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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

Brief Summary: 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.

Detailed Summary:
Sponsor: Garcia, Jose M., MD, PhD

Current Primary Outcome:

• Change of body weight [ Time Frame: 7 days ]
  The change of body weight(kg)will be measured between day 1 and day 7.
• Change of IGF-1 plasma levels [ Time Frame: 7 days ]
  The change of IGF-1 plasma levels will be measured between day 1 (prior to dosing) and day 7.
• Change of quality of life score [ Time Frame: 7 days ]
  The change of quality of life score (Anderson Symptom Assessment Scale, FACIT-F) will be measured between day 1 and day 7.

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Food Intake and Diary [ Time Frame: 7 days ]
  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.
• Appetite [ Time Frame: 7 days ]
  Change of appetite measured by a validated visual analogue scale between day 1 and day 7.
• Body Composition [ Time Frame: 7 days ]
  Body composition as measured by bio-impedance and dual-energy x-ray absorptiometry on days 1 and 7.
• Muscle strength [ Time Frame: 7 days ]
  Muscle strength as measured by handgrip strength and stair climbing power.
• Energy expenditure as measured by indirect calorimetry. [ Time Frame: 7 days ]
  Energy expenditure as measured by indirect calorimetry.
• Laboratory Assays [ Time Frame: 7 days ]
  Change in IGFBP-3, GH, CRP, IL-6, TNF-α and glucose between day 1 and day 7.
• fMRI [ Time Frame: 7 days ]
  Changes in reward from food as measured by brain functional Magnetic Resonance Imaging (fMRI).
• Functional Brain Connectivity [ Time Frame: 7 days ]
  Changes in functional brain connectivity as assessed by Resting State Functional Connectivity (RSFC) imaging and Diffusion Tensor Imaging (DTI).
• Safety Laboratory [ Time Frame: 7 days ]
  Clinical laboratory parameters: complete blood count (CBC), comprehensive metabolic panel (CMP), urinalysis (UA)
• Vital signs [ Time Frame: 7 days ]
• ECG [ Time Frame: 7 days ]
  Electrocardiogram (ECG) at days 1 and 7 before and 1 hour after dosing and on the post-study visit.
• Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 7 days ]
  Recording of any adverse events from day 1 to day 7.

Original Secondary Outcome:

• Food Intake and Diary [ Time Frame: 7 days ]
  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.
• Appetite [ Time Frame: 7 days ]
  Change of appetite measured by a validated visual analogue scale between day 1 and day 7.
• Body Composition [ Time Frame: 7 days ]
  Body composition as measured by bio-impedance on days 1 and 7.
• Muscle strength [ Time Frame: 7 days ]
  Muscle strength as measured by handgrip strength and stair climbing power.
• Energy expenditure as measured by indirect calorimetry. [ Time Frame: 7 days ]
  Energy expenditure as measured by indirect calorimetry.
• Laboratory Assays [ Time Frame: 7 days ]
  Change in IGFBP-3, GH, CRP, IL-6, TNF-α and glucose between day 1 and day 7.
• fMRI [ Time Frame: 7 days ]
  Changes in reward from food as measured by brain functional Magnetic Resonance Imaging (fMRI).
• Functional Brain Connectivity [ Time Frame: 7 days ]
  Changes in functional brain connectivity as assessed by Resting State Functional Connectivity (RSFC) imaging and Diffusion Tensor Imaging (DTI).
• Safety Laboratory [ Time Frame: 7 days ]
  Clinical laboratory parameters: complete blood count (CBC), comprehensive metabolic panel (CMP), urinalysis (UA)
• Vital signs [ Time Frame: 7 days ]
• ECG [ Time Frame: 7 days ]
  Electrocardiogram (ECG) at days 1 and 7 before and 1 hour after dosing and on the post-study visit.
• Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 7 days ]
  Recording of any adverse events from day 1 to day 7.

Information By: Garcia, Jose M., MD, PhD

Dates:
Date Received: June 5, 2012
Date Started: May 2012
Date Completion: December 2017
Last Updated: May 2, 2017
Last Verified: May 2017