Anamorelin HCl in the Treatment of Non-Small Cell Lung Cancer-Cachexia (NSCLC-C): An Extension Study (ROMANA 3)

This is a randomized, double-blind, parallel-group, placebo-controlled, extension study to assess the safety and efficacy of Anamorelin HCl in NSCLC-C patients.

• Anamorelin HCl (Drug)
• Placebo (Drug)

Primary outcomes

• Percentage of Participants With Treatment-emergent Adverse Events [Over the 12-week treatment period]: To Evaluate the Safety and Tolerability of Anamorelin HCl.

Secondary outcomes

• Change in Body Weight [Change in body weight from baseline of the original trial through Week 12 of this extension trial.]
• Change in Handgrip Strength of the Non-Dominant Hand [Change in HGS from baseline of the original trial through Week 12 of this extension trial.]
• Change in A/CS Domain Score [Change in FAACT A/CS Domain Score from baseline of the original trial through Week 12 of this extension trial]: Change in the Functional Assessment of Anorexia/Cachexia Treatment (FAACT) 12-item Additional Concerns Subscale (A/CS) domain score is a 12-item scale. Each item is answered on a 5-point Likert scale ranging from 0 (not at all) to 4 (very much). The 12-items are summed together to obtain the domain score. Note that negatively phrased questions are reverse scored so that higher scores always represent improvement/less symptom burden. The total possible score for the A/CS domain ranges from 0 (worst) to 48 (best).

Sex: All, ages: 18 Years to any

Inclusion Criteria:

* Has completed the Day 85 Visit in the original trial (Study HT-ANAM-301 or HT-ANAM-302) and is considered appropriate to continue to receive additional study drug; must start dosing on the extension study within 5 days of completing dosing on the original trial
* ECOG performance status ≤2
* Life expectancy of >4 months at time of screening
* If 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:

* Women who are pregnant or breast-feeding
* Had major surgery (central venous access placement and tumor biopsies are not considered major surgery) within 4 weeks prior to enrollment into the extension study; 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 known or symptomatic brain metastases
* Receiving strong CYP3A4 inhibitors
* 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
* Patients actively receiving a concurrent investigational agent, other than Anamorelin HCl

Participant flow

Outcome results

• Percentage of Participants With Treatment-emergent Adverse Events (Primary), Number, percentage of participants: Anamorelin HCl 52; Placebo 56
• Change in Body Weight (Secondary), Least squares mean, kg: Anamorelin HCl 3.06 +/- 0.631; Placebo 0.92 +/- 0.697
• Change in Handgrip Strength of the Non-Dominant Hand (Secondary), Least squares mean, kg: Anamorelin HCl -0.83 +/- 0.929; Placebo -0.55 +/- 1.036
• Change in A/CS Domain Score (Secondary), Least squares mean, scores on a scale: Anamorelin HCl 4.46 +/- 0.92; Placebo 3.24 +/- 1.01

Adverse events

Time frame: Adverse events that occurred during the clinical trial, commenced with the first dose of study drug through the 28 day post-treatment follow-up visit.

Serious adverse events by term

Other adverse events by term

• Results publication: Currow D, Temel JS, Abernethy A, Milanowski J, Friend J, Fearon KC. ROMANA 3: a phase 3 safety extension study of anamorelin in advanced non-small-cell lung cancer (NSCLC) patients with cachexia. Ann Oncol. 2017 Aug 1;28(8):1949-1956. doi: 10.1093/annonc/mdx192. PMID 28472437