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

This study is currently recruiting participants.
Verified May 2017 by Garcia, Jose M., MD, PhD
Sponsor:
ClinicalTrials.gov Identifier:
NCT01614990
First Posted: June 8, 2012
Last Update Posted: May 4, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborators:
VA Office of Research and Development
Baylor College of Medicine
AEterna Zentaris
Information provided by (Responsible Party):
Garcia, Jose M., MD, PhD
  Purpose
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.

Condition Intervention Phase
Cancer Cachexia Drug: Macimorelin Drug: Placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: Pilot Clinical Trial of Repeated Doses of Macimorelin to Assess Safety and Efficacy in Patients With Cancer Cachexia

Further study details as provided by Garcia, Jose M., MD, PhD:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Estimated Enrollment: 8
Study Start Date: May 2012
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Macimorelin Drug: Macimorelin
Subjects will receive macimorelin (1 mg/kg) and matching placebo (Powerade®) daily for 7 days.
Other Name: AEZS-130
Placebo Comparator: Placebo Drug: Placebo
placebo (Powerade®) daily for 7 days.
Other Name: Powerade®

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01614990


Locations
United States, Washington
Veterans Affairs Puget Sound Health Care System Recruiting
Seattle, Washington, United States, 98108
Contact: Jose M Garcia, MD, PhD    206-764-2984    jg77@uw.edu   
Contact: Lindsey J Anderson, PhD    206-277-6719 ext 6719    lindsey.anderson@va.gov   
Principal Investigator: Jose M Garcia, MD, PhD         
Sponsors and Collaborators
Garcia, Jose M., MD, PhD
VA Office of Research and Development
Baylor College of Medicine
AEterna Zentaris
Investigators
Principal Investigator: Jose M Garcia, MD, PhD University of Washington and Veterans Affairs Puget Sound Health Care System
  More Information

Responsible Party: Garcia, Jose M., MD, PhD
ClinicalTrials.gov Identifier: NCT01614990     History of Changes
Other Study ID Numbers: 00954
First Submitted: June 5, 2012
First Posted: June 8, 2012
Last Update Posted: May 4, 2017
Last Verified: May 2017

Additional relevant MeSH terms:
Cachexia
Wasting Syndrome
Emaciation
Weight Loss
Body Weight Changes
Body Weight
Signs and Symptoms
Metabolic Diseases
Nutrition Disorders