Percentage of positive and negative agreement between Macrilen™ and ITT based on prespecified GH cut-point values
How was Macrilen™ studied?
Macrilen™ was studied in 140 adults in a head-to-head trial vs the insulin tolerance test (ITT).1,a,b
How was Macrilen™ studied?
Macrilen™ was studied in 140 adults in a head-to-head trial vs the insulin tolerance test (ITT).1,a,b
Co-primary endpoints
Positive agreement was the percentage of subjects who tested positive for AGHD with both the ITT and Macrilen™ tests.
Negative agreement was the percentage of subjects who tested negative for AGHD with both the ITT and Macrilen™ tests.
Study design
- Open label
- Multicenter
- Randomized
- Crossover
- Single dose
The prespecified cut-point value for a diagnosis of AGHD with Macrilen™ was a maximally stimulated serum GH level of <2.8 ng/mL for the 4 blood draws.1
The prespecified cut-point value for a diagnosis of AGHD with Macrilen™ was a maximally stimulated serum GH level of <2.8 ng/mL for the 4 blood draws.1
aEnrolled safety and mITT populations: Of 166 enrolled subjects (137 with suspected AGHD and 29 healthy subjects), 157 subjects received at least 1 dose of the study drug and formed the safety population. Of the 157 subjects, 17 did not fulfill the mITT criterion (randomized subjects in whom both tests of the crossover were evaluable). Of the 140 subjects, 1 showed no measurable macimorelin plasma level attributed to noncompliance or a dosing error, and this patient’s data was removed from the efficacy analysis.
bStudied in adult subjects with different pretest probabilities of GH deficiency (GHD) and in healthy control subjects.1
Who was tested for AGHD?
In the head-to-head trial versus ITT, adults were segmented into the following groups to compare levels of agreement between Macrilen™ test results and ITT results.1
Who was tested for AGHD?
In the head-to-head trial versus ITT, adults were segmented into the following groups to compare levels of agreement between Macrilen™ test results and ITT results.1
High likelihoodof GHD
(n=38)
- Structural hypothalamic or pituitary lesions and low insulin-like growth factor-1 (IGF-1)
- 3 or more pituitary hormone deficiencies and low IGF-1
- Childhood-onset GHD with structural lesions and low IGF-1
- Structural hypothalamic or pituitary lesions and low insulin-like growth factor-1 (IGF-1)
- 3 or more pituitary hormone deficiencies and low IGF-1
- Childhood-onset GHD with structural lesions and low IGF-1
Intermediate likelihoodof GHD
(n=37)
- Subjects who do not qualify for either high or low likelihood
- Subjects who do not qualify for either high or low likelihood
Low likelihoodof GHD
(n=40)
- Subjects with only 1 risk factor for GHD, such as
- History of distant traumatic brain injury
- Only 1 pituitary hormone deficiency with otherwise normal pituitary function
- Isolated idiopathic childhood-onset GHD without additional pituitary deficits
- Subjects with only 1 risk factor for GHD, such as
- History of distant traumatic brain injury
- Only 1 pituitary hormone deficiency with otherwise normal pituitary function
- Isolated idiopathic childhood-onset GHD without additional pituitary deficits
Healthycontrols
(n=25)
- Healthy subjects matching subjects in high-likelihood group by sex, age (±5 years), BMI (±2 kg/m2), and estrogen status (females only)
- Healthy subjects matching subjects in high-likelihood group by sex, age (±5 years), BMI (±2 kg/m2), and estrogen status (females only)
Overall diagnostic accuracy of Macrilen™
Correlation overall in those with a high likelihood of AGHD1,2
Overall diagnostic accuracy of Macrilen™
Correlation overall in those with a high likelihood of AGHD1,2
Type of correlation
All subjects
High likelihood of AGHD
Positive
74%
89%
Negative
94%
100%
Overall
84%
89%
Positive correlation, subjects with both a positive ITT and Macrilen™ test. Negative correlation, subjects with both a negative ITT and Macrilen™ test. All subjects included those with a high (n=38), intermediate (n=37), or low (n=40) likelihood of AGHD, as well as healthy controls (n=25).1
cDefined as neuroglycopenic signs and symptoms confirmed by glucose less than 40 mg/dL.
Macrilen™ results were reproducible in more than 91% of patients tested, with accuracy comparable to ITT.1
Macrilen™ results were reproducible in more than 91% of patients tested, with accuracy comparable to ITT.1
Demonstrated safety profile in a clinical trial1
Common adverse events (AEs) reported in the Phase III clinical trial in <5% of subjects1
Demonstrated safety profile in a clinical trial1
Common adverse events (AEs) reported in the Phase III clinical trial in <5% of subjects1
No AEs led to failure to complete the Macrilen™ test.1
No AEs led to failure to compile the Macrilen™ test.1
How to prepare for dosing
Review the step-by-step process for preparing and administering Macrilen™.
How to prepare for dosing
Review the step-by-step process for preparing and administering Macrilen™.
How to order
Learn how you can start ordering Macrilen™ for your patients.
How to order
Learn how you can start ordering Macrilen™ for your patients.