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Drug Trials Snapshots: Marcrilen
HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.
The information provided in Snapshots highlights who participated in the clinical trials that supported the FDA approval of this drug, and whether there were differences among sex, race and age groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.
LIMITATIONS OF THIS SNAPSHOT:
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to the MACRILEN Package Insert for complete information.
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your health provider about the risks and benefits of a drug. Refer to the MACRILEN Package Insert for complete information.
MACRILEN (macimorelin acetate)
ma-kri-len
Aeterna Zentaris GMBH
Approval date: December 20, 2017
ma-kri-len
Aeterna Zentaris GMBH
Approval date: December 20, 2017
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
MACRILEN is a drug for the diagnosis of growth hormone deficiency in adults.
How is this drug used?
MACRILEN is available as granules in a pouch. The granules are dissolved in water by a healthcare professional and given by mouth only once in preparation for a blood test that can help detect growth hormone deficiency. The amount given depends on the patient’s weight.
What are the benefits of this drug?
The results of MACRILEN test may show growth hormone deficiency.
Were there any differences in how well the drug worked in clinical trials among sex, race and age?
- Sex: MACRILEN worked similarly in men and women.
- Race: The majority of patients were White. The number of patients of other races were limited; therefore, differences in how the drug worked among other races could not be determined.
- Age: The majority of patients were younger than the age of 65; the number of patients older than 65 years was small; therefore, differences in how the drug worked in older patients could not be determined.
What are the possible side effects?
MACRILEN may cause heart rhythm problems due to change on electric heart beat tracing (ECG) called QTc prolongation and should be avoided in patients receiving other drugs that also cause QTc prolongation.
The most common side effects are change in the sense of taste, headache, diarrhea, nausea and fatigue.
Were there any differences in side effects among sex, race and age?
- Sex: Overall, the risk of side effects was similar in men and women.
- Race: The majority of patients were White. The number of patients in other races was small. Therefore, differences in side effects in other races could not be determined.
- Age: The majority of patients were younger than 65 years of age. The number of patients older than 65 years of age was small. Therefore, differences in side effects in patients older than 65 years of age could not be determined.
WHO WAS IN THE CLINICAL TRIALS?
Who participated in the clinical trials?
The FDA approved MACRILEN based on evidence from a clinical trial (NCT02558829) in 157 participants, some were patients suspected of having growth hormone deficiency and some were healthy volunteers. The trial was conducted at 30 sites in 9 countries: 25 sites were in Europe (Austria, Germany, Spain, France, Italy, Poland, Serbia and the UK) and 5 sites were in the United States.
Figure 1 summarizes how many men and women were in the clinical trial.
Figure 1. Baseline Demographics by Sex – Safety Population
Clinical Trial Data
Figure 2 summarizes the percentage of participants by race enrolled in the clinical trial.
Figure 2. Baseline Demographics by Race – Safety Population
Clinical Trial Data
Table 1. Demographics by Race – Safety Population
| N of Participants | Percentage | |
|---|---|---|
| White | 135 | 86 |
| Black/African American | 3 | 2 |
| Asian | 5 | 3 |
| Other and Unknown | 14 | 9 |
Clinical Trial Data
Figure 3. Baseline Demographics by Age – Safety Population
Clinical Trial Data
How were the trials designed?
Trial participants were patients with suspected growth hormone deficiency as well as healthy volunteers. Trial participants were given MACRILEN by mouth once. Blood levels of growth hormone were measured before and up to 90 minutes after receiving MACRILEN. At a different time, the same participants were tested for growth hormone deficiency using a commonly used test (insulin tolerance test) where growth hormone levels are also measured before and after the test.
The benefit of MACRILEN in detecting hormone deficiency was assessed through comparison between the two tests.
GLOSSARY
CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.
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