Muscle Wasting, Non-Small Cell Lung Cancer
Conditions
Brief summary
The purpose of this study is to determine if the investigational drug GTx-024 can help patients with non small cell lung cancer increase physical function and maintain or gain muscle.
Detailed description
This is a randomized, double blind, placebo controlled, multicenter, multinational efficacy and safety study in subjects with non small cell lung cancer. Subjects will be evenly randomized to placebo or GTx-024 prior to initiation of first line chemotherapy. The primary efficacy analysis will be based on total Lean Body Mass (LBM) and physical function.
Interventions
DRUGGTx-024
subjects will be randomized to receive GTx-024 3mg softgel for the duration of the trial.
DRUGplacebo
subject will receive matching placebo for the duration of the trial
Sponsors
GTx
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
30 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* give voluntary, signed informed consent in accordance with institutional policies * be non-obese as defined as body mass index (BMI)\< or = 32 and weight \< 300 pounds (\< 136 kg) * have been diagnosed with Stage III or IV NSCLC * be prior to first line chemotherapy * planned first line chemotherapy regimen is platinum plus gemcitabine only or platinum plus vinorelbine only or platinum plus pemetrexed only * if surgery is part of the cancer treatment, screening for this study should be conducted at least 4 weeks (28 days) after surgery * life expectancy of \> 6 months * ECOG score \< or = 1 * serum creatinine \< or = 2.0 mg/dL * MALES - age \> or = 30 years * \- FEMALES - age \>or=30 years and clinically confirmed as postmenopausal.Subjects must have undergone the onset of spontaneous or surgical menopause prior to the start of this study. Spontaneous menopause is defined as the natural cessation of ovarian function as indicated by being amenorrheic for at least 12 months. If the subject has been amenorrheic for \>or=6 months but \<12 months they must have a serum FSH concentration of \>or=50 mIU/mL and an estradiol concentration of \<or=25 pg/mL. Surgical menopause is defined as bilateral oophorectomy. * MALES - subjects must agree to use a double barrier method of contraception during the study and for 3 months after study completion. This may include the following: condom + spermicide or condom + oral hormonal contraception * MALES - have a serum PSA of \< or = 4.0 ng/mL or a negative prostate biopsy (no prostate cancer) within 6 months of evaluation
Exclusion criteria
* Have, in the judgment of the Investigator, a clinically significant concurrent illness or psychological, familial, sociological, geographical or other concomitant condition that would not permit adequate follow-up and compliance with the study protocol * Have ALT/SGOT or AST/SGPT above 1.5 times the upper limit of normal (ULN) without evidence of liver metastases and above 5 times the ULN in subjects with evidence of liver metastases * have alkaline phosphatase greater than 3 times ULN and/or total bilirubin levels above 2 mg/dL at baseline * have biologic agents or kinase inhibitors as part of their first line chemotherapy regimen including, but not limited to bevacizumab (Avastin), gefitinib (Nexavar) and erlotinib (Tarceva) * cardiovascular: uncontrolled hypertension, congestive heart failure or angina * Pulmonary: Stage 4 chronic obstructive pulmonary disease (COPD) * positive screen for Hepatitis B consisting of HBsAg (Hepatitis B Surface Antigen), unless subject was diagnosed \> 10 years prior to enrollment and no evidence of active liver disease * currently taking testosterone, oxandrolone (Oxandrin), testosterone-like agents (such as dehydroepiandrosterone (DHEA), androstenedione, and other androgenic compounds including herbals), or antiandrogens; previous therapy with testosterone and testosterone-like agents is acceptable with a 30 day washout (if previous testosterone therapy was long term depot within the past 6 months, the site should contact the medical monitor for this study to determine appropriate washout period) * currently taking megestrol acetate (Megace), dronabinol (Marinol), medical marijuana (medical cannabis) or any prescription medication intended to increase appetite or treat unintentional weight loss * have a baseline stair climb time \>or=30 seconds (mean of two stair climbs) * have active cancer, other than NSCLC or non-melanoma carcinoma of the skin, within the previous two years.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Physical Function | Day 84 | Measure is the percentage of subjects at day 84 with stair climb power change \>=10% from their baseline value. |
| Lean Body Mass | Day 84 | Measure is the percentage of subjects at day 84 with lean body mass change \>=0% from their baseline value. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| GTx-024 subjects will be randomized to receive GTx-024 for the duration of the trail
GTx-024: subjects will be randomized to receive GTx-024 for the duration of the trial. | 159 |
| Placebo subjects will be randomized to receive placebo for the duration of the trial
placebo: subject will receive placebo for the duration of the trial | 161 |
| Total | 320 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 11 | 10 |
| Overall Study | Death | 19 | 18 |
| Overall Study | Lost to Follow-up | 3 | 0 |
| Overall Study | noncompliance | 1 | 2 |
| Overall Study | other-not provided | 9 | 4 |
| Overall Study | progressive disease | 11 | 8 |
| Overall Study | Protocol Violation | 0 | 1 |
| Overall Study | Withdrawal by Subject | 12 | 20 |
Baseline characteristics
| Characteristic | GTx-024 | Placebo | Total |
|---|---|---|---|
| Age, Continuous | 60.0 years | 62.0 years | 61.0 years |
| Sex: Female, Male Female | 45 Participants | 48 Participants | 93 Participants |
| Sex: Female, Male Male | 114 Participants | 113 Participants | 227 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 149 / 165 | 157 / 165 |
| serious Total, serious adverse events | 109 / 165 | 113 / 165 |
Outcome results
Primary
Lean Body Mass
Measure is the percentage of subjects at day 84 with lean body mass change \>=0% from their baseline value.
Time frame: Day 84
Population: Subjects included in the Full Analysis Set
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| GTx-024 | Lean Body Mass | 46.5 percentage of subjects |
| Placebo | Lean Body Mass | 37.9 percentage of subjects |
Primary
Physical Function
Measure is the percentage of subjects at day 84 with stair climb power change \>=10% from their baseline value.
Time frame: Day 84
Population: Subjects included in the Full Analysis Set
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| GTx-024 | Physical Function | 19.5 percentage of subjects |
| Placebo | Physical Function | 24.8 percentage of subjects |