Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome

What Was Tested

This registered phase 3 trial tested Carbetocin.

Carbetocin is an oxytocin analog (a man-made chemical that is like oxytocin).

This study will also evaluate the safety and tolerability of LV-101.

It had three arms (groups): Placebo (placebo comparator), one (experimental) and one (experimental).

The registry classes the interventions as drug.

More on the Plan

Safety and tolerability will be measured by adverse events, laboratory tests, and physical exams.

After the 8-week placebo-controlled period, there will be a long-term follow-up period of 56 weeks and an optional extension period after study week 64 during which all participants will receive active treatment with LV-101.

Who Took Part

Enrollment: 130 actual participants took part.

It was open to people of any sex aged from 7 years up to 18 years.

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

Examples of who could join: Genetically-confirmed Prader-Willi syndrome. Provide voluntary, written informed consent (parent(s) / legal guardian(s) of participant); provide voluntary, written assent (participants, as appropriate). PWS Nutritional Phase 3 (hyperphagic, rarely feels full).

The full record lists 3 inclusion and 10 exclusion criteria; among the exclusions: living in a group home; genetically diagnosed schaaf-yang syndrome or other genetic, hormonal, or chromosomal cognitive impairment; new food-related interventions, including environment or dietary restrictions, within 1 month of screening.

How the Study Was Run

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

Because people in this Carbetocin study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

The Carbetocin group and the comparison group ran side by side rather than switching.

As a double-blind Carbetocin study, the assignments were hidden from both participants and investigators until the end.

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

It started in 2018-11-20 with completion listed for 2020-05-13.

It ran at 24 sites in United States (19), Canada (4) and Australia (1).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Hyperphagia Behavior (time frame: Baseline to Week 8). Obsessive and Compulsive Behaviors (time frame: baseline to Week 8).

It also tracked 3 secondary outcomes, for example Anxiety, Global Impression and Hyperphagia Behavior (Subset).

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Carbetocin in 130 people.

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

More context: the Carbetocin compound page, all human trials.