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

Garcia, Jose M., MD, PhD15 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Cancer Cachexia

Interventions

MacimorelinDRUG

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

PlaceboDRUG

placebo (Powerade®) daily for 7 days.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion Criteria: 1. Subjects ≥18 years of age with histological diagnosis of incurable cancer (solid tumor), 2. ECOG performance status of 0-2, 3. Presence of cancer-related cachexia defined as an involuntary weight loss of at least 5% of the pre-illness body weight over the previous 6 months, and 4. Provide written informed consent prior to screening. Exclusion Criteria: 1. Obesity (body weight \>140 Kg); 2. Recent active excessive alcohol or illicit drug use; 3. Severe depression as determined by the investigator; 4. Other causes of cachexia such as: Liver disease (AST or ALT \> 3x normal levels); renal failure (creatinine \>1.5 mg/dL), untreated thyroid disease, class III-IV CHF, AIDS, severe COPD requiring use of home O2; 5. Inability to increase food intake (e.g., esophageal obstruction, intractable nausea and vomiting); 6. Any condition that would prevent the subject from performing the research procedures (e.g. unstable coronary artery disease); 7. Use of growth hormone, megestrol, Marinol, or any other anabolic agents, appetite stimulants (including corticosteroids other than dexamethasone at the time of IV chemotherapy administrations), tube feeding, or parenteral nutrition during the 1 month prior to entering the study; 8. Recent administration (less than 1 week) of highly emetogenic chemotherapy (Hesketh scale class 4-5); subjects may otherwise be undergoing chemotherapy. 9. Being female and pregnant, breast-feeding or of childbearing potential. (Note: Lack of childbearing potential for female patients is satisfied by: a) being post menopausal; b) being surgically sterile; c) practicing contraception with an oral contraceptive, intra-uterine device, diaphragm, or condom with spermicide for the duration of the study; or d) being sexually inactive. Confirmation that the patient is not pregnant will be established by a negative serum hCG pregnancy test at the time of enrollment. 10. Co-administration of drugs that prolong QT interval, CYP3A4 inducers, QTc equal to or greater than 450ms at screening, or other investigational agents (a wash-out period of five times the half life of drugs that prolong QT will be allowed with approval of prescriber). 11. Conditions that would preclude from successfully scanning subjects in MRI: * Claustrophobia (this would make lying in the scanner very uncomfortable); b. having a pacemaker, aneurysm clips, neurostimulators, cochlear implants, metal in eyes, steel worker, or other implants; c. History of Seizures d. History of head injuries resulting in loss of consciousness \> 10 minutes.

Locations (1)

Veterans Affairs Puget Sound Health Care System

Seattle, Washington, United States

Outcomes

Primary Outcomes

Change of Body Weight

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

Time frame: 7 days

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

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

Time frame: 7 days

Change of Quality of Life Score

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.

Time frame: 7 days

Secondary Outcomes

Food Intake and Diary

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.

Time frame: 7 days

Appetite (Visual Analog Scale [VAS] for Appetite)

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.

Data from ClinicalTrials.gov

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