
Part A: Liraglutide0
Part A: Placebo0
Part B: Liraglutide6.3
Part B: Placebo14.3
Part A+B: Liraglutide2.9
Part A+B: Placebo5.3
Secondary outcomeNumber· At week 52

Percentage of Participants Achieving ≥ 10% Reduction in Baseline BMI at Week 52

GroupValue (Percentage of participants)
Part A: Liraglutide11.8
Part A: Placebo0
Part B: Liraglutide14.3
Part B: Placebo28.6
Part A+B: Liraglutide12.9
Part A+B: Placebo11.1
Secondary outcomeNumber· At week 16

Percentage of Participants With no Increase in BMI SDS at Week 16

GroupValue (Percentage of participants)
Part A: Liraglutide66.7
Part A: Placebo75.0
Part B: Liraglutide87.5
Part B: Placebo100
Part A+B: Liraglutide76.5
Part A+B: Placebo84.2
Secondary outcomeNumber· At week 52

Percentage of Participants With no Increase in BMI SDS at Week 52

GroupValue (Percentage of participants)
Part A: Liraglutide70.6
Part A: Placebo72.7
Part B: Liraglutide78.6
Part B: Placebo100
Part A+B: Liraglutide73.3
Part A+B: Placebo81.3

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Part A: LiraglutideParticipants received once daily subcutaneous injection of liraglutide for 52 weeks. Dosing was initiated with liraglutide 0.6 mg daily for one week and increased in weekly dosage steps of 0.6 mg until a maximum tolerated dose (MTD) (as judged by the Investigator) or a dose of 3.0 mg liraglutide was reached, i.e., 0.6 mg (week 1), 1.2 mg (week 2), 1.8 mg (week 3), 2.4 mg (week 4) and 3.0 mg (week 5 to week 52).0 / 201 / 2016 / 20
Part B: LiraglutideParticipants received once daily subcutaneous injection of liraglutide for 52 weeks. For children with body weight ≥ 45 kg; dosing was initiated with liraglutide 0.6 mg daily for one week and increased in weekly dosage steps of 0.6 mg until a MTD (as judged by the Investigator) or a dose of 3.0 mg liraglutide was reached, i.e., 0.6 mg (week 1), 1.2 mg (week 2), 1.8 mg (week 3), 2.4 mg (week 4) and 3.0 mg (week 5 to week 52). For children with body weight \<45 kg: dosing was initiated with liraglutide 0.3 mg daily for one week and increased to 0.6 mg after the first week. Thereafter, the dose was increased in weekly steps of 0.6 mg until an MTD (as judged by the Investigator) or a dose of 2.4 mg liraglutide was reached. i.e., 0.3 mg (week 1), 0.6 mg (week 2), 1.2 mg (week 3), 1.8 mg (week 4), 2.4 mg (week 5 to week 52).0 / 172 / 1717 / 17
Part A: PlaceboParticipants received once daily subcutaneous injection of placebo matched to liraglutide for 16 weeks.0 / 120 / 1210 / 12
Part B: PlaceboParticipants received once daily subcutaneous injection of placebo matched to liraglutide for 16 weeks.0 / 71 / 74 / 7

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies1

Studies that cite this trial.

Studies with results

1 study on this page

Indexing hint: Randomized controlled trial · 2022

6 results

Liraglutide for Weight Management in Children and Adolescents With Prader-Willi Syndrome and Obesity.

Diene et al. 2022The Journal of clinical endocrinology and metabolismcited by 47

PMID 36181471PMC9759167DOI 10.1210/clinem/dgac549PUB-36181471

Free to read · publisher termsCC BY-NC-ND6 results
Read at sourceSee results (6)

Eligibility

Who could join, as posted.

Sex
All
Age
from 6 Years up to 18 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * 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 Exclusion Criteria: * 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

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