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Cachexia; CancerPhase 3NCT03743064

A Study to Evaluate the Efficacy and Safety of Anamorelin HCl for the Treatment of Malignancy Associated Weight Loss and Anorexia in Adult Patients With Advanced Non-Small Cell Lung Cancer (NSCLC) - 2

A Phase 3 study testing anamorelin HCl in people with Cachexia; Cancer and Non Small Cell Lung Cancer, run by Helsinn Healthcare SA at 65 sites in 8 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

The goal of this clinical trial was to compare the efficacy and safety of anamorelin HCl (the investigational drug) to that of placebo (tablet with no drug) in patients with advanced non-small cell lung cancer and cachexia (cancer-related weight loss). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Cachexia; Cancer, Non Small Cell Lung Cancer
What is being tested?
anamorelin HCl
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
318 participants
Where
65 sitesin 8 countries
Study dates
6 May 2019to 27 Dec 2022
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Signed written informed consent
  • Female or male ≥18 years of age
  • Documented histologic or cytologic diagnosis of American Joint Committee on Cancer (AJCC) Stage III or IV NSCLC. Stage III patient must have unresectable disease
  • Body mass index < 20 kg/m2 with involuntary weight loss of >2% within 6 months prior to screening
  • Ongoing problems with appetite/eating associated with the underlying cancer, as determined by having score of ≤ 17 points on the 5-item Anorexia Symptom Scale and ≤ 37 points on the 12-item FAACT A/CS
  • Patient receiving or not receiving systemic anti-cancer treatment at the time of screening are eligible to participate. Systemic anti-cancer treatment includes first, second, third treatment line with chemotherapy/radiation therapy, immunotherapy or targeted therapy.
Show all 18Show fewer
  • Patient not receiving systemic anti-cancer treatment is eligible if:
  • Not planning to receive anti-cancer treatment and/or at least 14 days must be elapsed from the completion of prior treatment at the day of screening, in case underwent previous cycle OR
  • Planning to receive anti-cancer treatment within 14 days from randomization and/or at least 14 days must be elapsed from the completion of prior treatment at the day of screening, in case underwent previous cycle OR
  • Patient on palliative care treatment
  • ECOG performance status 0,1 or 2 at screening
  • AST (SGOT) and ALT (SGPT) ≤ 3 x ULN or if hepatic metastases are present ≤ 5 x ULN
  • Adequate renal function, defined as creatinine ≤2 ULN, or calculated creatinine clearance >30 ml/minute
  • Female patient shall be: a) of non-childbearing potential or b) of childbearing potential using reliable contraceptive measures and having a negative urine pregnancy test within 24 hours prior to first dose of investigational product.
  • Notes:
  • Female patient of non-childbearing potential are defined as being in post-menopausal state since at least 1 year; or having documented surgical sterilization or hysterectomy at least 3 months before study participation.
  • Reliable contraceptive measures include implants, injectables, combined oral contraceptives, intrauterine devices, vasectomized partner or complete (long term) sexual abstinence.
  • The patient must be willing and able to comply with the protocol tests and procedures All inclusion criteria will be checked at screening visit (Visit 1).

Who cannot join

  • Patient with other forms of lung cancer (e.g., small cell, neuroendocrine tumors)
  • Woman who is pregnant or breast-feeding
  • Reversible causes of reduced food intake, as determined by the Investigator. These causes may include but are not limited to:
  • NCI CTCAE Grade 3 or 4 oral mucositis,
  • NCI CTCAE Grade 3 or 4 GI disorders [nausea, vomiting, diarrhea, and constipation],
  • mechanical obstructions making patient unable to eat, or
Show all 34Show fewer
  • severe depression
  • Patient undergoing major surgery (central venous access placement and tumor biopsies are not considered major surgery) within 4 weeks prior to randomization. Patient must be well recovered from acute effects of surgery prior to screening. Patient should not have plans to undergo major surgical procedures during the treatment period
  • Patient currently taking androgenic compounds (including but not limited to testosterone, testosterone-like agents, oxandrolone, megestrol acetate, corticosteroids, olanzapine, mirtazapine (however, long-term use of mirtazapine for depression for at least four weeks prior to screening is allowed), dronabinol or marijuana (cannabis) or any other prescription medication or off-label products intended to increase appetite or treat unintentional weight loss
  • Patient with pleural effusion requiring thoracentesis, pericardial effusion requiring drainage, edema or evidence of ascites
  • Patient with uncontrolled or significant cardiovascular disease, including:
  • History of myocardial infarction within the past 3 months
  • A-V block of second or third degree (may be eligible if currently have a pacemaker)
  • Unstable angina
  • Congestive heart failure within the past 3 months, if defined as NYHA class III-IV
  • Any history of clinically significant ventricular arrhythmias (such as ventricular tachycardia, ventricular fibrillation, Wolff-Parkinson-White (WPW) syndrome, or torsade de pointes)
  • Uncontrolled hypertension (blood pressure >150 mm Hg systolic and >95 mm Hg diastolic)
  • Heart rate < 50 beats per minute on pre-entry electrocardiogram and patient is symptomatic
  • Patient on drugs that may prolong the PR or QRS interval durations, such as any of the antiarrhythmic medications Class I (Fast sodium (Na) channel blockers)
  • Patient unable to readily swallow oral tablets
  • Patient with severe gastrointestinal disease (including esophagitis, gastritis, malabsorption)
  • Patient with history of gastrectomy
  • Patient with uncontrolled diabetes mellitus or unmonitored diabetes mellitus
  • Patient with cachexia caused by other reasons, as determined by the investigator such as:
  • Severe COPD requiring use of home O2,
  • New York Heart Association (NYHA) class III-IV heart failure
  • AIDS
  • Uncontrolled thyroid disease
  • Patient receiving strong CYP3A4 inhibitors within 14 days of randomization
  • Patient currently receiving tube feedings or parenteral nutrition (either total or partial).
  • Current excessive alcohol or illicit drug use
  • Any condition, including the presence of laboratory abnormalities, which in the Investigator's opinion, places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
  • Enrollment in a previous study with anamorelin HCl
  • Patient actively receiving a concurrent investigational agent, or having received an investigational agent within 28 days of Day 1 All exclusion criteria will be checked at screening visit (Visit 1).

Trial locations and contacts

65 sites in 8 countries.

65 sites in 8 countries

United States19 sites

  • Pacific Cancer Medical Center, Inc

    Anaheim, California, 92801

  • CBCC Global Research Inc

    Bakersfield, California, 93309

  • New Jersey Hematology Oncology Associates Inc

    Brick, New Jersey, 08724

  • Duke Cancer Center

    Durham, North Carolina, 27710

  • Hunterdon Hematology Oncology LLC

    Flemington, New Jersey, 08822

  • Compassionate Care Research Group, Inc., at Compassionate Cancer Care Medical Group, Inc.

    Fountain Valley, California, 92708

  • Cancer and Hematology Centers of Western Michigan

    Grand Rapids, Michigan, 49503

  • Marin Cancer Care

    Greenbrae, California, 94904

  • The University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030-4009

  • Dartmouth-Hitchcock Medical Center

    Lebanon, New Hampshire, 03756

  • Hematology Oncology Center at Nyack Hospital

    Nyack, New York, 10960

  • University of Rochester, Medical Center

    Rochester, New York, 14642

  • Siouxland Regional Cancer Center dba June E.Nylen Cancer Center

    Sioux City, Iowa, 51101

  • Presence Infusion Care

    Skokie, Illinois, 60077

  • Toledo Clinic Cancer Center-Toledo

    Toledo, Ohio, 43623

  • The University of Arizona Cancer Center - North Campus

    Tucson, Arizona, 85719

  • Smilow Cancer Hospital at Yale-New Haven

    Waterbury, Connecticut, 06708

  • Cancer Center of Kansas

    Wichita, Kansas, 67214

  • Bond & Steele Clinic P.A.

    Winter Haven, Florida, 33881

Other trials in United States

Russia10 sites

  • Republican Clinical Oncology Center

    Kazan', 420029

  • Pyatigorsk Interdistric Oncology Center

    Pyatigorsk, 357502

  • Oncology Center of Moskovskiy District

    Saint Petersburg, 196247

  • AV Medical Group

    Saint Petersburg, 197082

  • Palliative Care Center Devita

    Saint Petersburg, 197183

  • City Clinical Oncology Center

    Saint Petersburg, 198255

  • Samara Regional Clinical Oncology Center

    Samara, 443031

  • Ogaryov Mordovia National Research State University, Republican Oncology Center

    Saransk, 430032

  • Oncology Center #2

    Sochi, 354057

  • Volgograd Regional Clinical Oncology Center

    Volgograd, 400138

Other trials in Russia

Ukraine8 sites

  • Medical Center "MEDICAL PLAZA" of the Limited Liability Company "EKODNIPRO"

    Dnipro, Ukraine, 49055

  • Publ Non- Profit Ent. under Kharkiv Reg. Council

    Kharkiv, Kharkiev Region, 61166

  • Communal Non-Profit Enterprise "Regional Center of Oncology"

    Kharkiv, Kharkiev, 61070

  • Medical Center "VERUM" Limited Liability Company

    Kyiv, Kyviv, 3039

  • Private Enterprise "First Private Clinic"

    Kyiv, Ukraine, 03037

  • Public Entreprise "Poltava Regional Clinical Oncology Center under Poltava Regional Council"

    Poltava, Poltava Oblast, 36011

  • Public Non-Profit Enterprise 'Ternopil Regional Clinical Oncology Center'' under Temopil Regional Council

    Ternopil, Ukraine, 46023

  • Medical Center of Limited Liability Company "ONCOLIFE"

    Zaporizhzhya, Ukraine, 69059

Other trials in Ukraine

Poland7 sites

  • Wladyslaw Bieganski Regional Specialist Hospital, Clinical Oncology Department

    Grudziądz, 86-300

  • "VEGAMED" Non-Public Healthcare Facility

    Katowice, 40-060

  • MSF Institute Ltd. Santa Familia Medical Institute

    Lodz, 90-302

  • MED - POLONIA Ltd.

    Poznan, 60-693

  • Specialist Hospital in Prabuty sp. z o.o. [limited liability company], Department of Pulmonology

    Prabuty, 82-550

  • Maria Skfodowska-Curie Institute of Oncology, Department of Lung and Thoracic Cancers

    Warsaw, 02-781

  • Mazovian Oncology Hospital, Oncology Outpatient Clinic

    Wieliszew, 05-135

Other trials in Poland

Romania7 sites

  • SC Oncopremium Team SRL, Medical Oncology Department

    Baia Mare, Maramureş, 430291

  • Alexandru Trestioreanu Institute of Oncology

    Bucharest, 022328

  • "Prof. Dr. Ion Chiricuta" Institute of Oncology, Medical Oncology Department

    Cluj-Napoca, Cluj, 400015

  • "Sf. Nectarie" Oncology Center, Medical Oncology Department

    Craiova, Dolj, 200347

  • Sf. Ioan cel Nou Country Emergency Hospital, Oncology Department

    Suceava, Suceava, 720237

  • Topmed Medical Center, Medical Oncology Department

    Târgu Mureş, Murers, 540156

  • S.C. Oncomed SRL, Medical Oncology Department

    Timișoara, Timiș County, 300239

Other trials in Romania

Australia6 sites

  • Flinders Medical Centre

    Bedford Park, 5042

  • Calvary Central Districts Hospital

    Elizabeth Vale, South Australia, 5112

  • St Vincent's Hospital Melbourne

    Fitzroy, 3065

  • Barwon Health, The McKellar Centre

    North Geelong, 3215

  • The Royal Melbourne Hospital

    Parkville, 3050

  • Gold Coast University Hospital

    Southport, 4215

Other trials in Australia

Belgium5 sites

  • Jules Bordet Institut

    Brussels, 1000

  • Saint Luc University Hospital

    Brussels, 1200

  • Charleroi Grand Hospital (GHDC)

    Charleroi, 6000

  • University Hospital Antwerp (UZA)

    Edegem, 2650

  • General Hospital Delta

    Roeselare, 8800

Other trials in Belgium

Croatia3 sites

  • General Hospital Pula

    Pula, 52 100

  • University Hospital Center Split

    Split, 21000

  • University Hospital Center Zagreb

    Zagreb, 10000

Other trials in Croatia

About this study

Official title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Anamorelin HCl for the Treatment of Malignancy Associated Weight Loss and Anorexia in Adult Patients With Advanced Non-Small Cell Lung Cancer (NSCLC)

The goal of this clinical trial was to compare the efficacy and safety of anamorelin HCl (the investigational drug) to that of placebo (tablet with no drug) in patients with advanced non-small cell lung cancer and cachexia (cancer-related weight loss). The main question it aimed to answer was as follows: Do patients who receive anamorelin HCl gain more body weight and show more improvement in anorexia symptoms than those who receive placebo.

Approximately 316 patients were to be enrolled in the study. Of these patients, an equal number were to be assigned to each treatment group (anamorelin HCl or placebo). Participants were to take their assigned study drug by mouth once daily for a total of 24 weeks. During this treatment period, the patients were to visit the clinical study site every 3 weeks for health and other study-related assessments. Two weeks after the last treatment, patients were to receive a follow-up phone call.

Read the detailed description

The study was a multicenter, randomized, double-blind, parallel-group, placebo-controlled study to evaluate the efficacy and safety of anamorelin HCl. It was planned that approximately 316 patients with advanced NSCLC with cachexia were to be randomized 1:1 to anamorelin HCl 100 mg or placebo (158 patients per treatment group). The study treatment was to be taken orally once daily for a total of 24 weeks. Patients were instructed to take the study drug at least 1 hour before their first meal of the day.

Central randomization was stratified by line of systemic anti-cancer treatment (first line vs second line vs third line or higher), by type of anti-cancer therapy (immunotherapy vs non-immunotherapy), and by baseline score of 5 IASS (≤10 vs >10). Patients who had never received anti-cancer treatment prior to entering the study but who met all eligibility criteria were eligible to enter the study and were assigned to receive first line treatment in the Interactive Web Response System (IWRS).

Patients were to visit the site every 3 weeks for the study Treatment Period of 24 weeks. A follow-up telephone visit was to be scheduled at Week 26. Thus, patients were enrolled in the study for a maximum duration of 27 weeks (including a 1-week Screening Period, a 24-week Treatment Period, and a 2-week Follow-up Period). Each patient was scheduled to have a total of 10 planned visits plus 1 telephone contact for the Follow-up Visit.

Being tested
anamorelin HCl, Placebo Oral Tablet
Medical subject headings
Cachexia, Neoplasms, Carcinoma, Non-Small-Cell Lung

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • anamorelin HClDrug

    100 mg anamorelin HCl (administered as 100 mg tablets in the fasted condition)

  • Placebo Oral TabletDrug

    Placebo (administered as matching placebo tablets in the fasted condition)

Study groups in NCT03743064
Study groupRoleWhat they receive
100 mg anamorelin HClExperimental100 mg anamorelin HCl (administered as film-coated tablets in fasted condition) was to be taken orally once daily for a total of 24 weeks
Receives: Drug: anamorelin HCl
PlaceboPlacebo comparatorPlacebo (administered as matching placebo tablets in fasted condition) was to be taken orally once daily for a total of 24 weeks
Receives: Drug: Placebo Oral Tablet

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Mean Change From Baseline in Body Weight Over 12 Weeks

    This co-primary efficacy endpoint was mean change from baseline in body weight (kg) over 12 weeks in the anamorelin HCl group versus placebo group. Mean change was computed as sum of the changes from baseline over 12 weeks by the time of the last assessment (either week 12 or before in case of death), and then divided by the number of assessments (observed or imputed) from baseline up to the time of the last assessment.

    Measured: Mean change from baseline over 12 weeks.

  2. Mean Change From Baseline in 5-item Anorexia Symptom Subscale (5-IASS) Over 12 Weeks

    This co-primary efficacy endpoint was mean change from baseline in 5-IASS (points) over 12 weeks in the anamorelin HCl group versus placebo group. Mean change was computed as sum of the changes from baseline over 12 weeks by the time of the last assessment (either week 12 or before in case of death), and then divided by the number of assessments (observed or imputed) from baseline up to the time of the last assessment.

    FAACT-A/CS (Functional Assessment Anorexia Cachexia Therapy) is a 12-item measure of patients' perceptions of anorexia/cachexia symptoms and concerns. From this questionnaire, the 5-item section referring to anorexia symptoms (i.e., "good appetite," "interest in food drops," "food tastes unpleasant," "get full quickly," and "difficulty eating rich/heavy foods") was used to assess 5-IASS. The range of possible scores is 0-20. Higher scores indicate lower levels of symptom burden.

    Measured: Mean change from baseline over 12 weeks.

Secondary outcomes (4)

  1. Duration in Treatment Benefit (Weeks) From Baseline Over 12 Weeks in Body Weight (≥0 kg)

    The duration of treatment benefit over 12 weeks was measured as the period, or the sum of the periods, over 12 weeks (or less in case of death), in which the patient observed a change from baseline in body weight of ≥0 kg.

    Measured: Duration of treatment benefit from baseline over 12 weeks.

  2. Duration in Treatment Benefit (Weeks) From Baseline Over 12 Weeks in Body Weight (≥1.5 kg)

    The duration of treatment benefit over 12 weeks was measured as the period, or the sum of the periods, over 12 weeks (or less in case of death), in which the patient observed a change from baseline in body weight of ≥1.5 kg.

    Measured: Duration of treatment benefit from baseline over 12 weeks.

  3. Duration of Treatment Benefit (Weeks) From Baseline Over 12 Weeks in 5-IASS (≥0 Points)

    The duration of treatment benefit over 12 weeks was measured as the period, or the sum of the periods, over 12 weeks (or less in case of death), in which the patient observed a change from baseline in 5- IASS of ≥0 points.

    Measured: Duration of treatment benefit from baseline over 12 weeks.

  4. Duration of Treatment Benefit (Weeks) From Baseline Over 12 Weeks in 5-IASS (≥3 Points)

    The duration of treatment benefit over 12 weeks was measured as the period, or the sum of the periods, over 12 weeks (or less in case of death), in which the patient observed a change from baseline in 5- IASS of ≥3 points.

    Measured: Duration of treatment benefit from baseline over 12 weeks.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Nov 2018
  2. Study start6 May 2019
  3. Primary completion27 Dec 2022
  4. Study completion27 Dec 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Randomized, Double-Blind, Placebo-Controlled, Multicenter Study
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
318 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03743064
Sponsor's protocol ID
ANAM-17-21
Other IDs
2018-002927-40, 2018
Status last verified
Jun 2024
First submitted
14 Nov 2018
Last updated
6 Oct 2026
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing anamorelin HCl in people with Cachexia; Cancer and Non Small Cell Lung Cancer, run by Helsinn Healthcare SA at 65 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 18 inclusion and 34 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 6 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 65 sites in United States, Russia, Ukraine, Poland, Romania and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 26 Jun 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Mean Change From Baseline in Body Weight Over 12 Weeks (time frame: Mean change from baseline over 12 weeks.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03743064.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2026.

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