Prader Willi syndrome (PWS) MedDRA version: 20.0 Level: PT Classification code 10036476 Term: Prader-Willi syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Tesofensine
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Tesofensine
CAS Number: 195875-86-6
Current Sponsor code: NS2330
Other descriptive name: TESOFENSINE
Concentratio
Sponsors
Saniona A/S
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: All male and female pediatric subjects with confirmed diagnosis of PWS who completed the double blind phase (12 weeks) and the first OLE, will be invited to participate in the second open label 12 weeks extension phase, if deemed eligible by the Investigators. Are the trial subjects under 18? yes Number of subjects for this age range: 15 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: If subject/parents decide to not participate in OLE II, subject will finish the study based on the Protocol Amendment 1.6.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The purpose of this study is to investigate the safety and efficacy of co-administration of tesofensine/metoprolol treatment versus placebo in adult and pediatric subjects with Prader-Willi syndrome. The primary objective is to examine the effect of co-administration of tesofensine/metoprolol on body weight in subjects with PWS in an open labeled extension study.;Secondary Objective: The secondary objectives are: 1. To establish pharmacokinetic profile of tesofensine and metoprolol in subjects with PWS 2. To examine the change in hyperphagia-related behaviour in subjects with PWS by use of the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) 3. To examine the effect of co-administration of tesofensine/metoprolol on glycaemic control and lipid profile in subjects with PWS 4. To examine the effect of co-administration of tesofensine/metoprolol on HR and BP in subjects with PWS 5. To examine the effects of co-administration of tesofensine/metoprolol on body composition in subjects with PWS 6. To evaluate overall safety and tolerability of co-administration of the tesofensine/metoprolol in subjects with PWS ;Primary end point(s): The primary endpoint for this study is percent change from baseline to end of treatment periods in mean body weight.;Timepoint(s) of evaluation of this end point: Day 90, Day 180, Day 270 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary endpoints include: 1. Steady state concentrations of tesofensine and metoprolol as measured by trough values 2. Uptake and initial plateau concentrations of metoprolol as measured by samples taken 2 and 4 hours after dose administration 3. Change from baseline to end of the treatment periods in total HQCT score 4. Change from baseline to end of treatment periods in mean body weight (kg) 5. Change from baseline to end of treatment periods in fat- and fat-free mass (%) by dual X-ray absorptiometry (DXA) 6. Change from baseline to end of treatment periods in HR (bpm), SBP (mmHg), DBP (mmHg) 7. AEs, clinical labs, ECG There are additional exploratory endpoints: 1. Change from baseline to end of treatment periods in FPG, insulin 2. Change from baseline to end of treatment periods in lipid profile 3. Change from baseline to end of treatment periods in waist circumference (cm);Timepoint(s) of evaluation of this end point: From baseline to end of study treatment. | — |
Countries
Czech Republic, Hungary
Contacts
Public ContactClinical & Regulatory department
Saniona A/S
Outcome results
None listed