Trial · NCT01614990
Pilot Clinical Trial of Repeated Doses of Macimorelin to Assess Safety and Efficacy in Patients With Cancer Cachexia
NCT01614990, Phase 2, Completed, Garcia, Jose M., MD, PhD; results first posted 2024-03-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Pilot Clinical Trial of Repeated Doses of Macimorelin to Assess Safety and Efficacy in Patients With Cancer Cachexia. Trials, record NCT01614990. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01614990
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pilot Clinical Trial of Repeated Doses of Macimorelin to Assess Safety and Efficacy in Patients With Cancer Cachexia
- NCT number
- NCT01614990
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 15 participants, actual
- Lead sponsor
- Garcia, Jose M., MD, PhD
- Conditions
- Cancer Cachexia
- Interventions
- Macimorelin, Placebo
- First posted
- 2012-06-08
- Last updated
- 2024-03-20
- Results first posted
- 2024-03-19
Source ClinicalTrials.gov
Registered outcomes28
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change of Body Weight | 7 days | The change of body weight(kg)will be measured between day 1 and day (Day 7-Day 1). |
| Primary | Change of Insulin-like Growth Factor-1 (IGF-1) Plasma Levels | 7 days | The change of IGF-1 plasma levels will be measured between day 1 (prior to dosing) and day 7. |
| Primary | Change of Quality of Life Score | 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. |
| Secondary | Food Intake and Diary | 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. |
| Secondary | Appetite (Visual Analog Scale [VAS] for Appetite) | 7 days | 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. |
| Secondary | Handgrip Strength | 7 days | Change in muscle strength as measured by handgrip strength. |
| Secondary | Energy Expenditure as Measured by Indirect Calorimetry. | 7 days | Change in energy expenditure as measured by indirect calorimetry. |
| Secondary | Laboratory Assays | Day 1 to Day 7 | 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. |
| Secondary | Safety Laboratory (White Blood Cells) | Day 1 to Day 7 | Change in Clinical laboratory parameters: complete blood count (CBC) |
| Secondary | ECG | Day 1 to Day 7 | Change in electrocardiogram (ECG) at days 1 and 7 before and 1 hour after dosing and on the post-study visit. |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 registered outcomesOutcomes28
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change of Body Weight | 7 days | One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy); one patient in macimorelin (0.5 mg/kg) discontinued the study after receiving two doses due to non-compliance. Final number included for analysis was Macimorelin, N=9 and Placebo N=4. | kg | MEDIAN |
| PRIMARY | Change of Insulin-like Growth Factor-1 (IGF-1) Plasma Levels | 7 days | One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy); one patient in macimorelin (0.5 mg/kg) discontinued the study after receiving two doses due to non-compliance. Final number included for analysis was Macimorelin, N=9 and Placebo N=4 | ng/mL | MEDIAN |
| PRIMARY | Change of Quality of Life Score | 7 days | One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy); one patient in macimorelin (0.5 mg/kg) discontinued the study after receiving two doses due to non-compliance. Final number included for analysis was Macimorelin, N=9 and Placebo N=4 | score on a scale | MEDIAN |
| SECONDARY | Food Intake and Diary | 7 days | One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy); one patient in macimorelin (0.5 mg/kg) discontinued the study after receiving two doses due to non-compliance. Final number included for analysis was Macimorelin, N=9 and Placebo N=4 | kCals | MEDIAN |
| SECONDARY | Appetite (Visual Analog Scale [VAS] for Appetite) | 7 days | One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy); one patient in macimorelin (0.5 mg/kg) discontinued the study after receiving two doses due to non-compliance. Final number included for analysis was Macimorelin, N=9 and Placebo N=4 | units on a scale | MEDIAN |
| SECONDARY | Handgrip Strength | 7 days | One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy); one patient in macimorelin (0.5 mg/kg) discontinued the study after receiving two doses due to non-compliance. Final number included for analysis was Macimorelin, N=9 and Placebo N=4 | kg | MEDIAN |
| SECONDARY | Energy Expenditure as Measured by Indirect Calorimetry. | 7 days | One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy); one patient in macimorelin (0.5 mg/kg) discontinued the study after receiving two doses due to non-compliance. Final number included for analysis was Macimorelin, N=9 and Placebo N=4 | kcals | MEDIAN |
| SECONDARY | Laboratory Assays | Day 1 to Day 7 | One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy); one patient in macimorelin (0.5 mg/kg) discontinued the study after receiving two doses due to non-compliance. Final number included for analysis was Macimorelin, N=9 and Placebo N=4 | mcg/mL | MEDIAN |
| SECONDARY | Safety Laboratory (White Blood Cells) | Day 1 to Day 7 | One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy). Final number included for analysis was Macimorelin, N=10 and Placebo N=4 | 10^9 cells/L | MEDIAN |
| SECONDARY | ECG | Day 1 to Day 7 | One patient in placebo discontinued the study after one dose due to initiation of an exclusionary medication (QT-prolonging chemotherapy); one patient in macimorelin (0.5 mg/kg) discontinued the study after receiving two doses due to non-compliance. Final number included for analysis was Macimorelin, N=9 and Placebo N=4 | ms | MEDIAN |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 outcomesResults by group110
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change of Body Weight | Macimorelin | 0 | Standard Error 0.3 | kg | |
| Change of Body Weight | Placebo | 0.2 | Standard Error 0.2 | kg | |
| Change of Insulin-like Growth Factor-1 (IGF-1) Plasma Levels | Macimorelin | 9 | Standard Error 6.7 | ng/mL | |
| Change of Insulin-like Growth Factor-1 (IGF-1) Plasma Levels | Placebo | 23 | Standard Error 3.9 | ng/mL | |
| Change of Quality of Life Score | Macimorelin | ASAS | 13.5 | Standard Deviation 5.1 | score on a scale |
| Change of Quality of Life Score | Placebo | ASAS | 6.7 | Standard Deviation 7.8 | score on a scale |
| Change of Quality of Life Score | Macimorelin | FACIT-F | 0.9 | Standard Deviation 4.7 | score on a scale |
| Change of Quality of Life Score | Placebo | FACIT-F | 0.2 | Standard Deviation 5.3 | score on a scale |
| Food Intake and Diary | Macimorelin | Test Meal Caloric Intake | 59 | Standard Error 123 | kCals |
| Food Intake and Diary | Placebo | Test Meal Caloric Intake | -78 | Standard Error 130 | kCals |
1 to 10 of 110Source ClinicalTrials.gov
See all 110 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Macimorelin | 10 | |
| Overall Study | STARTED | Placebo | 5 | |
| Overall Study | COMPLETED | Macimorelin | 9 | |
| Overall Study | COMPLETED | Placebo | 4 | |
| Overall Study | NOT COMPLETED | Macimorelin | 1 | |
| Overall Study | NOT COMPLETED | Placebo | 1 |
Source ClinicalTrials.gov
Adverse events10
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Recurrent Abdominal Pain | Gastrointestinal disorders | Macimorelin | 1 | 10 | 1 |
| other | Recurrent Abdominal Pain | Gastrointestinal disorders | Placebo | 0 | 5 | 0 |
| other | Recurrent Nausea | Gastrointestinal disorders | Macimorelin | 1 | 10 | 1 |
| other | Recurrent Nausea | Gastrointestinal disorders | Placebo | 0 | 5 | 0 |
| other | Recurrent Herpes Breakout | Infections and infestations | Macimorelin | 1 | 10 | 1 |
| other | Recurrent Herpes Breakout | Infections and infestations | Placebo | 0 | 5 | 0 |
| other | Pedal Oedema | Musculoskeletal and connective tissue disorders | Macimorelin | 1 | 10 | 1 |
| other | Pedal Oedema | Musculoskeletal and connective tissue disorders | Placebo | 0 | 5 | 0 |
| serious | Forearm Soft Tissue Necrosis | Infections and infestations | Macimorelin | 1 | 10 | 1 |
| serious | Forearm Soft Tissue Necrosis | Infections and infestations | Placebo | 0 | 5 | 0 |
Source ClinicalTrials.gov
Adverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Macimorelin | 0 | 10 |
| Total, all-cause mortality | Placebo | 0 | 5 |
| Total, other adverse events | Macimorelin | 2 | 10 |
| Total, other adverse events | Placebo | 0 | 5 |
| Total, serious adverse events | Macimorelin | 1 | 10 |
| Total, serious adverse events | Placebo | 0 | 5 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Macimorelin vs Placebo: deaths from any cause | Trial comparisons |
| Macimorelin vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01614990
GET https://conductscience.org/api/trials/v1/NCT01614990/registered?page=2