Ruxolitinib for Cancer Cachexia

NCT04906746 | Active Not Recruiting Early Phase 1 DMC FDA Drug

• 1 other identifier: 2021-0475
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

To assess toxicity with use of Ruxolitinib in NSCLC cachexia patients; to associate levels of JAK/STAT signaling in blood, adipose, and muscle pre- and post-ruxolitinib treatment with changes in cachexia and anorexia.

Conditions

Stage IV Non-small Cell Lung Cancer; Cachexia

Keywords

Lung cancer; Anorexia; Metabolism; Adipose; Muscle wasting

Outcome Measures

Primary Outcomes (1)

• Number of non-small cell lung cancer cachexia patients with toxicity with the use of Ruxolitinib

  To assess toxicity with use of Ruxolitinib in NSCLC (non-small cell lung cancer) cachexia patients; to associate levels of JAK/STAT (Janus kinase-signal transducer and activator of transcription) signaling in blood, adipose, and muscle pre- and post-ruxolitinib treatment with changes in cachexia and anorexia. NCI's CTCAE v5.0 toxicity criteria will be used to measure Ruxolitinib-related side effects. Immunoblotting, FACS analyses, and other biologic metrics will be used to determine if Ruxolitinib can suppress cachexia-associated JAK/STAT activation in blood and peripheral tissues.

  3 months

Secondary Outcomes (7)

• Number of adverse events associated with ruxolitinib when administered to cancer cachexia patients

  3 months

• Number of participants with Objective Response (OR) as determined by RECIST criteria

  3 months

• Analysis providing any preliminary evidence that ruxolitinib improves quality of life (QoL) for cancer cachexia patients

  4 months

• Suppression of body weight loss in cancer cachexia patients

  4 months

• Analysis of any preliminary evidence suggesting that Ruxolitinib suppresses adipose and lean muscle loss in cancer cachexia patients.

  4 months

Study Arms (1)

Dosing Regimen for Ruxolitinib

EXPERIMENTAL

Level 0: Ruxolitinib 0 MG po bid for 1 month (Month 1) Level 1: Ruxolitinib 10 MG po bid for 1 month (Month 2) Level 2: Ruxolitinib 15 MG po bid for 1 month (Month 3)

Drug: Identify any dose-limiting toxicity (DLT) when ruxolitinib is administered to NSCLC cachexia patients.

Interventions

Identify any dose-limiting toxicity (DLT) when ruxolitinib is administered to NSCLC cachexia patients. DRUG

There will be intra-patient dose escalation of ruxolitinib in every patient enrolled on study.

Dosing Regimen for Ruxolitinib

Eligibility Criteria

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

You may qualify if:

• Male or female subjects at least 18 years of age;
• Ability to understand and the willingness to sign a written informed consent;
• Histological or biopsy proven Non-Small Cell Lung Cancer (squamous or non-squamous);
• ECOG performance status of 0-2;
• Patients with evidence of:
• cancer cachexia, defined by the International Cancer Cachexia Consensus Definition (\>5% weight loss over the preceding 6 months prior to diagnosis); OR
• Patients with evidence of cancer pre-cachexia, defined by the International Cancer Cachexia Consensus Definition (0 to \<=5% weight loss over the preceding 6 months prior to diagnosis);
• Any de novo stage IV NSCLC disease diagnosis as defined by AJCC 8th edition staging. Staged with PET/CT, MRI brain, or other acceptable staging tool; measurable disease as defined by RECIST 1.1;
• Adequate end-organ function, based on routine clinical and laboratory workup and institutional guidelines, as determined by oncology team offering patient standard of care therapy, including:
• ANC \>1,000 cells/µl, Platelets \> 100,000 cells/µl, Hemoglobin \> 10.0 g/dl;
• Serum creatinine ≤ 1.5 x ULN or calculated creatinine clearance ≥ 45 ml/min;
• Total bilirubin ≤ 1.5 x ULN (or direct bilirubin below the ULN), AST and ALT ≤ 2.5 x ULN;
• International normalized ratio (INR) (or prothrombin time (PT)) and activated partial thromboplastin time (aPTT) ≤ 1.5 x ULN unless participant is receiving anticoagulant therapy, if values are within the intended therapeutic range;
• Women of child-bearing potential (WOCBP) and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 90 days following completion of therapy. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately; A female of child-bearing potential is any woman (regardless of sexual orientation, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria: a. Has not undergone a hysterectomy or bilateral oophorectomy; or b. Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months);
• Male subjects who are surgically sterile or are using a medically acceptable form of contraception for 90 days following the completion of therapy;

You may not qualify if:

• Subjects with confirmed stage I-III NSCLC;
• Patients whose tumors have actionable mutations treatable with targeted therapies;
• Patients with no evidence of cancer cachexia, defined by the International Cancer Cachexia Consensus Definition (\>5% weight loss over the preceding 6 months prior to diagnosis); OR Patients with no evidence of cancer pre-cachexia, defined by the International Cancer Cachexia Consensus Definition (0 to \<=5% weight loss over the preceding 6 months prior to diagnosis);
• Active malignancy other than lung cancer that requires concurrent treatment other than hormonal therapy and is deemed by the treating physicians to be likely to affect the subject's survival duration;
• Subjects who have not recovered or have disease control from prior treatment-related to toxicities judged by treating physician;
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to ruxolitinib or other agents used in study;
• Uncontrolled intercurrent illness including, but not limited to, serious ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that, in the opinion of the investigator, would limit compliance with study requirements;
• Significant bacterial, fungal, parasitic, or viral infection requiring treatment;
• Previous treatment with a JAK inhibitor;
• Uncontrolled congestive heart failure (New York Heart Association Classification 3 or 4), angina, myocardial infarction, cerebrovascular accident, coronary/peripheral artery bypass graft surgery, transient ischemic attack, or pulmonary embolism within 3 months prior to initiation of ruxolitinib;
• Females who are pregnant, breast-feeding or plan to become pregnant;
• Participation in other clinical trials either to treat diagnosed lung or other cancers (patients on registry trials are eligible);
• Requirement for treatment with drugs that may, in the judgment of the treating investigator, create a risk for a precipitous change in patient's health;
• Any other conditions that, in the Investigator's opinion, might indicate the subject to be unsuitable for the study;
• Life expectancy of less than 6 months;

Sponsors & Collaborators

• Tu Dan: lead
• Incyte Corporation: collaborator

Study Sites (1)

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

MeSH Terms

Conditions

Carcinoma, Non-Small-Cell Lung; Cachexia; Lung Neoplasms; Anorexia; Muscular Atrophy

Condition Hierarchy (Ancestors)

Carcinoma, Bronchogenic; Bronchial Neoplasms; Respiratory Tract Neoplasms; Thoracic Neoplasms; Neoplasms by Site; Neoplasms; Lung Diseases; Respiratory Tract Diseases; Weight Loss; Body Weight Changes; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Thinness; Signs and Symptoms, Digestive; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Atrophy; Pathological Conditions, Anatomical

Study Officials

• Tu Dan, MD

  University of Texas Southwestern Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Model Details: Open label, single arm, pilot study, Intrapatient dose escalation

Study Record Dates

• First Submitted: May 13, 2021
• First Posted: May 28, 2021
• Study Start: February 23, 2022
• Primary Completion: September 27, 2024
• Study Completion: December 1, 2025
• Last Updated: July 28, 2025

Record last verified: 2025-07

Locations

US (1)