Nutritional and Metabolic Diseases

All Conditions

Symptoms and General Pathology

Wasting Syndrome

Cachexia

Syndrome

Weight Loss

Body Weight

Body Weight Changes

Thinness

Metabolic Diseases

Nutrition Disorders

Subjects will receive macimorelin (1 mg/kg) and matching placebo (Powerade®) daily for 7 days.

Macimorelin

Placebo

Jose M Garcia, MD, PhD

The purpose of this study is to evaluate the safety and efficacy of repeated oral administration of macimorelin at different doses daily for 1 week for the treatment of cancer cachexia.

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

The change of body weight(kg)will be measured between day 1 and day (Day 7-Day 1).

The change of IGF-1 plasma levels will be measured between day 1 (prior to dosing) and day 7.

The change of quality of life score (Anderson Symptom Assessment Scale \[ASAS; absolute score\], Functional Assessment of Chronic Illness-Fatigue \[FACIT-F; total score\]) will be measured between day 1 and day 7. The ASAS is a validated measure of quality of life (QOL) associated with symptom clusters commonly seen in cancer patient populations. The absolute score ranges from 0-100 with higher scores indicative of worse cancer symptoms. The FACIT-F is a validated measure of QOL associated with fatigue related to chronic illness. To account for missing items, the FACIT-F total score (sum of subscales) was adjusted by calculating the percent total score of items completed with the following:

(FACIT-F Total Score)/(N items completed ×4 (maximum possible score for each item ) × 100% Scores range from 0 to 160 with higher scores indicative of greater fatigue. Tests consists of 5 subscales: Physical, Social/Family, Emotional, and Functional Well-Being, and Additional Concerns.

Food intake as measured by a food diary to be recorded for 3 days before days 1 and 7 and by a test meal done at screening and on day 7.

Change of appetite measured by a validated visual analogue scale between day 1 and day 7. The VAS is a one-item measure that quantifies subjective rating with a 100 mm line anchored at 0 mm with the words "Not at all" and at 100 mm with the word "Extremely" where the participant makes a mark on the line indicative of their current hunger level. Higher scores are indicative of greater perceived hunger.

Change in muscle strength as measured by handgrip strength.

Change in energy expenditure as measured by indirect calorimetry.

Change in Insulin-Like Growth Factor Binding Protein-3 (IGFBP-3), Highly Sensitive C-Reactive Protein (CRP), and glucose between day 1 and day 7.

Change in Clinical laboratory parameters: complete blood count (CBC)

Change in electrocardiogram (ECG) at days 1 and 7 before and 1 hour after dosing and on the post-study visit.

Recording of any adverse events from day 1 to day 7.

SCP was assessed on Day 1 \& 7. Participants were asked to climb a standard flight of hospital stairs (13 steps, 15.3 cm each) as quickly as possible, using the handrail if necessary. Two-three trials were attempted with one minute of rest in-between. The shortest completion time was transformed into power and used for analysis where:

W= (body mass (kg) × acceleration of gravity (9.81 m⁄s\^2 )× vertical distance (1.99 m))/(time (seconds))

Change in percent predicted (REE) as measured by indirect calorimetry.

Change in respiratory quotient (the ratio of volume CO2 released to volume of O2 utilized) as measured by indirect calorimetry.

Change in Growth Hormone (GH) between day 1 and day 7.

Change in Clinical laboratory parameters: complete blood count (CBC) and complete metabolic panel

Change in Clinical laboratory parameters: complete metabolic panel

VA Office of Research and Development

Baylor College of Medicine

AEterna Zentaris

Garcia, Jose M., MD, PhD

Macimorelin: Subjects will receive macimorelin (1 mg/kg) and matching placebo (Powerade®) daily for 7 days.

Placebo: placebo (Powerade®) daily for 7 days.

Total

Age, Categorical

Age, Continuous

Sex: Female, Male

White, non-Hispanic

White, Hispanic

Black

Race/Ethnicity, Customized

United States

Region of Enrollment

Height

Weight

IGF-1

The ASAS is a validated measure of quality of life (QOL) associated with symptom clusters commonly seen in cancer patient populations. The absolute score ranges from 0-100 with higher scores indicative of worse cancer symptoms.

Anderson Symptom Assessment Scale (ASAS; absolute score)

The FACIT-F is a validated measure of QOL associated with fatigue related to chronic illness. To account for missing items, the FACIT-F total score (sum of subscales) was adjusted by calculating the percent total score of items completed with the following:

(FACIT-F Total Score)/(N items completed ×4 (maximum possible score for each item ) × 100% Scores range from 0 to 160 with higher scores indicative of greater fatigue. Tests consists of 5 subscales: Physical, Social/Family, Emotional, and Functional Well-Being, and Additional Concerns.

Functional Assessment of Chronic Illness-Fatigue (FACIT-F; total score)

Jose Garcia, MD, PhD

One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy); one patient in macimorelin (0.5 mg/kg) discontinued the study after receiving two doses due to non-compliance. Final number included for analysis was Macimorelin, N=9 and Placebo N=4.

Change of Body Weight

One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy); one patient in macimorelin (0.5 mg/kg) discontinued the study after receiving two doses due to non-compliance. Final number included for analysis was Macimorelin, N=9 and Placebo N=4

Change of Insulin-like Growth Factor-1 (IGF-1) Plasma Levels

ASAS

FACIT-F

Change of Quality of Life Score

Test Meal Caloric Intake

Food Diary

Food Intake and Diary

Appetite (Visual Analog Scale [VAS] for Appetite)

Handgrip Strength

Energy Expenditure as Measured by Indirect Calorimetry.

IGFBP-3

hs-CRP

Glucose

Laboratory Assays

One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy). Final number included for analysis was Macimorelin, N=10 and Placebo N=4

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

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