RANDOMIZED CLINICAL TRIAL OF THE NOVEL ORAL GHRELIN MIMETIC MACIMORELIN IN THE TREATMENT OF CANCER CACHEXIA: STUDY DESIGN AND PRELIMINARY RESULTS

Program: Abstracts - Orals, Featured Poster Presentations, and Posters
Session: MON 327-337-Neuroendocrine Tumors
Clinical
Monday, June 17, 2013: 1:45 PM-3:45 PM
Expo Halls ABC (Moscone Center)

Poster Board MON-327
Sumbul A Ali*1 and Jose Manuel Garcia2
1Baylor College of Medicine, Houston, TX, 2MEDVAMC/ Baylor Coll of Med, Houston, TX
Background: Cancer cachexia, defined as weight loss of ≥5% over a 6 month period in the absence of simple starvation and anorexia is associated with increased mortality and poor quality of life. However, there are currently no treatments available for this devastating complication of the disease. Ghrelin, an orexigenic hormone and its analogues have been shown to improve appetite and result in weight gain in cachectic patients in preliminary studies. The purpose of this study is to evaluate the short term safety and efficacy of the novel oral ghrelin mimetic macimorelin in treatment of cancer cachexia.

Study Design: This is a randomized placebo controlled double blind pilot clinical trial of macimorelin in patients with cancer cachexia.  Subjects with cancer cachexia and good functional status are randomized to receive macimorelin or placebo for 7 days. The primary objectives are to assess the safety and efficacy of macimorelin and its effect on weight, IGF-1 level and quality of life as assessed by validated questionnaires: ECOG, Karnofsky scale, Anderson Symptom Assessment Scale and Functional Assessment of Chronic Illness Therapy-Fatigue scores. Secondarily we are studying changes in IGFBP-3 and GH levels, food intake assessed by a food diary and a test meal, appetite gauged by visual analogue scale, body composition by bio-impedance, muscle strength by hand grip strength and stair climbing power, and changes in reward from food and functional brain connectivity measured by functional MRI between days 1 and 7. Safety parameters including laboratory analyses, EKG and adverse events are assessed at each visit.

Results: The trial is ongoing and 4 out of 18 subjects have completed the study to date. All are men, age 68±9 years old, BMI 22.8±3.4 kg/m2, with different cancers in advanced stages. All subjects have tolerated the study medication and procedures without any difficulty. No adverse effects have been reported and the safety labs and EKG measures have been uneventful. We are still blinded to the treatment allocation as per protocol. Unblinding will occur once the first 10 subjects have finished the study, anticipated to occur approximately in May 2013 and these results will be discussed at the meeting.

Conclusion: In summary, this randomized clinical trial of the oral ghrelin mimetic macimorelin in the setting of cancer cachexia will explore if macimorelin is effective and well tolerated in cachectic patients with advanced cancer.

Disclosure: JMG: Principal Investigator, Aeterna Zentaris, Principal Investigator, Helsinn Therapeutics. Nothing to Disclose: SAA

*Please take note of The Endocrine Society's News Embargo Policy at http://www.endo-society.org/endo2013/media.cfm

Sources of Research Support: Supported by the Michael E. DeBakey Veterans Affairs Medical Center in Houston, Texas, Dept. of Veterans Affairs (MERIT awards BX000507 and CX000174 and MEDVAMC Pilot Seed Grant) and Aeterna Zentaris Inc.
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