Trial Outcomes & Findings for The Effects of Thalidomide on Symptom Clusters (NCT NCT00379353)

NCT ID: NCT00379353

Last Updated: 2013-02-04

Results Overview

ESAS assessment of appetite (symptom) where the severity at the time of assessment is rated from 0 to 10 on a numerical scale; with 0 meaning that the symptom is absent and 10 that it is the worst possible severity. Evaluated at baseline \[± 3 days\], 2 weeks\[± 3 days\] and 4 weeks \[± 3 days\]

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

32 participants

Primary outcome timeframe

Baseline to Day 29

Results posted on

2013-02-04

Participant Flow

Recruitment Period: 10/18/2006 through 10/27/2008. All participants recruited from UTMD Anderson Cancer Center.

The trial is now closed to new participant entry due to low accrual. Of the 31 participants recruited, 4 were not eligible for evaluation.

Participant milestones

Participant milestones
Measure
Group 1: Thalidomide
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
Two placebo capsules orally, once a day for 14 days.
Overall Study
STARTED
16
15
Overall Study
COMPLETED
5
9
Overall Study
NOT COMPLETED
11
6

Reasons for withdrawal

Reasons for withdrawal
Measure
Group 1: Thalidomide
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
Two placebo capsules orally, once a day for 14 days.
Overall Study
Hospitalization
3
3
Overall Study
Withdrawal by Subject
5
2
Overall Study
Death
1
0
Overall Study
Cardiac Tamponade
1
0
Overall Study
Non-compliance
1
0
Overall Study
Conflicting drug treatment
0
1

Baseline Characteristics

The Effects of Thalidomide on Symptom Clusters

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Group 1: Thalidomide
n=16 Participants
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Total
n=31 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
12 Participants
n=5 Participants
11 Participants
n=7 Participants
23 Participants
n=5 Participants
Age, Categorical
>=65 years
4 Participants
n=5 Participants
4 Participants
n=7 Participants
8 Participants
n=5 Participants
Age Continuous
60 years
n=5 Participants
65 years
n=7 Participants
60 years
n=5 Participants
Sex: Female, Male
Female
7 Participants
n=5 Participants
5 Participants
n=7 Participants
12 Participants
n=5 Participants
Sex: Female, Male
Male
9 Participants
n=5 Participants
10 Participants
n=7 Participants
19 Participants
n=5 Participants
Region of Enrollment
United States
16 participants
n=5 Participants
15 participants
n=7 Participants
31 participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline to Day 29

ESAS assessment of appetite (symptom) where the severity at the time of assessment is rated from 0 to 10 on a numerical scale; with 0 meaning that the symptom is absent and 10 that it is the worst possible severity. Evaluated at baseline \[± 3 days\], 2 weeks\[± 3 days\] and 4 weeks \[± 3 days\]

Outcome measures

Outcome measures
Measure
Group 1: Thalidomide
n=16 Participants
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
n=16 Participants
Two placebo capsules orally, once a day for 14 days.
Thalidomide (Day 29)
n=16 Participants
Thalidomide 100 mg capsules orally, once a day for 14 days.
Placebo (Baseline)
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 15)
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 29)
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Change in Symptoms as Measured by Edmonton Symptom Assessment Scale (ESAS)
Pain
3 units on a scale
Interval 0.0 to 8.0
3 units on a scale
Interval 0.0 to 10.0
4 units on a scale
Interval 0.0 to 8.0
3 units on a scale
Interval 2.0 to 5.0
6.5 units on a scale
Interval 0.0 to 6.5
2.5 units on a scale
Interval 2.5 to 3.0
Change in Symptoms as Measured by Edmonton Symptom Assessment Scale (ESAS)
Fatigue
7 units on a scale
Interval 2.0 to 8.0
5.5 units on a scale
Interval 1.0 to 9.0
5 units on a scale
Interval 2.0 to 6.0
6.5 units on a scale
Interval 4.25 to 8.0
5 units on a scale
Interval 2.5 to 5.5
5 units on a scale
Interval 3.25 to 8.0
Change in Symptoms as Measured by Edmonton Symptom Assessment Scale (ESAS)
Appetite
6 units on a scale
Interval 3.0 to 10.0
4.5 units on a scale
Interval 1.0 to 8.0
7 units on a scale
Interval 0.0 to 10.0
6.5 units on a scale
Interval 0.0 to 6.5
3 units on a scale
Interval 2.0 to 7.0
5.5 units on a scale
Interval 2.25 to 8.0
Change in Symptoms as Measured by Edmonton Symptom Assessment Scale (ESAS)
Depression
6 units on a scale
Interval 0.0 to 10.0
1.5 units on a scale
Interval 0.0 to 7.0
2 units on a scale
Interval 0.0 to 5.0
2.5 units on a scale
Interval 0.0 to 4.7
2 units on a scale
Interval 0.0 to 3.0
2 units on a scale
Interval 0.0 to 3.75

SECONDARY outcome

Timeframe: Baseline to Day 29

12-item symptom-specific subscale of the FACT-G designed to measure participants' additional concerns about their anorexia/cachexia during the previous 7 days. Participant rates concerns from 0 to 4 (0= not at all, 4= very much), combined are the 12 items subscales for a total of 0 to 48 where the higher number would represent greater concern.

Outcome measures

Outcome measures
Measure
Group 1: Thalidomide
n=16 Participants
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Thalidomide (Day 29)
Thalidomide 100 mg capsules orally, once a day for 14 days.
Placebo (Baseline)
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 15)
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 29)
Two placebo capsules orally, once a day for 14 days.
Functional Assessment of Anorexia/Cachexia Therapy (FAACT)
Day 15
32 units on a scale
Interval 13.0 to 38.0
28 units on a scale
Interval 21.0 to 32.0
Functional Assessment of Anorexia/Cachexia Therapy (FAACT)
Baseline
22.5 units on a scale
Interval 9.0 to 36.0
22.5 units on a scale
Interval 19.0 to 32.0
Functional Assessment of Anorexia/Cachexia Therapy (FAACT)
Day 29
25 units on a scale
Interval 19.0 to 38.0
27 units on a scale
Interval 22.0 to 39.0

SECONDARY outcome

Timeframe: Baseline to Day 29

The FACIT-F consists of 27 general quality of life questions divided into 4 domains (physical, social, emotional and functional), plus a 13-item fatigue subscore. The participant rates the intensity of fatigue and its related symptoms on a scale of 0-4 (0= not at all, 4= very much) where the 13-item fatigue subscore totals are combined for a total of 0 to 52, with the higher number representing greater fatigue.

Outcome measures

Outcome measures
Measure
Group 1: Thalidomide
n=16 Participants
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Thalidomide (Day 29)
Thalidomide 100 mg capsules orally, once a day for 14 days.
Placebo (Baseline)
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 15)
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 29)
Two placebo capsules orally, once a day for 14 days.
Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F)
Baseline
21 units on a scale
Interval 7.0 to 35.0
20 units on a scale
Interval 15.5 to 25.0
Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F)
Day 15
24.5 units on a scale
Interval 4.0 to 39.0
24 units on a scale
Interval 17.0 to 31.0
Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F)
Day 29
16 units on a scale
Interval 9.0 to 34.0
24 units on a scale
Interval 18.0 to 32.0

SECONDARY outcome

Timeframe: Baseline to Day 29

The HADS is a self-report rating scale of 14 items on a 4-point Likert scale (range 0-3). It is designed to measure anxiety and depression (7 items for each subscale). The total score is the sum of the 14 items, and for each subscale the score is the sum of the respective seven items (ranging from 0-21). The higher the score The higher the score, the more likely the patient is showing signs of anxiety and as a result may benefit from a counseling/supportive session.

Outcome measures

Outcome measures
Measure
Group 1: Thalidomide
n=16 Participants
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Thalidomide (Day 29)
Thalidomide 100 mg capsules orally, once a day for 14 days.
Placebo (Baseline)
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 15)
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 29)
Two placebo capsules orally, once a day for 14 days.
Hospital Anxiety and Depression Scale (HADS) HADS-A (Anxiety)
Baseline
9 units on a scale
Interval 2.0 to 16.0
7 units on a scale
Interval 4.0 to 9.0
Hospital Anxiety and Depression Scale (HADS) HADS-A (Anxiety)
Day 15
9 units on a scale
Interval 1.0 to 9.0
4 units on a scale
Interval 2.0 to 10.0
Hospital Anxiety and Depression Scale (HADS) HADS-A (Anxiety)
Day 29
6 units on a scale
Interval 5.0 to 10.0
6.5 units on a scale
Interval 4.2 to 10.0

SECONDARY outcome

Timeframe: Baseline to Day 29

The HADS is a self-report rating scale of 14 items on a 4-point Likert scale (range 0-3). It is designed to measure anxiety and depression (7 items for each subscale). The total score is the sum of the 14 items, and for each subscale the score is the sum of the respective seven items (ranging from 0-21). The higher the score, the more likely the patient is showing signs of depression and as a result may benefit from a counseling/supportive session.

Outcome measures

Outcome measures
Measure
Group 1: Thalidomide
n=16 Participants
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Thalidomide (Day 29)
Thalidomide 100 mg capsules orally, once a day for 14 days.
Placebo (Baseline)
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 15)
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 29)
Two placebo capsules orally, once a day for 14 days.
Hospital Anxiety and Depression Scale (HADS) HADS-D (Depression)
Baseline
10 units on a scale
Interval 4.0 to 17.0
8 units on a scale
Interval 3.0 to 10.0
Hospital Anxiety and Depression Scale (HADS) HADS-D (Depression)
Day 15
8 units on a scale
Interval 3.0 to 13.0
8.5 units on a scale
Interval 3.0 to 12.0
Hospital Anxiety and Depression Scale (HADS) HADS-D (Depression)
Day 29
8 units on a scale
Interval 4.0 to 17.0
6.59 units on a scale
Interval 4.2 to 10.0

SECONDARY outcome

Timeframe: Baseline to Day 29

PSQI measures the quality and patterns of sleep. It differentiates poor from good sleep by measuring subjective sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction. A participant indicates how frequently each item was experienced on a scale from 0 to 3. The 7 component scores are then summed to obtain a global sleep score that can range from 0 to 21. A score of \>/= 5 indicates poor sleepers.

Outcome measures

Outcome measures
Measure
Group 1: Thalidomide
n=16 Participants
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Thalidomide (Day 29)
Thalidomide 100 mg capsules orally, once a day for 14 days.
Placebo (Baseline)
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 15)
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 29)
Two placebo capsules orally, once a day for 14 days.
Pittsburgh Sleep Quality Index (PSQI)
Baseline
9.5 units on a scale
Interval 7.5 to 14.7
11 units on a scale
Interval 5.2 to 15.0
Pittsburgh Sleep Quality Index (PSQI)
Day 15
10.5 units on a scale
Interval 5.25 to 12.7
8.5 units on a scale
Interval 3.25 to 13.5
Pittsburgh Sleep Quality Index (PSQI)
Day 29
8 units on a scale
Interval 3.0 to 10.0
8 units on a scale
Interval 4.0 to 12.0

SECONDARY outcome

Timeframe: Baseline to Day 29

BMI, commonly used to measure overweight and obesity, is a measure of body fat based on a person's weight and height.

Outcome measures

Outcome measures
Measure
Group 1: Thalidomide
n=16 Participants
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
n=16 Participants
Two placebo capsules orally, once a day for 14 days.
Thalidomide (Day 29)
n=16 Participants
Thalidomide 100 mg capsules orally, once a day for 14 days.
Placebo (Baseline)
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 15)
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 29)
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Change in Body Composition as Measured by Body Mass Index (BMI)
21.1 kg/m^2
Interval 20.2 to 25.4
21.2 kg/m^2
Interval 20.75 to 23.8
23.4 kg/m^2
Interval 19.6 to 28.2
22.3 kg/m^2
Interval 19.4 to 26.9
23.7 kg/m^2
Interval 19.4 to 26.9
22.1 kg/m^2
Interval 19.1 to 26.9

SECONDARY outcome

Timeframe: Baseline to Day 15

Cytokines Levels of IL-1β and its receptor IL RA, IL-6 its receptor IL-6R, and TNF-α and its receptors (i.e. tumor necrosis factor receptor (TNFR)) of TNFR1, TNFR2, IL-10, IL-8(serum) measured at baseline, Days 15 and 29. Multiplex bead Immunoassay used to measure serum/plasma levels of IL-1, IL-6, TNF-α, IL-10, IL-8 and their receptors where assay sensitivity for the cytokines was 3-6 pg/mL. Serum IL-10, IL-1β, IL-1RA, IL-6R, sTNF-RI, sTNF-R2 were also analyzed using an enzyme-linked immunosorbent assay device. Lowering cytokine levels can decrease fatigue, increase appetite and decrease anxiety and depression.

Outcome measures

Outcome measures
Measure
Group 1: Thalidomide
n=16 Participants
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
n=16 Participants
Two placebo capsules orally, once a day for 14 days.
Thalidomide (Day 29)
n=15 Participants
Thalidomide 100 mg capsules orally, once a day for 14 days.
Placebo (Baseline)
n=15 Participants
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 15)
Two placebo capsules orally, once a day for 14 days.
Placebo (Day 29)
Two placebo capsules orally, once a day for 14 days.
Change in Serum Cytokines and Receptors
IL-1b
0.52 pg/mL
Interval 0.32 to 0.74
0.67 pg/mL
Interval 0.29 to 2.77
0.83 pg/mL
Interval 0.22 to 1.4
0.57 pg/mL
Interval 0.43 to 0.68
Change in Serum Cytokines and Receptors
IL1RA
677 pg/mL
Interval 501.0 to 784.0
290.7 pg/mL
Interval -901.0 to 515.0
579 pg/mL
Interval 266.0 to 755.0
102.3 pg/mL
Interval -639.6 to 356.3
Change in Serum Cytokines and Receptors
Tumor necrosis factor receptor 2 (TNFR2)
8788 pg/mL
Interval 3286.0 to 21126.0
75.4 pg/mL
Interval -693.0 to 4319.0
6788.2 pg/mL
Interval 5440.0 to 14446.0
-107.8 pg/mL
Interval -1726.0 to 1766.0
Change in Serum Cytokines and Receptors
IL-6
54.1 pg/mL
Interval 16.8 to 280.3
85 pg/mL
Interval 18.0 to 140.0
76.4 pg/mL
Interval 19.1 to 133.0
-6.51 pg/mL
Interval -37.9 to 26.4
Change in Serum Cytokines and Receptors
Tumor necrosis factor -a (TNF-a)
18.2 pg/mL
Interval 13.8 to 123.1
6.4 pg/mL
Interval -24.8 to 108.8
21.2 pg/mL
Interval 17.8 to 37.1
6.24 pg/mL
Interval -12.8 to 14.6
Change in Serum Cytokines and Receptors
IL-17
3.2 pg/mL
Interval 1.75 to 39.7
9.2 pg/mL
Interval 3.1 to 17.0
12.07 pg/mL
Interval 4.3 to 122.6
11 pg/mL
Interval 3.4 to 316.3
Change in Serum Cytokines and Receptors
IL-8
36.1 pg/mL
Interval 29.7 to 68.1
2.12 pg/mL
Interval -7.5 to 11.2
139.9 pg/mL
Interval 54.1 to 289.1
-3.7 pg/mL
Interval -20.9 to 50.3
Change in Serum Cytokines and Receptors
IL-6R
6264 pg/mL
Interval 4321.0 to 30159.0
5446 pg/mL
Interval 3530.0 to 16596.0
13567.4 pg/mL
Interval 7186.0 to 17164.4
10059.7 pg/mL
Interval 5117.0 to 19944.0
Change in Serum Cytokines and Receptors
Tumor necrosis factor receptor 1 (TNFR1)
2007.2 pg/mL
Interval 418.6 to 2593.2
-77.2 pg/mL
Interval -161.7 to 604.6
6788 pg/mL
Interval 793.1 to 34000.9
99.4 pg/mL
Interval -356.4 to 539.8
Change in Serum Cytokines and Receptors
IL10
2.58 pg/mL
Interval 1.66 to 7.77
2.58 pg/mL
Interval 1.6 to 7.7
2.83 pg/mL
Interval 1.31 to 4.93
2.6 pg/mL
Interval 1.4 to 6.8

Adverse Events

Group 1: Thalidomide

Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths

Group 2: Placebo

Serious events: 2 serious events
Other events: 7 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Group 1: Thalidomide
n=16 participants at risk
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
n=15 participants at risk
Two placebo capsules orally, once a day for 14 days.
Blood and lymphatic system disorders
Anemia
12.5%
2/16 • Number of events 2
13.3%
2/15 • Number of events 2
Gastrointestinal disorders
Constipation
6.2%
1/16 • Number of events 1
6.7%
1/15 • Number of events 1
Nervous system disorders
Dizziness
6.2%
1/16 • Number of events 1
0.00%
0/15
Nervous system disorders
Drowsiness
0.00%
0/16
6.7%
1/15 • Number of events 1
Infections and infestations
Infection
12.5%
2/16 • Number of events 2
6.7%
1/15 • Number of events 1
Psychiatric disorders
Insomnia
0.00%
0/16
6.7%
1/15 • Number of events 1
Vascular disorders
Thromboembolism
0.00%
0/16
6.7%
1/15 • Number of events 1

Other adverse events

Other adverse events
Measure
Group 1: Thalidomide
n=16 participants at risk
100 mg capsules orally, once a day for 14 days
Group 2: Placebo
n=15 participants at risk
Two placebo capsules orally, once a day for 14 days.
Blood and lymphatic system disorders
Anemia
25.0%
4/16 • Number of events 4
33.3%
5/15 • Number of events 5
Gastrointestinal disorders
Constipation
25.0%
4/16 • Number of events 4
46.7%
7/15 • Number of events 7
Gastrointestinal disorders
Diarrhea
6.2%
1/16 • Number of events 1
6.7%
1/15 • Number of events 1
Nervous system disorders
Dizziness
18.8%
3/16 • Number of events 3
33.3%
5/15 • Number of events 5
Nervous system disorders
Drowsiness
25.0%
4/16 • Number of events 4
33.3%
5/15 • Number of events 5
Infections and infestations
Infection
0.00%
0/16
13.3%
2/15 • Number of events 2
Psychiatric disorders
Insomnia
25.0%
4/16 • Number of events 4
33.3%
5/15 • Number of events 5
Nervous system disorders
Numbness
6.2%
1/16 • Number of events 1
40.0%
6/15 • Number of events 6

Additional Information

Dr. Eduardo Bruera, Professor

University of Texas M.D. Anderson Cancer Center

Phone: 713-563-4157

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place