Evidence map›Paper›PMID 36860137›Full record

Trial reportJournal of cachexia, sarcopenia and muscle2023

Pilot clinical trial of macimorelin to assess safety and efficacy in patients with cancer cachexia.

Megan Herodes, Lindsey J Anderson, Samuel Shober, Ellen A Schur, Solomon A Graf, Nicola Ammer, Ramiro Salas, Marco Marcelli, Jose M Garcia

Registry-linked trialAbstract readRandomized Controlled Trial
In one paragraph

Trial report in Journal of cachexia, sarcopenia and muscle, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01614990 (Pilot Clinical Trial of Repeated Doses of Macimorelin to Assess Safety and Efficacy in Patients With Cancer Cachexia), which is not on this map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT01614990 phase2completednot on this map

Pilot Clinical Trial of Repeated Doses of Macimorelin to Assess Safety and Efficacy in Patients With Cancer Cachexia

TypeinterventionalSponsorGarcia, Jose M., MD, PhDRan2012 to 2021Enrolled15ConditionsCancer CachexiaArmsMacimorelin, Placebo
3 · Its place in the literature

Who cites it

7 citing papers in PubMed.

  1. Trial
  2. The effects of tissue inflammation on cancer cachexia.Biochimica et biophysica acta. Molecular basis of disease · 2026
    Review
  3. Cancer cachexia: molecular basis and therapeutic advances.Signal transduction and targeted therapy · 2026
    Review
  4. Review
  5. Review
  6. Review
  7. Current Therapeutic Targets in Cancer Cachexia: A Pathophysiologic Approach.American Society of Clinical Oncology educational book. American Society of Clinical Oncology. Annual Meeting · 2023
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Megan HerodesDivision of Gerontology and Geriatric Medicine, School of Medicine, University of Washington, Seattle, WA, USA.ORCID 0000-0002-9844-0375
Lindsey J AndersonDivision of Gerontology and Geriatric Medicine, School of Medicine, University of Washington, Seattle, WA, USA.
Samuel ShoberDivision of Gerontology and Geriatric Medicine, School of Medicine, University of Washington, Seattle, WA, USA.
Ellen A SchurDivision of General Internal Medicine, Department of Medicine, University of Washington, Seattle, WA, USA.
Solomon A GrafHospital and Specialty Medicine, Veterans Affairs Puget Sound Health Care System, Seattle, WA, USA.
Nicola AmmerDepartment of Clinical Development, Aeterna Zentaris GmbH, Frankfurt, Germany.
Ramiro SalasDepartments of Psychiatry and Neuroscience, Baylor College of Medicine, Houston, TX, USA.
Marco MarcelliMedical Care Line, Michael E. DeBakey Veterans Affairs Medical Center; Department of Medicine, Endocrinology, Diabetes and Metabolism, Baylor College of Medicine, Houston, TX, USA.
Jose M GarciaDivision of Gerontology and Geriatric Medicine, School of Medicine, University of Washington, Seattle, WA, USA.ORCID 0000-0002-4245-1753

Funding

Vector and Transgenic Mouse CoreP30DK017047 · NIDDK · UNIVERSITY OF WASHINGTON · PI Julie Mathieu · 1986 to 2026
$41.4M
PILOT STUDY--CLINICAL NUTRITION RESEARCHP30DK035816 · NIDDK · UNIVERSITY OF WASHINGTON · PI Ellen A Schur · 1986 to 2026
$30.4M
Diabetes, Obesity and Metabolism Training ProgramT32DK007247 · NIDDK · UNIVERSITY OF WASHINGTON · PI GREGORY J MORTON, KRISTINA Marie UTZSCHNEIDER · 1986 to 2026
$10.0M
Improving cancer-related fatigue, sexual dysfunction and quality of life in older men with cancer and androgen deficiencyR01AG061558 · NIA · SEATTLE INST FOR BIOMEDICAL/CLINICAL RES · PI BASARIA, SHEHZAD, DEL FABBRO, EGIDIO · 2019 to 2025
$3.4M
Improving Patient-Important Outcomes with Testosterone Replacement in Hypogonadal Men with a Prior History of CancerR01CA239208 · NCI · SEATTLE INST FOR BIOMEDICAL/CLINICAL RES · PI BASARIA, SHEHZAD, GARCIA, JOSE M. · 2019 to 2025
$3.3M
Intramuscular Mechanisms of Cancer CachexiaR01AR067319 · NIAMS · UNIVERSITY OF TEXAS HLTH SCI CTR HOUSTON · PI LI, YI-PING · 2015 to 2019
$2.2M
Brain connectivity and genetics as predictors of opioid abuse treatment outcomesI01CX001937 · VA · MICHAEL E DEBAKEY VA MEDICAL CENTER · PI SALAS, RAMIRO · 2021 to 2025
–
The Role of Ghrelin and the GHSR-1a receptor in Sarcopenic ObesityI01BX002807 · VA · VA PUGET SOUND HEALTHCARE SYSTEM · PI GARCIA, JOSE M. · 2016 to 2025
–
Mechanisms of action of ghrelin in muscle and adipose tissue in cancer-related caI01BX000507 · VA · MICHAEL E DEBAKEY VA MEDICAL CENTER · PI GARCIA, JOSE M. · 2009 to 2013
–
BLRD VA I01 BX000507BLRD VA I01 BX002807CSRD VA I01 CX001937NCI NIH HHS R01 CA239208NIAMS NIH HHS R01 AR067319NIA NIH HHS R01 AG061558NIDDK NIH HHS P30 DK017047NIDDK NIH HHS P30 DK035816NIDDK NIH HHS T32 DK007247RRD VA IK2 RX003245
6 · The paper itself

Abstract

backgroundCancer cachexia is associated with reduced body weight, appetite and quality of life (QOL) with no approved treatments. Growth hormone secretagogues like macimorelin have potential to mitigate these effects.

methodsThis pilot study assessed the safety and efficacy of macimorelin for 1 week. Efficacy was defined a priori as 1-week change in body weight (≥0.8 kg), plasma insulin-like growth factor (IGF)-1 (≥50 ng/mL) or QOL (≥15%). Secondary outcomes included food intake, appetite, functional performance, energy expenditure and safety laboratory parameters. Patients with cancer cachexia were randomized to 0.5 or 1.0 mg/kg macimorelin or placebo; outcomes were assessed non-parametrically.

resultsParticipants receiving at least one of either macimorelin dose were combined (N = 10; 100% male; median age = 65.50 ± 2.12) and compared with placebo (N = 5; 80% male; median age = 68.00 ± 6.19). Efficacy criteria achieved: body weight (macimorelin N = 2; placebo N = 0; P = 0.92); IGF-1 (macimorelin N = 0; placebo N = 0); QOL by Anderson Symptom Assessment Scale (macimorelin N = 4; placebo N = 1; P = 1.00) or Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F; macimorelin N = 3; placebo N = 0; P = 0.50). No related serious or non-serious adverse events were reported. In macimorelin recipients, change in FACIT-F was directly associated with change in body weight (r = 0.92, P = 0.001), IGF-1 (r = 0.80, P = 0.01), and caloric intake (r = 0.83, P = 0.005), and inversely associated with change in energy expenditure (r = -0.67, P = 0.05).

conclusionsDaily oral macimorelin for 1 week was safe and numerically improved body weight and QOL in patients with cancer cachexia compared with placebo. Longer term administration should be evaluated for mitigation of cancer-induced reductions in body weight, appetite and QOL in larger studies.

Indexed as

CachexiaNeoplasmsAgedBody WeightFemaleHumansIndolesInsulin-Like Growth Factor IMaleMiddle AgedPilot ProjectsQuality of LifeTryptophanIndolesInsulin-Like Growth Factor ImacimorelinTryptophanappetitecancer cachexiaghrelin receptor agonistgrowth hormone secretagoguemacimorelin

Identifiers

PMID36860137
PMCPMC10067502

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.