Co-administration of Tesofensine/Metoprolol in Subjects With Prader-Willi Syndrome (PWS)

What Was Tested

This registered phase 2 trial tested Tesofensine (non-peptide).

It had two arms (groups): Tesofensine/Metoprolol (experimental) and Tesofensine/Metoprolol placebo (placebo comparator).

Tesofensine + metoprolol administered once a day, in the morning with a meal.

Placebo tablets matching tesofensine + metoprolol administered once a day, in the morning with meal.

The registry classes the interventions as drug.

More on the Plan

Study medication will be administered for 91 days.

Who Took Part

Enrollment: 18 actual participants took part.

It was open to people of any sex aged from 12 years up to 30 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Males and females with a confirmed genetic diagnosis of Prader-Willi syndrome. Step 1: Adults aged 18-30. Step 2: Adolescents aged 12-17. Body Mass Index (BMI):. Step 1: Adults with ≥25 kg/m2.

The full record lists 14 inclusion and 20 exclusion criteria; among the exclusions: step 1: adults with \>140/90; step 2: adolescents with ≥95th percentile for gender, age, and height; heart rate (hr) ≥ 90, \<50 bpm.

How the Study Was Run

Design: randomized, parallel-group and quadruple-blind.

The participants were allocated by chance, so the Tesofensine (non-peptide) group and the comparison group should start out alike.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Neither the participants nor the staff measuring them knew who was on Tesofensine (non-peptide), so expectations could not easily colour the readings.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2017-03-30 with completion listed for 2019-07-22.

It ran at 2 sites in Czechia (1) and Hungary (1).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Percent Change From Baseline to End of Treatment in Mean Body Weight (time frame: DB Step 1: Day 1 to Day 91; DB Step 2: Day 1 to Day 91; OLE I: Day 91 to Day 181; OLE II: Day 181 to Day 271).

It also tracked 15 secondary outcomes, for example Change From Baseline to End of Treatment in Mean Body Weight, Change From Baseline to End of Treatment in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Score, Steady State Concentrations of Tesofensine and Metoprolol as Measured by Trough Values, Change From Baseline to End of Treatment in Fat- and Fat Free Mass (%) by Dual X-ray Absorptiometry (DEXA), Change From Baseline to End of Treatment in Bone Mineral Density (BMD) by Dual X-ray Absorptiometry (DEXA) and Change From Baseline to End of Treatment in Bone Mineral Content (BMC) by Dual X-ray Absorptiometry (DEXA).

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Tesofensine (non-peptide) in 18 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Tesofensine (non-peptide) compound page, all human trials.