Pilot Clinical Trial of Repeated Doses of Macimorelin to Assess Safety and Efficacy in Patients With Cancer Cachexia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Change of Insulin-like Growth Factor-1 (IGF-1) Plasma Levels
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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:
- •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.
研究组 & 干预措施
Macimorelin
干预措施: Macimorelin (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Change of Insulin-like Growth Factor-1 (IGF-1) Plasma Levels
时间窗: 7 days
The change of IGF-1 plasma levels will be measured between day 1 (prior to dosing) and day 7.
Change of Body Weight
时间窗: 7 days
The change of body weight(kg)will be measured between day 1 and day (Day 7-Day 1).
Change of Quality of Life Score
时间窗: 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.
次要结局
- Food Intake and Diary(7 days)
- Appetite (Visual Analog Scale [VAS] for Appetite)(7 days)
- Safety Laboratory (White Blood Cells)(Day 1 to Day 7)
- Safety Laboratory (Red Blood Cells)(Day 1 to Day 7)
- Safety Laboratory (Hemoglobin [HGB]; Mean Corpuscular Hemoglobin [MCH] Concentration; Protein; Total Albumin)(Day 1 to Day 7)
- Safety Laboratory (Potassium)(Day 1 to Day 7)
- Energy Expenditure as Measured by Indirect Calorimetry.(7 days)
- Change in Stair Climbing Power (SCP)(Day 1 to Day 7)
- Change in Respiratory Quotient(Day 1 to Day 7)
- Laboratory Assays (Growth Hormone)(Day 1 to Day 7)
- Handgrip Strength(7 days)
- Laboratory Assays(Day 1 to Day 7)
- ECG(Day 1 to Day 7)
- Number of Participants With Adverse Events as a Measure of Safety and Tolerability(7 days)
- Change in Percent Predicted Resting Energy Expenditure (REE)(Day 1 to Day 7)
- Laboratory Safety (Hematocrit [HCT]; Red Cell Distribution Width [RCDW]; Neutrophil; Lymphocyte; Monocyte; Eosinophil; Basophil)(Day 1 to Day 7)
- Safety Laboratory (Mean Corpuscular Volume [MCV])(Day 1 to Day 7)
- Safety Laboratory (Mean Corpuscular Hemoglobin [MCH])(Day 1 to Day 7)
- Safety Laboratory (Estimated Glomerular Filtration Rate [eGFR])(Day 1 to Day 7)
- Safety Laboratory (Platelet)(Day 1 to Day 7)
- Safety Laboratory {Blood Urea Nitrogen [BUN]; Creatinine; Calcium)(Day 1 to Day 7)
- Safety Laboratory (Alanine Transaminase [ALT]; Aspartate Aminotransferase [AST])(Day 1 to Day 7)
- Safety Laboratory (Sodium; Chloride; Carbon Dioxide)(Day 1 to Day 7)
- ECG (Heart Rate [HR])(Day 1 to Day 7)
- Safety Laboratory (Alkaline Phosphatase)(Day 1 to Day 7)