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| Name | Class |
|---|---|
| VA Office of Research and Development | FED |
| Baylor College of Medicine | OTHER |
| AEterna Zentaris | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Macimorelin | Active Comparator |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Macimorelin | Drug | Subjects will receive macimorelin (1 mg/kg) and matching placebo (Powerade®) daily for 7 days. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change of Body Weight | The change of body weight(kg)will be measured between day 1 and day (Day 7-Day 1). | 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. | 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. | 7 days |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 7 days |
| Appetite (Visual Analog Scale [VAS] for Appetite) |
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Inclusion Criteria:
Exclusion Criteria:
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);
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:
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| Name | Affiliation | Role |
|---|---|---|
| Jose M Garcia, MD, PhD | University of Washington and Veterans Affairs Puget Sound Health Care System | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Veterans Affairs Puget Sound Health Care System | Seattle | Washington | 98108 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36860137 | Result | 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. | |
| 29575771 | Derived | Maldonado 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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Trial was closed prior to target recruitment due to poor accrual and study relocation.
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| ID | Title | Description |
|---|---|---|
| FG000 | Macimorelin | Macimorelin: Subjects will receive macimorelin (1 mg/kg) and matching placebo (Powerade®) daily for 7 days. |
| FG001 | Placebo | Placebo: placebo (Powerade®) daily for 7 days. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Macimorelin | Macimorelin: Subjects will receive macimorelin (1 mg/kg) and matching placebo (Powerade®) daily for 7 days. |
| BG001 | Placebo | Placebo: placebo (Powerade®) daily for 7 days. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change of Body Weight | The change of body weight(kg)will be measured between day 1 and day (Day 7-Day 1). | 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. | Posted | Median | Standard Error | kg | 7 days |
|
From consenting to 16 days post last study visit (~23 days)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Macimorelin | Macimorelin: Subjects will receive macimorelin (1 mg/kg) and matching placebo (Powerade®) daily for 7 days. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Forearm Soft Tissue Necrosis | Infections and infestations | Non-systematic Assessment | Unrelated serious adverse event occurred in one patient who received 0.5 mg/kg macimorelin - hospitalized for a radiation-related infection following initiation of radiation treatment. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Recurrent Abdominal Pain | Gastrointestinal disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Jose Garcia, MD, PhD | Geriatric Research, Education and Clinical Center, VA Puget Sound Health Care System | (206) 764-2984 | jg77@uw.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Feb 6, 2018 | Mar 8, 2023 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| C582727 | macimorelin |
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| Placebo | Drug | placebo (Powerade®) daily for 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.
| 7 days |
| Handgrip Strength | Change in muscle strength as measured by handgrip strength. | 7 days |
| Energy Expenditure as Measured by Indirect Calorimetry. | Change in energy expenditure as measured by indirect calorimetry. | 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. | Day 1 to Day 7 |
| Safety Laboratory (White Blood Cells) | Change in Clinical laboratory parameters: complete blood count (CBC) | 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. | 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. | 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)) | Day 1 to Day 7 |
| Change in Percent Predicted Resting Energy Expenditure (REE) | Change in percent predicted (REE) as measured by indirect calorimetry. | 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. | Day 1 to Day 7 |
| Laboratory Assays (Growth Hormone) | Change in Growth Hormone (GH) between day 1 and day 7. | Day 1 to Day 7 |
| Safety Laboratory (Red Blood Cells) | Change in Clinical laboratory parameters: complete blood count (CBC) | 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 | 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) | Day 1 to Day 7 |
| Safety Laboratory (Mean Corpuscular Volume [MCV]) | Change in Clinical laboratory parameters: complete blood count (CBC) | Day 1 to Day 7 |
| Safety Laboratory (Mean Corpuscular Hemoglobin [MCH]) | Change in Clinical laboratory parameters: complete blood count (CBC) | Day 1 to Day 7 |
| Safety Laboratory (Platelet) | Change in Clinical laboratory parameters: complete blood count (CBC) | Day 1 to Day 7 |
| Safety Laboratory {Blood Urea Nitrogen [BUN]; Creatinine; Calcium) | Change in Clinical laboratory parameters: complete metabolic panel | Day 1 to Day 7 |
| Safety Laboratory (Sodium; Chloride; Carbon Dioxide) | Change in Clinical laboratory parameters: complete metabolic panel | Day 1 to Day 7 |
| Safety Laboratory (Potassium) | Change in Clinical laboratory parameters: complete metabolic panel | Day 1 to Day 7 |
| Safety Laboratory (Alkaline Phosphatase) | Change in Clinical laboratory parameters: complete metabolic panel | Day 1 to Day 7 |
| Safety Laboratory (Estimated Glomerular Filtration Rate [eGFR]) | Change in Clinical laboratory parameters: complete metabolic panel | Day 1 to Day 7 |
| Safety Laboratory (Alanine Transaminase [ALT]; Aspartate Aminotransferase [AST]) | Change in Clinical laboratory parameters: complete metabolic panel | 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. | Day 1 to Day 7 |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Median | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| BMI | Median | Standard Deviation | kg/m^2 |
|
| Height | Median | Standard Deviation | cm |
|
| Weight | Median | Standard Deviation | kg |
|
| IGF-1 | Median | Standard Deviation | ng/mL |
|
| Anderson Symptom Assessment Scale (ASAS; absolute score) | 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. | Median | Standard Deviation | units on a scale |
|
| Functional Assessment of Chronic Illness-Fatigue (FACIT-F; total score) | 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. | Median | Standard Deviation | units on a scale |
|
|
|
| Primary | 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. | 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 | Posted | Median | Standard Error | ng/mL | 7 days |
|
|
|
| Primary | 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. | 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 | Posted | Median | Standard Deviation | score on a scale | 7 days |
|
|
|
| Secondary | 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. | 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 | Posted | Median | Standard Error | kCals | 7 days |
|
|
|
| Secondary | 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. | 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 | Posted | Median | Standard Error | units on a scale | 7 days |
|
|
|
| Secondary | Handgrip Strength | Change in muscle strength as measured by handgrip strength. | 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 | Posted | Median | Standard Error | kg | 7 days |
|
|
|
| Secondary | Energy Expenditure as Measured by Indirect Calorimetry. | Change in energy expenditure as measured by indirect calorimetry. | 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 | Posted | Median | Standard Error | kcals | 7 days |
|
|
|
| Secondary | 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. | 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 | Posted | Median | Standard Error | mcg/mL | Day 1 to Day 7 |
|
|
|
| Secondary | Safety Laboratory (White Blood Cells) | Change in Clinical laboratory parameters: complete blood count (CBC) | 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 | Posted | Median | Standard Error | 10^9 cells/L | Day 1 to Day 7 |
|
|
|
| Secondary | ECG | Change in electrocardiogram (ECG) at days 1 and 7 before and 1 hour after dosing and on the post-study visit. | 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 | Posted | Median | Standard Error | ms | Day 1 to Day 7 |
|
|
|
| Secondary | Number of Participants With Adverse Events as a Measure of Safety and Tolerability | Recording of any adverse events from day 1 to day 7. | Posted | Count of Participants | Participants | 7 days |
|
|
|
| Secondary | 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)) | 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 | Posted | Median | Standard Error | W | Day 1 to Day 7 |
|
|
|
| Secondary | Change in Percent Predicted Resting Energy Expenditure (REE) | Change in percent predicted (REE) as measured by indirect calorimetry. | 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 | Posted | Median | Standard Error | % of predicted REE used | Day 1 to Day 7 |
|
|
|
| Secondary | Change in Respiratory Quotient | Change in respiratory quotient (the ratio of volume CO2 released to volume of O2 utilized) as measured by indirect calorimetry. | 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 | Posted | Median | Standard Error | Ratio | Day 1 to Day 7 |
|
|
|
| Secondary | Laboratory Assays (Growth Hormone) | Change in Growth Hormone (GH) between day 1 and 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 | Posted | Median | Standard Error | ng/mL | Day 1 to Day 7 |
|
|
|
| Secondary | Safety Laboratory (Red Blood Cells) | Change in Clinical laboratory parameters: complete blood count (CBC) | 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 | Posted | Median | Standard Error | 10^6 cells/mcL | Day 1 to Day 7 |
|
|
|
| Secondary | 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 | 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 | Posted | Median | Standard Error | g/dL | Day 1 to Day 7 |
|
|
|
| Secondary | Laboratory Safety (Hematocrit [HCT]; Red Cell Distribution Width [RCDW]; Neutrophil; Lymphocyte; Monocyte; Eosinophil; Basophil) | Change in Clinical laboratory parameters: complete blood count (CBC) | 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 | Posted | Median | Standard Error | % of cells | Day 1 to Day 7 |
|
|
|
| Secondary | Safety Laboratory (Mean Corpuscular Volume [MCV]) | Change in Clinical laboratory parameters: complete blood count (CBC) | 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 | Posted | Median | Standard Error | fl | Day 1 to Day 7 |
|
|
|
| Secondary | Safety Laboratory (Mean Corpuscular Hemoglobin [MCH]) | Change in Clinical laboratory parameters: complete blood count (CBC) | 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 | Posted | Median | Standard Error | pg | Day 1 to Day 7 |
|
|
|
| Secondary | Safety Laboratory (Platelet) | Change in Clinical laboratory parameters: complete blood count (CBC) | 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 | Posted | Median | Standard Error | 10^3 cells/mcL | Day 1 to Day 7 |
|
|
|
| Secondary | Safety Laboratory {Blood Urea Nitrogen [BUN]; Creatinine; Calcium) | Change in Clinical laboratory parameters: complete metabolic panel | 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 | Posted | Median | Standard Error | mg/dL | Day 1 to Day 7 |
|
|
|
| Secondary | Safety Laboratory (Sodium; Chloride; Carbon Dioxide) | Change in Clinical laboratory parameters: complete metabolic panel | 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 | Posted | Median | Standard Error | mEq/L | Day 1 to Day 7 |
|
|
|
| Secondary | Safety Laboratory (Potassium) | Change in Clinical laboratory parameters: complete metabolic panel | 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 | Posted | Median | Standard Error | mmol/L | Day 1 to Day 7 |
|
|
|
| Secondary | Safety Laboratory (Alkaline Phosphatase) | Change in Clinical laboratory parameters: complete metabolic panel | 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 | Posted | Median | Standard Error | IU/L | Day 1 to Day 7 |
|
|
|
| Secondary | Safety Laboratory (Estimated Glomerular Filtration Rate [eGFR]) | Change in Clinical laboratory parameters: complete metabolic panel | 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 | Posted | Median | Standard Error | mL/min/1.73 m^2 | Day 1 to Day 7 |
|
|
|
| Secondary | Safety Laboratory (Alanine Transaminase [ALT]; Aspartate Aminotransferase [AST]) | Change in Clinical laboratory parameters: complete metabolic panel | 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 | Posted | Median | Standard Error | U/L | Day 1 to Day 7 |
|
|
|
| Secondary | 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. | 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 | Posted | Median | Standard Error | bpm | Day 1 to Day 7 |
|
|
|
| 0 |
| 10 |
| 1 |
| 10 |
| 2 |
| 10 |
| EG001 | Placebo | Placebo: placebo (Powerade®) daily for 7 days. | 0 | 5 | 0 | 5 | 0 | 5 |
|
| Recurrent Nausea | Gastrointestinal disorders | Non-systematic Assessment |
|
| Recurrent Herpes Breakout | Infections and infestations | Non-systematic Assessment |
|
| Pedal Oedema | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
Not provided
Not provided
Not provided
| Glucose |
|
| Pre-Dose QT Interval (ms) |
|
| Pre-Dose QT Corrected for HR Interval (QTC; ms) |
|
| Post-Dose PR Interval (ms) |
|
| Post-Dose QRS Complex (ms) |
|
| Post-Dose QT Interval (ms) |
|
| Post-Dose QTC (ms) |
|
| Protein |
|
| Total Albumin |
|
| Neutrophil |
|
| Lymphocyte |
|
| Monocyte |
|
| Eosinophil |
|
| Basophil |
|
| Calcium |
|
| Carbon Dioxide |
|