Trial Outcomes & Findings for A Safety and Efficacy Study of Xyrem® in Subjects With Fibromyalgia (NCT NCT00371137)
NCT ID: NCT00371137
Last Updated: 2011-12-29
Results Overview
Percentage of pain VAS responders. Subjects with a \>= 30% reduction in pain VAS from baseline to endpoint (week 14) were considered responders; all other subjects were considered non-responders. Missing data were handled using BOCF (Baseline Observation Carried Forward). The pain VAS ranges from 0 (no pain) to 100 (worst imaginable pain). The pain VAS was collected morning, afternoon and evening. Baseline is the average value recorded for the measure during the week prior to the end-of-baseline visit. Endpoint is the average value recorded for the measure during the week prior to week 14.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
548 participants
Primary outcome timeframe
Baseline to Week 14
Results posted on
2011-12-29
Participant Flow
Participant milestones
| Measure |
Placebo
Placebo taken as two equally divided nightly doses
|
Xyrem 4.5g
Xyrem 4.5g taken as two equally divided nightly doses
|
Xyrem 6.0g
Xyrem 6.0g taken as two equally divided nightly doses
|
|---|---|---|---|
|
Overall Study
STARTED
|
183
|
182
|
183
|
|
Overall Study
Treated
|
183
|
182
|
182
|
|
Overall Study
COMPLETED
|
111
|
119
|
104
|
|
Overall Study
NOT COMPLETED
|
72
|
63
|
79
|
Reasons for withdrawal
| Measure |
Placebo
Placebo taken as two equally divided nightly doses
|
Xyrem 4.5g
Xyrem 4.5g taken as two equally divided nightly doses
|
Xyrem 6.0g
Xyrem 6.0g taken as two equally divided nightly doses
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
20
|
35
|
42
|
|
Overall Study
Withdrawal by Subject
|
11
|
10
|
15
|
|
Overall Study
Lost to Follow-up
|
6
|
3
|
5
|
|
Overall Study
Lack of Efficacy
|
30
|
12
|
13
|
|
Overall Study
Sponsor Decision
|
2
|
1
|
2
|
|
Overall Study
Physician Decision
|
1
|
1
|
1
|
|
Overall Study
Protocol Violation
|
2
|
1
|
1
|
Baseline Characteristics
A Safety and Efficacy Study of Xyrem® in Subjects With Fibromyalgia
Baseline characteristics by cohort
| Measure |
Placebo
n=183 Participants
Placebo taken as two equally divided nightly doses
|
Xyrem 4.5g
n=182 Participants
Xyrem 4.5g taken as two equally divided nightly doses
|
Xyrem 6.0g
n=183 Participants
Xyrem 6.0g taken as two equally divided nightly doses
|
Total
n=548 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age Continuous
|
46.5 years
STANDARD_DEVIATION 11.43 • n=5 Participants
|
47.0 years
STANDARD_DEVIATION 11.76 • n=7 Participants
|
47.5 years
STANDARD_DEVIATION 10.61 • n=5 Participants
|
47.0 years
STANDARD_DEVIATION 11.26 • n=4 Participants
|
|
Age, Customized
18 - 39 years
|
40 participants
n=5 Participants
|
57 participants
n=7 Participants
|
40 participants
n=5 Participants
|
137 participants
n=4 Participants
|
|
Age, Customized
40 - 49 years
|
70 participants
n=5 Participants
|
47 participants
n=7 Participants
|
57 participants
n=5 Participants
|
174 participants
n=4 Participants
|
|
Age, Customized
50 - 64 years
|
64 participants
n=5 Participants
|
69 participants
n=7 Participants
|
78 participants
n=5 Participants
|
211 participants
n=4 Participants
|
|
Age, Customized
>=65 years
|
9 participants
n=5 Participants
|
9 participants
n=7 Participants
|
8 participants
n=5 Participants
|
26 participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
167 Participants
n=5 Participants
|
166 Participants
n=7 Participants
|
167 Participants
n=5 Participants
|
500 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
48 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
8 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
33 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
167 Participants
n=5 Participants
|
164 Participants
n=7 Participants
|
167 Participants
n=5 Participants
|
498 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
183 participants
n=5 Participants
|
182 participants
n=7 Participants
|
183 participants
n=5 Participants
|
548 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 14Percentage of pain VAS responders. Subjects with a \>= 30% reduction in pain VAS from baseline to endpoint (week 14) were considered responders; all other subjects were considered non-responders. Missing data were handled using BOCF (Baseline Observation Carried Forward). The pain VAS ranges from 0 (no pain) to 100 (worst imaginable pain). The pain VAS was collected morning, afternoon and evening. Baseline is the average value recorded for the measure during the week prior to the end-of-baseline visit. Endpoint is the average value recorded for the measure during the week prior to week 14.
Outcome measures
| Measure |
Placebo
n=183 Participants
Placebo taken as two equally divided nightly doses
|
Xyrem 4.5g
n=182 Participants
Xyrem 4.5g taken as two equally divided nightly doses
|
Xyrem 6.0g
n=183 Participants
Xyrem 6.0g taken as two equally divided nightly doses
|
|---|---|---|---|
|
Pain VAS (Visual Analog Scale) Response. Percentage of Subjects With a Greater Than or Equal to 30% Reduction in Pain VAS From Baseline (BOCF).
Responder
|
27.3 Percentage of Participants
|
46.2 Percentage of Participants
|
39.3 Percentage of Participants
|
|
Pain VAS (Visual Analog Scale) Response. Percentage of Subjects With a Greater Than or Equal to 30% Reduction in Pain VAS From Baseline (BOCF).
Non - Responder
|
72.7 Percentage of Participants
|
53.8 Percentage of Participants
|
60.7 Percentage of Participants
|
Adverse Events
Placebo
Serious events: 1 serious events
Other events: 105 other events
Deaths: 0 deaths
Xyrem 4.5g
Serious events: 0 serious events
Other events: 149 other events
Deaths: 0 deaths
Xyrem 6.0g
Serious events: 2 serious events
Other events: 145 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=183 participants at risk
Placebo taken as two equally divided nightly doses
|
Xyrem 4.5g
n=182 participants at risk
Xyrem 4.5g taken as two equally divided nightly doses
|
Xyrem 6.0g
n=182 participants at risk
Xyrem 6.0g taken as two equally divided nightly doses
|
|---|---|---|---|
|
Gastrointestinal disorders
Colitis
|
0.55%
1/183 • Number of events 1
|
0.00%
0/182
|
0.00%
0/182
|
|
Nervous system disorders
Sleep Paralysis
|
0.00%
0/183
|
0.00%
0/182
|
0.55%
1/182 • Number of events 1
|
|
Nervous system disorders
Unresponsive to stimuli
|
0.00%
0/183
|
0.00%
0/182
|
0.55%
1/182 • Number of events 1
|
|
Gastrointestinal disorders
Large Intestine Perforation
|
0.00%
0/183
|
0.00%
0/182
|
0.55%
1/182 • Number of events 1
|
|
Infections and infestations
Abscess Intestinal
|
0.00%
0/183
|
0.00%
0/182
|
0.55%
1/182 • Number of events 1
|
|
Infections and infestations
Diverticulitis
|
0.00%
0/183
|
0.00%
0/182
|
0.55%
1/182 • Number of events 1
|
Other adverse events
| Measure |
Placebo
n=183 participants at risk
Placebo taken as two equally divided nightly doses
|
Xyrem 4.5g
n=182 participants at risk
Xyrem 4.5g taken as two equally divided nightly doses
|
Xyrem 6.0g
n=182 participants at risk
Xyrem 6.0g taken as two equally divided nightly doses
|
|---|---|---|---|
|
Cardiac disorders
Palpatations
|
0.00%
0/183
|
2.2%
4/182
|
1.1%
2/182
|
|
Ear and labyrinth disorders
Vertigo
|
0.00%
0/183
|
0.55%
1/182
|
2.2%
4/182
|
|
Eye disorders
Vision Blurred
|
0.00%
0/183
|
1.1%
2/182
|
2.7%
5/182
|
|
Gastrointestinal disorders
Nausea
|
5.5%
10/183
|
14.3%
26/182
|
21.4%
39/182
|
|
Gastrointestinal disorders
Vomiting
|
3.8%
7/183
|
4.4%
8/182
|
10.4%
19/182
|
|
Gastrointestinal disorders
Diarrhoea
|
2.7%
5/183
|
5.5%
10/182
|
7.7%
14/182
|
|
Gastrointestinal disorders
Constipation
|
1.6%
3/183
|
2.2%
4/182
|
2.2%
4/182
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
0.55%
1/183
|
2.7%
5/182
|
1.1%
2/182
|
|
Gastrointestinal disorders
Dyspepsia
|
2.7%
5/183
|
0.55%
1/182
|
1.1%
2/182
|
|
General disorders
Fatigue
|
2.7%
5/183
|
2.2%
4/182
|
4.4%
8/182
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
5.5%
10/183
|
3.8%
7/182
|
4.4%
8/182
|
|
Infections and infestations
Nasopharyngitis
|
3.8%
7/183
|
4.4%
8/182
|
4.4%
8/182
|
|
Infections and infestations
Sinusitis
|
0.55%
1/183
|
6.6%
12/182
|
4.4%
8/182
|
|
Infections and infestations
Urinary Tract Infection
|
1.1%
2/183
|
3.8%
7/182
|
1.1%
2/182
|
|
Infections and infestations
Bronchitis
|
1.1%
2/183
|
2.2%
4/182
|
1.6%
3/182
|
|
Investigations
Weight Decreased
|
0.55%
1/183
|
2.7%
5/182
|
2.7%
5/182
|
|
Investigations
Blood Pressure Increased
|
0.55%
1/183
|
1.6%
3/182
|
2.2%
4/182
|
|
Investigations
Weight Increased
|
2.2%
4/183
|
0.55%
1/182
|
0.55%
1/182
|
|
Metabolism and nutrition disorders
Decreased Appetite
|
1.1%
2/183
|
2.7%
5/182
|
1.6%
3/182
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
1.6%
3/183
|
3.3%
6/182
|
3.3%
6/182
|
|
Musculoskeletal and connective tissue disorders
Muscle Spasms
|
2.2%
4/183
|
3.8%
7/182
|
2.2%
4/182
|
|
Musculoskeletal and connective tissue disorders
Pain in Extremity
|
0.55%
1/183
|
2.7%
5/182
|
2.7%
5/182
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.55%
1/183
|
3.8%
7/182
|
0.00%
0/182
|
|
Musculoskeletal and connective tissue disorders
Fibromyalgia
|
0.55%
1/183
|
2.2%
4/182
|
1.6%
3/182
|
|
Nervous system disorders
Headache
|
10.9%
20/183
|
14.8%
27/182
|
23.1%
42/182
|
|
Nervous system disorders
Dizziness
|
2.7%
5/183
|
13.2%
24/182
|
17.0%
31/182
|
|
Nervous system disorders
Somnolence
|
4.9%
9/183
|
2.2%
4/182
|
4.4%
8/182
|
|
Nervous system disorders
Paraesthesia
|
0.00%
0/183
|
3.8%
7/182
|
2.7%
5/182
|
|
Nervous system disorders
Migraine
|
2.2%
4/183
|
1.1%
2/182
|
1.1%
2/182
|
|
Nervous system disorders
Sinus Headache
|
1.1%
2/183
|
2.2%
4/182
|
0.55%
1/182
|
|
Nervous system disorders
Tremor
|
0.55%
1/183
|
0.55%
1/182
|
2.2%
4/182
|
|
Nervous system disorders
Disturbance in Attention
|
0.00%
0/183
|
2.2%
4/182
|
0.00%
0/182
|
|
Psychiatric disorders
Anxiety
|
1.1%
2/183
|
6.6%
12/182
|
5.5%
10/182
|
|
Psychiatric disorders
Insomnia
|
2.2%
4/183
|
1.6%
3/182
|
4.9%
9/182
|
|
Psychiatric disorders
Depression
|
2.2%
4/183
|
1.6%
3/182
|
2.7%
5/182
|
|
Psychiatric disorders
Nightmare
|
3.3%
6/183
|
1.1%
2/182
|
1.6%
3/182
|
|
Psychiatric disorders
Abnormal Dreams
|
1.1%
2/183
|
2.2%
4/182
|
1.1%
2/182
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal Pain
|
1.1%
2/183
|
2.2%
4/182
|
4.9%
9/182
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
1.1%
2/183
|
1.6%
3/182
|
2.7%
5/182
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
0.55%
1/183
|
2.7%
5/182
|
2.2%
4/182
|
|
Skin and subcutaneous tissue disorders
Rash
|
2.2%
4/183
|
2.7%
5/182
|
1.1%
2/182
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
1.6%
3/183
|
2.7%
5/182
|
1.1%
2/182
|
|
Skin and subcutaneous tissue disorders
Night Sweats
|
0.55%
1/183
|
2.7%
5/182
|
0.55%
1/182
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
0.00%
0/183
|
0.55%
1/182
|
2.2%
4/182
|
Additional Information
Grace Wang, MD, Medical Monitor & Director of Clinical Development
Jazz Pharmaceuticals, Inc.
Phone: 650-496-3777
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER