Cancer CachexiaPhase 2NCT01614990
Pilot Clinical Trial of Repeated Doses of Macimorelin to Assess Safety and Efficacy in Patients With Cancer Cachexia
A Phase 2 study testing Macimorelin in people with Cancer Cachexia, run by Garcia, Jose M., MD, PhD at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cancer Cachexia
- What is being tested?
- Macimorelin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- May 2012to Dec 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects ≥18 years of age with histological diagnosis of incurable cancer (solid tumor),
- ECOG performance status of 0-2,
- 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
- Provide written informed consent prior to screening.
Who cannot join
- Obesity (body weight >140 Kg);
- Recent active excessive alcohol or illicit drug use;
- Severe depression as determined by the investigator;
- 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;
- Inability to increase food intake (e.g., esophageal obstruction, intractable nausea and vomiting);
- Any condition that would prevent the subject from performing the research procedures (e.g. unstable coronary artery disease);
Show all 12Show fewer
- 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;
- Recent administration (less than 1 week) of highly emetogenic chemotherapy (Hesketh scale class 4-5); subjects may otherwise be undergoing chemotherapy.
- 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.
- 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).
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Veterans Affairs Puget Sound Health Care System
Seattle, Washington, 98108
About this study
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.
- Conditions
- Cancer Cachexia
- Being tested
- Macimorelin, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MacimorelinDrug
Also called AEZS-130
Subjects will receive macimorelin (1 mg/kg) and matching placebo (Powerade®) daily for 7 days.
PlaceboDrug
Also called Powerade®
placebo (Powerade®) daily for 7 days.
| Study group | Role | What they receive |
|---|---|---|
| Macimorelin | Active comparator | Not described Receives: Drug: Macimorelin |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of Body Weight
The change of body weight(kg)will be measured between day 1 and day (Day 7-Day 1).
Measured: 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.
Measured: 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.
Measured: 7 days
Secondary outcomes (25)
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.
Measured: 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.
Measured: 7 days
Handgrip Strength
Change in muscle strength as measured by handgrip strength.
Measured: 7 days
Energy Expenditure as Measured by Indirect Calorimetry.
Change in energy expenditure as measured by indirect calorimetry.
Measured: 7 days
Laboratory Assays
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.
Measured: Day 1 to Day 7
Safety Laboratory (White Blood Cells)
Change in Clinical laboratory parameters: complete blood count (CBC)
Measured: Day 1 to Day 7
ECG
Change in electrocardiogram (ECG) at days 1 and 7 before and 1 hour after dosing and on the post-study visit.
Measured: Day 1 to Day 7
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Recording of any adverse events from day 1 to day 7.
Measured: 7 days
Change in Stair Climbing Power (SCP)
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))
Measured: Day 1 to Day 7
Change in Percent Predicted Resting Energy Expenditure (REE)
Change in percent predicted (REE) as measured by indirect calorimetry.
Measured: Day 1 to Day 7
Change in Respiratory Quotient
Change in respiratory quotient (the ratio of volume CO2 released to volume of O2 utilized) as measured by indirect calorimetry.
Measured: Day 1 to Day 7
Laboratory Assays (Growth Hormone)
Change in Growth Hormone (GH) between day 1 and day 7.
Measured: Day 1 to Day 7
Safety Laboratory (Red Blood Cells)
Change in Clinical laboratory parameters: complete blood count (CBC)
Measured: Day 1 to Day 7
Safety Laboratory (Hemoglobin [HGB]; Mean Corpuscular Hemoglobin [MCH] Concentration; Protein; Total Albumin)
Change in Clinical laboratory parameters: complete blood count (CBC) and complete metabolic panel
Measured: Day 1 to Day 7
Laboratory Safety (Hematocrit [HCT]; Red Cell Distribution Width [RCDW]; Neutrophil; Lymphocyte; Monocyte; Eosinophil; Basophil)
Change in Clinical laboratory parameters: complete blood count (CBC)
Measured: Day 1 to Day 7
Safety Laboratory (Mean Corpuscular Volume [MCV])
Change in Clinical laboratory parameters: complete blood count (CBC)
Measured: Day 1 to Day 7
Safety Laboratory (Mean Corpuscular Hemoglobin [MCH])
Change in Clinical laboratory parameters: complete blood count (CBC)
Measured: Day 1 to Day 7
Safety Laboratory (Platelet)
Change in Clinical laboratory parameters: complete blood count (CBC)
Measured: Day 1 to Day 7
Safety Laboratory {Blood Urea Nitrogen [BUN]; Creatinine; Calcium)
Change in Clinical laboratory parameters: complete metabolic panel
Measured: Day 1 to Day 7
Safety Laboratory (Sodium; Chloride; Carbon Dioxide)
Change in Clinical laboratory parameters: complete metabolic panel
Measured: Day 1 to Day 7
Safety Laboratory (Potassium)
Change in Clinical laboratory parameters: complete metabolic panel
Measured: Day 1 to Day 7
Safety Laboratory (Alkaline Phosphatase)
Change in Clinical laboratory parameters: complete metabolic panel
Measured: Day 1 to Day 7
Safety Laboratory (Estimated Glomerular Filtration Rate [eGFR])
Change in Clinical laboratory parameters: complete metabolic panel
Measured: Day 1 to Day 7
Safety Laboratory (Alanine Transaminase [ALT]; Aspartate Aminotransferase [AST])
Change in Clinical laboratory parameters: complete metabolic panel
Measured: Day 1 to Day 7
ECG (Heart Rate [HR])
Change in electrocardiogram (ECG) at days 1 and 7 before and 1 hour after dosing and on the post-study visit.
Measured: Day 1 to Day 7
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2012 (estimated)
- Study startMay 2012
- Primary completionJul 2020
- Study completionDec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cancer CachexiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Garcia, Jose M., MD, PhD
- Collaborators
- VA Office of Research and Development, Baylor College of Medicine, AEterna Zentaris
- Responsible party
- Sponsor
- Investigators
- Jose M Garcia, MD, PhD, Principal investigator, University of Washington and Veterans Affairs Puget Sound Health Care System
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01614990
- Sponsor's protocol ID
- 00954
- Status last verified
- Mar 2024
- First submitted
- 5 Jun 2012
- Last updated
- 20 Mar 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Herodes M, Anderson LJ, Shober S, Schur EA, Graf SA, Ammer N, Salas R, Marcelli M, Garcia JM. Pilot clinical trial of macimorelin to assess safety and efficacy in patients with cancer cachexia. J Cachexia Sarcopenia Muscle. 2023 Apr;14(2):835-846. doi: 10.1002/jcsm.13191. Epub 2023 Mar 1.
PubMed 36860137Maldonado M, Molfese DL, Viswanath H, Curtis K, Jones A, Hayes TG, Marcelli M, Mediwala S, Baldwin P, Garcia JM, Salas R. The habenula as a novel link between the homeostatic and hedonic pathways in cancer-associated weight loss: a pilot study. J Cachexia Sarcopenia Muscle. 2018 Jun;9(3):497-504. doi: 10.1002/jcsm.12286. Epub 2018 Mar 25.
PubMed 29575771
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Clinical Trial of Repeated Doses of Macimorelin to Assess Safety and Efficacy in Patients With Cancer Cachexia
- ClinicalTrials.gov ID
- NCT01614990
- Acronym / sponsor ID
- 00954
- Conditions
- Cancer Cachexia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Garcia, Jose M., MD, PhD
- Collaborators
- VA Office of Research and Development, Baylor College of Medicine, AEterna Zentaris
- Study start
- May 2012
- Primary completion
- Jul 2020 (actual)
- Study completion
- Dec 2021 (actual)
- First posted
- 8 Jun 2012 (estimated)
- Results
- Posted 19 Mar 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Macimorelin | Active comparator | Drug: Macimorelin |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Subjects ≥18 years of age with histological diagnosis of incurable cancer (solid tumor),
- ECOG performance status of 0-2,
- 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
- Provide written informed consent prior to screening.
Key exclusion criteria (12 listed)
- Obesity (body weight >140 Kg);
- Recent active excessive alcohol or illicit drug use;
- Severe depression as determined by the investigator;
- 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;
- Inability to increase food intake (e.g., esophageal obstruction, intractable nausea and vomiting);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MacimorelinAlso: AEZS-130Subjects will receive macimorelin (1 mg/kg) and matching placebo (Powerade®) daily for 7 days. | Drug | Macimorelin |
| PlaceboAlso: Powerade®placebo (Powerade®) daily for 7 days. | Drug | Placebo |
Primary outcome
Change of Body Weight
Time frame: 7 days
The change of body weight(kg)will be measured between day 1 and day (Day 7-Day 1).
Change of Insulin-like Growth Factor-1 (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 [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.
25 secondary outcomes
Food Intake and Diary
Time frame: 7 days
Appetite (Visual Analog Scale [VAS] for Appetite)
Time frame: 7 days
Handgrip Strength
Time frame: 7 days
Energy Expenditure as Measured by Indirect Calorimetry.
Time frame: 7 days
Laboratory Assays
Time frame: Day 1 to Day 7
Safety Laboratory (White Blood Cells)
Time frame: Day 1 to Day 7
ECG
Time frame: Day 1 to Day 7
Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Time frame: 7 days
Change in Stair Climbing Power (SCP)
Time frame: Day 1 to Day 7
Change in Percent Predicted Resting Energy Expenditure (REE)
Time frame: Day 1 to Day 7
Change in Respiratory Quotient
Time frame: Day 1 to Day 7
Laboratory Assays (Growth Hormone)
Time frame: Day 1 to Day 7
Safety Laboratory (Red Blood Cells)
Time frame: Day 1 to Day 7
Safety Laboratory (Hemoglobin [HGB]; Mean Corpuscular Hemoglobin [MCH] Concentration; Protein; Total Albumin)
Time frame: Day 1 to Day 7
Laboratory Safety (Hematocrit [HCT]; Red Cell Distribution Width [RCDW]; Neutrophil; Lymphocyte; Monocyte; Eosinophil; Basophil)
Time frame: Day 1 to Day 7
Safety Laboratory (Mean Corpuscular Volume [MCV])
Time frame: Day 1 to Day 7
Safety Laboratory (Mean Corpuscular Hemoglobin [MCH])
Time frame: Day 1 to Day 7
Safety Laboratory (Platelet)
Time frame: Day 1 to Day 7
Safety Laboratory {Blood Urea Nitrogen [BUN]; Creatinine; Calcium)
Time frame: Day 1 to Day 7
Safety Laboratory (Sodium; Chloride; Carbon Dioxide)
Time frame: Day 1 to Day 7
Safety Laboratory (Potassium)
Time frame: Day 1 to Day 7
Safety Laboratory (Alkaline Phosphatase)
Time frame: Day 1 to Day 7
Safety Laboratory (Estimated Glomerular Filtration Rate [eGFR])
Time frame: Day 1 to Day 7
Safety Laboratory (Alanine Transaminase [ALT]; Aspartate Aminotransferase [AST])
Time frame: Day 1 to Day 7
ECG (Heart Rate [HR])
Time frame: Day 1 to Day 7
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 36860137Herodes M, Anderson LJ, Shober S, Schur EA, Graf SA, Ammer N, Salas R, Marcelli M, Garcia JM. Pilot clinical trial of macimorelin to assess safety and efficacy in patients with cancer cachexia. J Cachexia Sarcopenia Muscle. 2023 Apr;14(2):835-846. doi: 10.1002/jcsm.13191. Epub 2023 Mar 1.
- Derived from this study: PubMed 29575771Maldonado M, Molfese DL, Viswanath H, Curtis K, Jones A, Hayes TG, Marcelli M, Mediwala S, Baldwin P, Garcia JM, Salas R. The habenula as a novel link between the homeostatic and hedonic pathways in cancer-associated weight loss: a pilot study. J Cachexia Sarcopenia Muscle. 2018 Jun;9(3):497-504. doi: 10.1002/jcsm.12286. Epub 2018 Mar 25.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jose M Garcia, MD, PhD principal investigator
University of Washington and Veterans Affairs Puget Sound Health Care System
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Macimorelin in people with Cancer Cachexia, run by Garcia, Jose M., MD, PhD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Mar 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of Body Weight (time frame: 7 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01614990, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Mar 2024.