Trial Outcomes & Findings for Anamorelin HCl in the Treatment of Non-Small Cell Lung Cancer-Cachexia (NSCLC-C): An Extension Study (ROMANA 3) (NCT NCT01395914)

NCT ID: NCT01395914

Last Updated: 2017-09-14

Results Overview

To Evaluate the Safety and Tolerability of Anamorelin HCl.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 513 participants
• Primary outcome timeframe: Over the 12-week treatment period
• Results posted on: 2017-09-14

Participant Flow

Patients who completed dosing in either of the original trials of anamorelin HCl in the treatment of NSCLC-C (HT-ANAM-301 or HT-ANAM-302) were able to enroll in this study and continue to receive the study drug to which they were assigned, either anamorelin HCl 100 mg or placebo QD for an additional 12 weeks.

The primary purpose of this extension study was to permit patients who completed dosing in the original 12-week trials to have the option of continuing to receive randomized study drug for an additional 12 weeks, to further evaluate the safety and tolerability of anamorelin HCl.

Participant milestones

Measure	Anamorelin HCl Active drug; 100 mg tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.	Placebo Placebo tablets identical in appearance to active tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.
Overall Study STARTED	345	168
Overall Study ITT Population	345	168
Overall Study Safety Population	343	167
Overall Study COMPLETED	273	131
Overall Study NOT COMPLETED	72	37

Reasons for withdrawal

Measure	Anamorelin HCl Active drug; 100 mg tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.	Placebo Placebo tablets identical in appearance to active tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.
Overall Study Unrelated to study drug AE	6	1
Overall Study Death	23	17
Overall Study Other	9	3
Overall Study Withdrawal by patient	27	14
Overall Study Study drug-related AE	1	0
Overall Study Lost to Follow-up	6	2

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Anamorelin HCl n=345 Participants Active drug; 100 mg tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.	Placebo n=168 Participants Placebo tablets identical in appearance to active tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.	Total n=513 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	236 Participants n=5 Participants	120 Participants n=7 Participants	356 Participants n=5 Participants
Age, Categorical >=65 years	109 Participants n=5 Participants	48 Participants n=7 Participants	157 Participants n=5 Participants
Sex: Female, Male Female	83 Participants n=5 Participants	43 Participants n=7 Participants	126 Participants n=5 Participants
Sex: Female, Male Male	262 Participants n=5 Participants	125 Participants n=7 Participants	387 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	344 Participants n=5 Participants	166 Participants n=7 Participants	510 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Region of Enrollment Poland	107 participants n=5 Participants	60 participants n=7 Participants	167 participants n=5 Participants
Region of Enrollment Slovenia	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Region of Enrollment Spain	5 participants n=5 Participants	2 participants n=7 Participants	7 participants n=5 Participants
Region of Enrollment Belgium	2 participants n=5 Participants	2 participants n=7 Participants	4 participants n=5 Participants
Region of Enrollment Czech Republic	11 participants n=5 Participants	3 participants n=7 Participants	14 participants n=5 Participants
Region of Enrollment France	1 participants n=5 Participants	2 participants n=7 Participants	3 participants n=5 Participants
Region of Enrollment Germany	3 participants n=5 Participants	0 participants n=7 Participants	3 participants n=5 Participants
Region of Enrollment Hungary	39 participants n=5 Participants	16 participants n=7 Participants	55 participants n=5 Participants
Region of Enrollment Italy	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Region of Enrollment Netherlands	1 participants n=5 Participants	0 participants n=7 Participants	1 participants n=5 Participants
Region of Enrollment Canada	3 participants n=5 Participants	1 participants n=7 Participants	4 participants n=5 Participants
Region of Enrollment United States	16 participants n=5 Participants	4 participants n=7 Participants	20 participants n=5 Participants
Region of Enrollment Belarus	8 participants n=5 Participants	6 participants n=7 Participants	14 participants n=5 Participants
Region of Enrollment Israel	4 participants n=5 Participants	2 participants n=7 Participants	6 participants n=5 Participants
Region of Enrollment Serbia	4 participants n=5 Participants	2 participants n=7 Participants	6 participants n=5 Participants
Region of Enrollment Ukraine	64 participants n=5 Participants	33 participants n=7 Participants	97 participants n=5 Participants
Region of Enrollment Russian Federation	69 participants n=5 Participants	31 participants n=7 Participants	100 participants n=5 Participants
Region of Enrollment Australia	7 participants n=5 Participants	3 participants n=7 Participants	10 participants n=5 Participants

PRIMARY outcome

Timeframe: Over the 12-week treatment period

Population: Safety Population, defined as patients who received any extension trial study drug.

To Evaluate the Safety and Tolerability of Anamorelin HCl.

Outcome measures

Measure	Anamorelin HCl n=343 Participants Active drug; 100 mg tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.	Placebo n=167 Participants Placebo tablets identical in appearance to active tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.
Percentage of Participants With Treatment-emergent Adverse Events	52 percentage of participants	56 percentage of participants

SECONDARY outcome

Timeframe: Change in body weight from baseline of the original trial through Week 12 of this extension trial.

Population: Intent-to-Treat Population

Outcome measures

Measure	Anamorelin HCl n=345 Participants Active drug; 100 mg tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.	Placebo n=168 Participants Placebo tablets identical in appearance to active tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.
Change in Body Weight	3.06 kg Standard Error 0.631	0.92 kg Standard Error 0.697

SECONDARY outcome

Timeframe: Change in HGS from baseline of the original trial through Week 12 of this extension trial.

Population: Intent-to-Treat Population

Outcome measures

Measure	Anamorelin HCl n=345 Participants Active drug; 100 mg tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.	Placebo n=168 Participants Placebo tablets identical in appearance to active tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.
Change in Handgrip Strength of the Non-Dominant Hand	-0.83 kg Standard Error 0.929	-0.55 kg Standard Error 1.036

SECONDARY outcome

Timeframe: 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).

Outcome measures

Measure	Anamorelin HCl n=345 Participants Active drug; 100 mg tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.	Placebo n=168 Participants Placebo tablets identical in appearance to active tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.
Change in A/CS Domain Score	4.46 scores on a scale Standard Error 0.92	3.24 scores on a scale Standard Error 1.01

Adverse Events

Anamorelin HCl

Serious events: 44 serious events

Other events: 94 other events

Deaths: 0 deaths

Placebo

Serious events: 21 serious events

Other events: 57 other events

Deaths: 0 deaths

Serious adverse events

Measure	Anamorelin HCl n=343 participants at risk Active drug; 100 mg tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.	Placebo n=167 participants at risk Placebo tablets identical in appearance to active tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.
Injury, poisoning and procedural complications Splenic rupture	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Colon cancer metastatic	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasm progression	4.7% 16/343 • Number of events 16 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	4.2% 7/167 • Number of events 7 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Tumour haemorrhage	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Cardiac disorders Angina unstable	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Cardiac disorders Cardiopulmonary failure	0.00% 0/343 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.60% 1/167 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Cardiac disorders Pericardial effusion	0.00% 0/343 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.60% 1/167 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Congenital, familial and genetic disorders Tracheo-oesophageal fistula	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.58% 2/343 • Number of events 2 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.60% 1/167 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.00% 0/343 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.60% 1/167 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.58% 2/343 • Number of events 2 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.60% 1/167 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Blood and lymphatic system disorders Anaemia	0.87% 3/343 • Number of events 3 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.60% 1/167 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Blood and lymphatic system disorders Febrile neutropenia	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.60% 1/167 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Blood and lymphatic system disorders Thrombocytopenia	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	1.2% 2/167 • Number of events 2 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Nervous system disorders Cerebrovascular accident	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Nervous system disorders Convulsion	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Nervous system disorders Nervous system disorder	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
General disorders Asthenia	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.60% 1/167 • Number of events 2 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
General disorders Death	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	1.8% 3/167 • Number of events 3 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
General disorders Oedema peripheral	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
General disorders Multi-organ failure	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
General disorders Sudden death	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Gastrointestinal disorders Duodenal ulcer	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Gastrointestinal disorders Gastric haemorrhage	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.60% 1/167 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Gastrointestinal disorders Oesophagitis	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Gastrointestinal disorders Vomiting	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Infections and infestations Lower respiratory tract infection	0.29% 1/343 • Number of events 1 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Infections and infestations Pneumonia	1.2% 4/343 • Number of events 4 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	0.00% 0/167 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.

Other adverse events

Measure	Anamorelin HCl n=343 participants at risk Active drug; 100 mg tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.	Placebo n=167 participants at risk Placebo tablets identical in appearance to active tablets; oral administration QD for 12 weeks, at least 1 hour before the first meal of the day.
Blood and lymphatic system disorders Anaemia	13.1% 45/343 • Number of events 56 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	15.0% 25/167 • Number of events 37 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Blood and lymphatic system disorders Neutropenia	5.2% 18/343 • Number of events 21 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	4.8% 8/167 • Number of events 8 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
General disorders Asthenia	5.5% 19/343 • Number of events 20 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	8.4% 14/167 • Number of events 14 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.
Metabolism and nutrition disorders Decreased appetite	3.5% 12/343 • Number of events 13 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.	6.0% 10/167 • Number of events 10 • 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. Adverse events that occurred following the signature of the informed consent, but prior to the first dose of study drug were not reported as adverse events in this trial. The adverse event reporting period also ended if the patient began an alternative therapy within 28 days of the last administration of study drug.

Additional Information

Richard K. Bourne, Ph.D.

Helsinn Therapeutics (US), Inc.

Phone: 732-603-2852

Email: richard.bourne@helsinn.com

Results disclosure agreements

• Principal investigator is a sponsor employee The Sponsor intends to publish the complete study results in a timely manner that is appropriate to the project. Separate publication of a portion of the study results by any PI is discouraged.
• Publication restrictions are in place

Restriction type: OTHER