Investigation of a New, Oral Growth Hormone Secretagogue, AEZS-130 as a Growth Hormone Stimulation Test.

What Was Tested

The trial (phase 3) is built around GHRH (Growth Hormone-Releasing Hormone) and Macimorelin (non-peptide).

The diagnosis of growth hormone deficiency (GHD) in adults is established by laboratory testing in patients with an appropriate clinical history of hypothalamic pituitary disease.

Two tests that are considered to be gold standard tests for the diagnosis of GHD are the insulin tolerance test (ITT) and growth hormone releasing hormone (GHRH) combined with L-arginine (L-ARG).

However, these tests are either bothersome (given intravenously) to the patient or are linked with side effects.

It had two arms (groups): AEZS-130 ( formerly ARD-07) (experimental) and L-ARG+GHRH (active comparator).

The registry classes the interventions as drug.

More on the Plan

Thirty control subjects (i.e., without AGHD) were matched to the 30 AGHD patients who were not previously matched.

Matching was based upon gender, age, BMI, and estrogen status for females.

There was no cross over due to the unavailability of GHRH (Geref) in the United States.

Under Amendment #4 to this protocol, 10 additional AGHD subjects were to be enrolled and matched as described above.

Who Took Part

The registry lists 101 actual participants took part.

It was open to people of any sex aged from 18 years with no upper age limit.

Healthy volunteers could join, not only people with the condition.

Examples of who could join: Undergone normal growth and development. Normal serum prolactin (PRL) concentrations. Females should have a history of regular, age-appropriate menses. Males should have normal serum testosterone concentrations. Matched GHD subject already enrolled in study; matched in terms of sex, age, BMI and Estrogen status (women only).

The full record lists 5 inclusion and 20 exclusion criteria; among the exclusions: inability or unwillingness to comply with study medication; pregnancy or lactation; treatment with any drugs that might prolong qt/qtc.

How the Study Was Run

The set-up was randomized, crossover and open-label.

Because people in this GHRH (Growth Hormone-Releasing Hormone) study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

In the crossover set-up each of the participants took both GHRH (Growth Hormone-Releasing Hormone) and the comparison in turn, acting as their own control.

Nothing was hidden in this open-label GHRH (Growth Hormone-Releasing Hormone) study: the participants and staff both knew the treatment.

It started in 2007-06 with completion listed for 2011-07.

It ran at 13 sites in United States (13).

Its stated primary purpose is diagnostic.

What Was Measured

Primary outcome: Receiver Operating Characteristic (ROC) Analysis on Peak GH (Growth Hormon) Concentrations.

It also tracked 3 secondary outcomes, for example Peak Insulin-Like Growth Factor (IGF)-1 Concentration Following Treatment, Classification and Regression Tree (CART) Analysis of Peak Growth Hormone (GH) Following Macimorelin Administration and Number of Participants With Drug Related Adverse Events (AEs).

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of GHRH (Growth Hormone-Releasing Hormone) and Macimorelin (non-peptide) in 101 people.

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

More context: the GHRH (Growth Hormone-Releasing Hormone) compound page and the Macimorelin (non-peptide) compound page, all human trials.