Pilot Clinical Trial of Repeated Doses of Macimorelin to Assess Safety and Efficacy in Patients With Cancer Cachexia

What Was Tested

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

It had two arms (groups): Macimorelin (active comparator) and Placebo (placebo comparator).

The registry classes the interventions as drug.

Who Took Part

The registry lists 15 actual participants took part.

It was open to people of any sex.

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

Examples of who could join: Subjects ≥18 years of age with histological diagnosis of incurable cancer (solid tumor),. ECOG performance status of 0-2,. Provide written informed consent prior to screening.

The full record lists 4 inclusion and 12 exclusion criteria; among the exclusions: obesity (body weight \>140 kg);; recent active excessive alcohol or illicit drug use;; severe depression as determined by the investigator;.

How the Study Was Run

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

Random assignment meant chance, not the researchers, decided who got Macimorelin (non-peptide) and who got the comparison, which limits bias.

The Macimorelin (non-peptide) group and the comparison group ran side by side rather than switching.

Blinding on both sides kept the participants and researchers unaware of who received Macimorelin (non-peptide).

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

It started in 2012-05 with completion listed for 2020-07.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Change of Body Weight (time frame: 7 days). Change of Insulin-like Growth Factor-1 (IGF-1) Plasma Levels (time frame: 7 days). Change of Quality of Life Score (time frame: 7 days).

It also tracked 25 secondary outcomes, for example Food Intake and Diary, Appetite (Visual Analog Scale [VAS] for Appetite), Handgrip Strength, Energy Expenditure as Measured by Indirect Calorimetry, Laboratory Assays and Safety Laboratory (White Blood Cells).

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Macimorelin (non-peptide) in 15 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 Macimorelin (non-peptide) compound page, all human trials.