Safety and Efficacy of Anamorelin HCl in Patients With Non-Small Cell Lung Cancer-Cachexia (ROMANA 1)

Anamorelin HCl in the Treatment of Non-Small Cell Lung Cancer-Cachexia (NSCLC-C): A Randomized Double-Blind Placebo-Controlled Multicenter Phase III Study to Evaluate the Safety and Efficacy of Anamorelin HCl in Patients With NSCLC-C

The administration of Anamorelin in patients with Stage III-IV non-small cell lung cancer-cachexia (NSCLC-C) is expected to increase appetite, lean body mass, weight gain, and muscle strength.

This is a randomized, double-blind, placebo-controlled, multicenter study to assess the safety and efficacy of Anamorelin in patients with non-small cell lung cancer-cachexia (NSCLC-C). The primary efficacy analysis will include the treatment difference in the change in lean body mass and physical function. Pharmacokinetic (PK) samples will also be collected at Day 43 visit for population PK.

Inclusion Criteria:

• Documented diagnosis of unresectable Stage III or Stage IV NSCLC
• Patients may be receiving maintenance chemotherapy
• Patients planning to initiate a new chemotherapy and/or radiation therapy regimen may do so only within ± 14 days of randomization
• Patients may have completed a chemotherapy and/or radiation therapy and/or have no plan to initiate a new regimen within 12 weeks from randomization; at least 14 days must elapse from the completion of the chemotherapy and/or radiation therapy prior to randomization
• Involuntary weight loss of ≥5% body weight within 6 months prior to screening or a screening body mass index (BMI) <20 kg/m2
• Body mass index ≤30 kg/m2
• Life expectancy of >4 months at time of screening
• ECOG performance status ≤2
• Adequate hepatic function, defined as AST and ALT levels ≤5 x upper limit of normal
• Adequate renal function, defined as creatinine ≤2 x upper limit of normal, or calculated creatinine clearance >30 ml/minute
• Ability to understand and comply with the procedures for the HGS evaluation
• If a woman of childbearing potential or a fertile man, he/she must agree to use an effective form of contraception during the study and for 30 days following the last dose of study drug (an effective form of contraception is abstinence, a hormonal contraceptive, or a double-barrier method)
• Must be willing and able to give signed informed consent and, in the opinion of the Investigator, to comply with the protocol tests and procedures

Exclusion Criteria:

• Other forms of lung cancer (e.g., small cell, mesothelioma)
• Women who are pregnant or breast-feeding
• Known HIV, hepatitis (B&C), or active tuberculosis
• Had major surgery (central venous access placement and tumor biopsies are not considered major surgery) within 4 weeks prior to randomization; patients must be well recovered from acute effects of surgery prior to screening; patients should not have plans to undergo major surgical procedures during the treatment period
• Currently taking prescription medications intended to increase appetite or treat weight loss; these include, but are not limited to, testosterone, androgenic compounds, megestrol acetate, methylphenidate, and dronabinol
• Inability to readily swallow oral tablets; patients with severe gastrointestinal disease (including esophagitis, gastritis, malabsorption, or obstructive symptoms) or intractable or frequent vomiting are excluded
• Has an active, uncontrolled infection
• Has uncontrolled diabetes mellitus
• Has untreated clinically relevant hypothyroidism
• Has known or symptomatic brain metastases
• Receiving strong CYP3A4 inhibitors within 14 days of randomization
• Receiving tube feedings or parenteral nutrition (either total or partial); patients must have discontinued these treatments for at least 6 weeks prior to Day 1, and throughout the study duration
• Other clinical diagnosis, ongoing or intercurrent illness that in the Investigator's opinion would prevent the patient's participation
• Has had previous exposure to Anamorelin HCl
• Patients actively receiving a concurrent investigational agent