Anamorelin Study for Advanced Pancreatic Cancer

NCT04844970 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: LHMC 20193054
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief Summary

Multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of anamorelin HCl. Approximately 100 subjects with advanced PDAC and cachexia will be randomized 1:1 to anamorelin HCl 100 mg or placebo, taken orally once daily (QD) for a total of 25 weeks. Subjects will be instructed to take the study drug at least 1 hour before their first meal of the day

Conditions

Metastatic Pancreatic Cancer

Keywords

Phase II; Randomized; double-blind; placebo-controlled; Anamorelin HCI; Cachexia; Anorexia; Weight loss; FAACT A/CS

Outcome Measures

Primary Outcomes (1)

• Percent Weight Change From Baseline to Week 25

  Does anamorelin HCl dosed at 100mg per day vs. placebo demonstrate superiority on body weight gain and improvement in anorexia symptoms in patients undergoing first-line chemotherapy for incurable pancreatic cancer.

  25 weeks from baseline

Secondary Outcomes (7)

• Anorexia Questionnaire

  from baseline to week 13

• Survival

  25 weeks

• Radiologic Response to Chemotherapy

  from baseline to week 13

• Weight Gain

  from baseline to week 25 (end of the study)

• Fatigue Questionnaire

  from baseline to week 13

Other Outcomes (2)

• Unplanned Visits

  From baseline to week 25 (end of the study)

• Chemotherapy Dose Change

  from baseline to week 13

Study Arms (2)

Anamorelin

EXPERIMENTAL

Patients randomized to anamorelin HCL will take it daily for 24 weeks starting 3-5 days prior to chemotherapy

Drug: Anamorelin Hydrochloride

Placebo

PLACEBO COMPARATOR

Patients randomized to placebo will take it daily for 24 weeks starting 3-5 days prior to chemotherapy

Drug: Placebo

Interventions

Anamorelin Hydrochloride DRUG

Anamorelin HCl is an orally-active selective ghrelin receptor agonist which has shown anabolic and appetite-stimulating effects.

Anamorelin

Placebo DRUG

Anamorelin placebo

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed written informed consent
• Female or male ≥18 years of age
• Documented histologic or cytologic diagnosis of American Joint Committee on Cancer (AJCC) unresectable or metastatic pancreatic adenocarcinoma
• Body mass index \< 20 kg/m2 with involuntary weight loss or \>5% 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
• Subjects eligible to receive first line palliative chemotherapy
• ECOG performance status 0 or 1 at screening
• Acceptable hepatic function as defined by total bilirubin \< 1.6 mg/dl unless associated with Gilbert syndrome, then total bilirubin \< 2 x ULN. AST (SGOT) and ALT (SGPT) ≤ 2.5 x ULN or if hepatic metastases are present ≤ 5 x ULN
• Appropriate treatment with pancreatic enzyme replacement prior to trial initiation
• Female subjects shall be:
• of non-childbearing potential or
• of childbearing potential using reliable contraceptive measures and having a negative urine pregnancy test within 24 hours prior to first dose of investigational product.

You may not qualify if:

• Patient with other forms of pancreatic cancer (e.g. neuroendocrine tumors)
• Patient undergoing major surgery within 4 weeks of randomization or plans to undergo major surgery during study period.
• Women who are pregnant or breastfeeding
• Patient with alternative cause of cachexia as determined by the investigator including: a) severe COPD requiring O2, b) severe heart failure (NYHA Class III- IV), c) second malignancy
• Reversible causes of reduced food intake as determined by the investigator including but not limited to: severe mucositis (\>=NCI CTCAE grade 3), mechanical obstruction, severe nausea, vomiting, or diarrhea (\>=NCI CTCAE grade 3)
• Patient unable to swallow pills
• Patient with history of bariatric surgery, gastrectomy, or malabsorption disorder (gastritis, esophagitis)
• Patient with recent use of CYP3A4 inhibitors
• Patient with current daily use of therapies that may increase the QRS interval durations
• Patient currently taking medications/compounds intended to increase appetite or decrease weight loss (e.g. testosterone, megestrol acetate, cannabis products, methylphenidate, corticosteroids, olanzapine, mirtazapine (allowed if \>4 weeks of use as therapy for depression)
• Patient with current use of tube feeding or parenteral feeding
• 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)

Sponsors & Collaborators

• Lahey Clinic: lead
• Helsinn Healthcare SA: collaborator
• Quartesian LLC: collaborator

Study Sites (1)

Lahey Hospital & Medical Center

Burlington, Massachusetts, 01805, United States

Location

MeSH Terms

Conditions

Pancreatic Neoplasms; Cachexia; Anorexia; Weight Loss

Interventions

anamorelin

Condition Hierarchy (Ancestors)

Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Endocrine Gland Neoplasms; Digestive System Diseases; Pancreatic Diseases; Endocrine System Diseases; Body Weight Changes; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Thinness; Signs and Symptoms, Digestive

Limitations and Caveats

This study closed to accrual early, with the limited number of participants, we were unable to analyze the data.

Results Point of Contact

• Title: Julia Roache, Manger, Research Programs
• Organization: Lahey Hospital & Medical Center

julia.roache@lahey.org

7817443055

Study Officials

• Keith Stuart

  Lahey Hospital & Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: double-blind. Neither the investigator nor the participant would know the assigned drug/placebo
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: One group receives Anamorelin, and the other group receives placebo.

Study Record Dates

• First Submitted: March 9, 2021
• First Posted: April 14, 2021
• Study Start: April 1, 2023
• Primary Completion: November 7, 2023
• Study Completion: November 7, 2023
• Last Updated: November 17, 2025
• Results First Posted: November 17, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)