Trial Outcomes & Findings for Study of GTx-024 on Muscle Wasting (Cachexia) Cancer. (NCT NCT00467844)
NCT ID: NCT00467844
Last Updated: 2014-07-11
Results Overview
Change in total body lean mass as measured by dual energy x-ray absorptiometry (DEXA)from baseline to 4 months.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
159 participants
Primary outcome timeframe
Baseline to Four Months
Results posted on
2014-07-11
Participant Flow
Participant milestones
| Measure |
GTx -024
1 mg
|
GTx-024
3 mg
|
3 mg of Placebo
Placebo
|
|---|---|---|---|
|
Overall Study
STARTED
|
53
|
54
|
52
|
|
Overall Study
COMPLETED
|
34
|
34
|
38
|
|
Overall Study
NOT COMPLETED
|
19
|
20
|
14
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of GTx-024 on Muscle Wasting (Cachexia) Cancer.
Baseline characteristics by cohort
| Measure |
GTx -024
n=53 Participants
1 mg
|
GTx-024
n=54 Participants
3 mg
|
3 mg of Placebo
n=52 Participants
Placebo
|
Total
n=159 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
25 Participants
n=93 Participants
|
22 Participants
n=4 Participants
|
24 Participants
n=27 Participants
|
71 Participants
n=483 Participants
|
|
Age, Categorical
>=65 years
|
28 Participants
n=93 Participants
|
32 Participants
n=4 Participants
|
28 Participants
n=27 Participants
|
88 Participants
n=483 Participants
|
|
Age, Continuous
|
65.7 years
STANDARD_DEVIATION 10.03 • n=93 Participants
|
66.3 years
STANDARD_DEVIATION 10.51 • n=4 Participants
|
65.6 years
STANDARD_DEVIATION 8.73 • n=27 Participants
|
65.9 years
STANDARD_DEVIATION 9.74 • n=483 Participants
|
|
Sex: Female, Male
Female
|
19 Participants
n=93 Participants
|
20 Participants
n=4 Participants
|
17 Participants
n=27 Participants
|
56 Participants
n=483 Participants
|
|
Sex: Female, Male
Male
|
34 Participants
n=93 Participants
|
34 Participants
n=4 Participants
|
35 Participants
n=27 Participants
|
103 Participants
n=483 Participants
|
|
Region of Enrollment
United States
|
39 participants
n=93 Participants
|
37 participants
n=4 Participants
|
36 participants
n=27 Participants
|
112 participants
n=483 Participants
|
|
Region of Enrollment
Argentina
|
14 participants
n=93 Participants
|
17 participants
n=4 Participants
|
16 participants
n=27 Participants
|
47 participants
n=483 Participants
|
PRIMARY outcome
Timeframe: Baseline to Four MonthsPopulation: The number of participants were those subjects in the modified intent-to-treat population (defined as subjects with at least one post baseline DEXA for LBM) who had baseline and 4 month DEXA results for LBM (observed cases).
Change in total body lean mass as measured by dual energy x-ray absorptiometry (DEXA)from baseline to 4 months.
Outcome measures
| Measure |
GTx-024 1 mg
n=32 Participants
|
GTx-024 3 mg
n=34 Participants
|
Placebo
n=34 Participants
Placebo
|
|---|---|---|---|
|
The Efficacy of GTx-024 on Total Body Lean Mass.
|
1.55 kg
Interval -2.06 to 12.64
|
0.98 kg
Interval -4.84 to 11.54
|
0.02 kg
Interval -5.81 to 7.46
|
SECONDARY outcome
Timeframe: Four MonthsPopulation: The subjects were in the MITT population (had a post baseline DEXA) and had a month 4 stair climb assessment (observed cases).
Change in stair climb power from baseline to 4 months. Stair climb power is defined power (watts)=\[9.8 m/sec\*\*2\]\*\[weight (kg)\]\*\[height of 12 steps(meters)\]/ \[time (seconds) up the 12 steps\].
Outcome measures
| Measure |
GTx-024 1 mg
n=31 Participants
|
GTx-024 3 mg
n=28 Participants
|
Placebo
n=36 Participants
Placebo
|
|---|---|---|---|
|
To Assess the Efficacy of GTx-024 on Muscle Function (Performance) as Measured by Stair Climb.
|
19.93 watts
Interval -235.3 to 110.1
|
12.84 watts
Interval -77.7 to 93.1
|
11.34 watts
Interval -156.4 to 56.4
|
Adverse Events
GTx -024
Serious events: 14 serious events
Other events: 47 other events
Deaths: 0 deaths
GTx-024
Serious events: 15 serious events
Other events: 49 other events
Deaths: 0 deaths
3 mg of Placebo
Serious events: 17 serious events
Other events: 46 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
GTx -024
n=53 participants at risk
1 mg
|
GTx-024
n=54 participants at risk
3 mg
|
3 mg of Placebo
n=52 participants at risk
Placebo
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
5.7%
3/53
|
0.00%
0/54
|
0.00%
0/52
|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
3.8%
2/53
|
0.00%
0/54
|
5.8%
3/52
|
|
Blood and lymphatic system disorders
Neutropenia
|
1.9%
1/53
|
1.9%
1/54
|
0.00%
0/52
|
|
Blood and lymphatic system disorders
Pancytopenia
|
0.00%
0/53
|
0.00%
0/54
|
1.9%
1/52
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/53
|
1.9%
1/54
|
0.00%
0/52
|
|
Cardiac disorders
Atrial Fibrillation
|
1.9%
1/53
|
0.00%
0/54
|
0.00%
0/52
|
|
Cardiac disorders
Myocardial infarction
|
1.9%
1/53
|
0.00%
0/54
|
0.00%
0/52
|
|
Gastrointestinal disorders
Diarrhoea
|
1.9%
1/53
|
0.00%
0/54
|
0.00%
0/52
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/53
|
0.00%
0/54
|
1.9%
1/52
|
|
Gastrointestinal disorders
gastric ulcer perforation
|
0.00%
0/53
|
0.00%
0/54
|
1.9%
1/52
|
|
Gastrointestinal disorders
lower gastrointestinal haemorrhage
|
0.00%
0/53
|
1.9%
1/54
|
0.00%
0/52
|
|
Gastrointestinal disorders
pancreatitis
|
0.00%
0/53
|
0.00%
0/54
|
1.9%
1/52
|
|
Gastrointestinal disorders
rectal obstruction
|
0.00%
0/53
|
1.9%
1/54
|
0.00%
0/52
|
|
Gastrointestinal disorders
small intestinal obstruction
|
0.00%
0/53
|
0.00%
0/54
|
1.9%
1/52
|
|
Gastrointestinal disorders
upper gastrointestinal haemorrhage
|
0.00%
0/53
|
1.9%
1/54
|
0.00%
0/52
|
|
Infections and infestations
cellulitis
|
0.00%
0/53
|
1.9%
1/54
|
0.00%
0/52
|
|
Infections and infestations
diverticulitis
|
0.00%
0/53
|
0.00%
0/54
|
1.9%
1/52
|
|
Infections and infestations
pneumonia
|
3.8%
2/53
|
5.6%
3/54
|
3.8%
2/52
|
|
Infections and infestations
sepsis
|
0.00%
0/53
|
0.00%
0/54
|
1.9%
1/52
|
|
Infections and infestations
septic shock
|
0.00%
0/53
|
1.9%
1/54
|
0.00%
0/52
|
|
Infections and infestations
staphylococcal skin infection
|
0.00%
0/53
|
0.00%
0/54
|
1.9%
1/52
|
|
Injury, poisoning and procedural complications
compression fracture
|
0.00%
0/53
|
1.9%
1/54
|
0.00%
0/52
|
|
Injury, poisoning and procedural complications
hip fracture
|
1.9%
1/53
|
0.00%
0/54
|
0.00%
0/52
|
|
Injury, poisoning and procedural complications
patella fracture
|
1.9%
1/53
|
0.00%
0/54
|
1.9%
1/52
|
|
Injury, poisoning and procedural complications
road traffic accident
|
0.00%
0/53
|
1.9%
1/54
|
0.00%
0/52
|
|
Injury, poisoning and procedural complications
Therapeutic agent toxicity
|
0.00%
0/53
|
0.00%
0/54
|
0.00%
0/52
|
|
Metabolism and nutrition disorders
diabetes mellitus
|
1.9%
1/53 • Number of events 1
|
0.00%
0/54
|
0.00%
0/52
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
lung neoplasm malignant
|
0.00%
0/53
|
—
0/0
|
1.9%
1/52 • Number of events 1
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
malignant neoplasm progression
|
9.4%
5/53 • Number of events 6
|
13.0%
7/54
|
15.4%
8/52 • Number of events 9
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases to central nervous system
|
0.00%
0/53
|
1.9%
1/54
|
0.00%
0/52
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
metastatic pain
|
0.00%
0/53
|
1.9%
1/54
|
0.00%
0/52
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
non small cell lung cancer
|
1.9%
1/53 • Number of events 1
|
0.00%
0/54
|
1.9%
1/52 • Number of events 1
|
|
Nervous system disorders
brain stem infarction
|
1.9%
1/53 • Number of events 1
|
0.00%
0/54
|
0.00%
0/52
|
|
Renal and urinary disorders
renal failure acute
|
1.9%
1/53 • Number of events 1
|
0.00%
0/54
|
0.00%
0/52
|
|
Respiratory, thoracic and mediastinal disorders
acute pulmonary oedema
|
1.9%
1/53 • Number of events 1
|
0.00%
0/54
|
0.00%
0/52
|
|
Respiratory, thoracic and mediastinal disorders
chronic obstructive pulmonary disease
|
0.00%
0/53
|
1.9%
1/54
|
0.00%
0/52
|
|
Respiratory, thoracic and mediastinal disorders
dyspnoea
|
1.9%
1/53 • Number of events 1
|
0.00%
0/54
|
0.00%
0/52
|
Other adverse events
| Measure |
GTx -024
n=53 participants at risk
1 mg
|
GTx-024
n=54 participants at risk
3 mg
|
3 mg of Placebo
n=52 participants at risk
Placebo
|
|---|---|---|---|
|
Blood and lymphatic system disorders
anaemia
|
24.5%
13/53
|
13.0%
7/54
|
15.4%
8/52
|
|
Blood and lymphatic system disorders
neutropenia
|
5.7%
3/53
|
9.3%
5/54
|
3.8%
2/52
|
|
Blood and lymphatic system disorders
thrombocytopenia
|
5.7%
3/53
|
9.3%
5/54
|
5.8%
3/52
|
|
Gastrointestinal disorders
abdominal pain
|
3.8%
2/53
|
14.8%
8/54
|
5.8%
3/52
|
|
Gastrointestinal disorders
constipation
|
17.0%
9/53
|
11.1%
6/54
|
3.8%
2/52
|
|
Gastrointestinal disorders
diarrhoea
|
15.1%
8/53
|
16.7%
9/54
|
13.5%
7/52
|
|
Gastrointestinal disorders
vomiting
|
17.0%
9/53
|
7.4%
4/54
|
11.5%
6/52
|
|
Gastrointestinal disorders
asthenia
|
1.9%
1/53
|
13.0%
7/54
|
7.7%
4/52
|
|
Gastrointestinal disorders
fatigue
|
15.1%
8/53
|
25.9%
14/54
|
21.2%
11/52
|
|
General disorders
pyrexia
|
9.4%
5/53
|
14.8%
8/54
|
5.8%
3/52
|
|
Infections and infestations
pneumonia
|
3.8%
2/53
|
7.4%
4/54
|
3.8%
2/52
|
|
Investigations
weight decreased
|
9.4%
5/53
|
14.8%
8/54
|
9.6%
5/52
|
|
Metabolism and nutrition disorders
anorexia
|
9.4%
5/53 • Number of events 5
|
11.1%
6/54
|
3.8%
2/52 • Number of events 2
|
|
Metabolism and nutrition disorders
dehydration
|
1.9%
1/53 • Number of events 1
|
13.0%
7/54 • Number of events 7
|
11.5%
6/52 • Number of events 6
|
|
Musculoskeletal and connective tissue disorders
back pain
|
9.4%
5/53 • Number of events 5
|
1.9%
1/54 • Number of events 1
|
5.8%
3/52 • Number of events 4
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
malignant neoplasm progression
|
11.3%
6/53 • Number of events 8
|
14.8%
8/54 • Number of events 8
|
15.4%
8/52 • Number of events 9
|
|
Nervous system disorders
headache
|
5.7%
3/53 • Number of events 3
|
9.3%
5/54 • Number of events 7
|
1.9%
1/52 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
3.8%
2/53 • Number of events 2
|
16.7%
9/54 • Number of events 9
|
11.5%
6/52 • Number of events 6
|
|
Respiratory, thoracic and mediastinal disorders
dyspnoea
|
13.2%
7/53 • Number of events 8
|
7.4%
4/54 • Number of events 6
|
9.6%
5/52 • Number of events 5
|
|
Gastrointestinal disorders
Nausea
|
24.5%
13/53
|
11.1%
6/54
|
13.5%
7/52
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60