Trial Outcomes & Findings for A Safety and Efficacy Study of Xyrem® (Sodium Oxybate) to Treat Fibromyalgia. (NCT NCT00423813)
NCT ID: NCT00423813
Last Updated: 2012-01-24
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
573 participants
Primary outcome timeframe
Baseline to Week 14
Results posted on
2012-01-24
Participant Flow
Participant milestones
| Measure |
Placebo
Placebo taken as two equally divided nightly doses
|
Xyrem (Sodium Oxybate) 4.5g
Xyrem 4.5g taken as 2 equally divided nightly doses
|
Xyrem (Sodium Oxybate) 6.0g
Xyrem 6.0g taken as 2 equally divided nightly doses
|
|---|---|---|---|
|
Overall Study
STARTED
|
188
|
195
|
190
|
|
Overall Study
Treated
|
188
|
194
|
189
|
|
Overall Study
COMPLETED
|
131
|
129
|
116
|
|
Overall Study
NOT COMPLETED
|
57
|
66
|
74
|
Reasons for withdrawal
| Measure |
Placebo
Placebo taken as two equally divided nightly doses
|
Xyrem (Sodium Oxybate) 4.5g
Xyrem 4.5g taken as 2 equally divided nightly doses
|
Xyrem (Sodium Oxybate) 6.0g
Xyrem 6.0g taken as 2 equally divided nightly doses
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
11
|
30
|
40
|
|
Overall Study
Withdrawal by Subject
|
6
|
8
|
5
|
|
Overall Study
Lost to Follow-up
|
5
|
4
|
4
|
|
Overall Study
Lack of Efficacy
|
23
|
18
|
19
|
|
Overall Study
Physician Decision
|
2
|
0
|
0
|
|
Overall Study
Sponsor Decision
|
0
|
1
|
0
|
|
Overall Study
Protocol Violation
|
9
|
4
|
4
|
|
Overall Study
Pregnancy
|
0
|
0
|
1
|
|
Overall Study
Subject started night shift work
|
1
|
0
|
0
|
|
Overall Study
Will start exclusionary medications
|
0
|
1
|
0
|
|
Overall Study
Undergoing tests for Cushings Syndrome
|
0
|
0
|
1
|
Baseline Characteristics
A Safety and Efficacy Study of Xyrem® (Sodium Oxybate) to Treat Fibromyalgia.
Baseline characteristics by cohort
| Measure |
Placebo
n=188 Participants
Placebo taken as two equally divided nightly doses
|
Xyrem (Sodium Oxybate) 4.5g
n=195 Participants
Xyrem 4.5g taken as 2 equally divided nightly doses
|
Xyrem (Sodium Oxybate) 6.0g
n=190 Participants
Xyrem 6.0g taken as 2 equally divided nightly doses
|
Total
n=573 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age Continuous
|
46.8 years
STANDARD_DEVIATION 9.73 • n=5 Participants
|
46.6 years
STANDARD_DEVIATION 10.76 • n=7 Participants
|
46.4 years
STANDARD_DEVIATION 11.62 • n=5 Participants
|
46.6 years
STANDARD_DEVIATION 10.72 • n=4 Participants
|
|
Age, Customized
18 - 39 years
|
43 participants
n=5 Participants
|
45 participants
n=7 Participants
|
53 participants
n=5 Participants
|
141 participants
n=4 Participants
|
|
Age, Customized
40 - 49 years
|
62 participants
n=5 Participants
|
72 participants
n=7 Participants
|
58 participants
n=5 Participants
|
192 participants
n=4 Participants
|
|
Age, Customized
50 - 64 years
|
81 participants
n=5 Participants
|
71 participants
n=7 Participants
|
69 participants
n=5 Participants
|
221 participants
n=4 Participants
|
|
Age, Customized
>= 65 years
|
2 participants
n=5 Participants
|
7 participants
n=7 Participants
|
10 participants
n=5 Participants
|
19 participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
168 Participants
n=5 Participants
|
175 Participants
n=7 Participants
|
170 Participants
n=5 Participants
|
513 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
20 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
60 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
4 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
|
10 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
39 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
173 Participants
n=5 Participants
|
182 Participants
n=7 Participants
|
169 Participants
n=5 Participants
|
524 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Region of Enrollment
France
|
9 participants
n=5 Participants
|
8 participants
n=7 Participants
|
7 participants
n=5 Participants
|
24 participants
n=4 Participants
|
|
Region of Enrollment
United States
|
124 participants
n=5 Participants
|
123 participants
n=7 Participants
|
133 participants
n=5 Participants
|
380 participants
n=4 Participants
|
|
Region of Enrollment
Poland
|
8 participants
n=5 Participants
|
8 participants
n=7 Participants
|
6 participants
n=5 Participants
|
22 participants
n=4 Participants
|
|
Region of Enrollment
Spain
|
18 participants
n=5 Participants
|
20 participants
n=7 Participants
|
14 participants
n=5 Participants
|
52 participants
n=4 Participants
|
|
Region of Enrollment
Netherlands
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
3 participants
n=5 Participants
|
4 participants
n=4 Participants
|
|
Region of Enrollment
Germany
|
22 participants
n=5 Participants
|
28 participants
n=7 Participants
|
20 participants
n=5 Participants
|
70 participants
n=4 Participants
|
|
Region of Enrollment
Italy
|
2 participants
n=5 Participants
|
4 participants
n=7 Participants
|
4 participants
n=5 Participants
|
10 participants
n=4 Participants
|
|
Region of Enrollment
United Kingdom
|
4 participants
n=5 Participants
|
4 participants
n=7 Participants
|
3 participants
n=5 Participants
|
11 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=188 Participants
Placebo taken as two equally divided nightly doses
|
Xyrem (Sodium Oxybate) 4.5g
n=195 Participants
Xyrem 4.5g taken as 2 equally divided nightly doses
|
Xyrem (Sodium Oxybate) 6.0g
n=190 Participants
Xyrem 6.0g taken as 2 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
|
20.2 Percentage of Participants
|
35.4 Percentage of Participants
|
35.3 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
|
79.8 Percentage of Participants
|
64.6 Percentage of Participants
|
64.7 Percentage of Participants
|
Adverse Events
Placebo
Serious events: 3 serious events
Other events: 126 other events
Deaths: 0 deaths
Xyrem (Sodium Oxybate) 4.5g
Serious events: 2 serious events
Other events: 146 other events
Deaths: 0 deaths
Xyrem (Sodium Oxybate) 6.0g
Serious events: 3 serious events
Other events: 156 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=188 participants at risk
Placebo taken as two equally divided nightly doses
|
Xyrem (Sodium Oxybate) 4.5g
n=194 participants at risk
Xyrem 4.5g taken as 2 equally divided nightly doses
|
Xyrem (Sodium Oxybate) 6.0g
n=189 participants at risk
Xyrem 6.0g taken as 2 equally divided nightly doses
|
|---|---|---|---|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal Pain
|
0.53%
1/188 • Number of events 1
|
0.00%
0/194
|
0.00%
0/189
|
|
Injury, poisoning and procedural complications
Contusion
|
0.53%
1/188 • Number of events 1
|
0.00%
0/194
|
0.00%
0/189
|
|
Injury, poisoning and procedural complications
Joint Sprain
|
0.53%
1/188 • Number of events 1
|
0.00%
0/194
|
0.00%
0/189
|
|
Injury, poisoning and procedural complications
Scratch
|
0.53%
1/188 • Number of events 1
|
0.00%
0/194
|
0.00%
0/189
|
|
Vascular disorders
Secondary Hypertension
|
0.53%
1/188 • Number of events 1
|
0.00%
0/194
|
0.00%
0/189
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/188
|
0.52%
1/194 • Number of events 1
|
0.00%
0/189
|
|
Gastrointestinal disorders
Inguinal Hernia
|
0.00%
0/188
|
0.52%
1/194 • Number of events 1
|
0.00%
0/189
|
|
Musculoskeletal and connective tissue disorders
Rotator Cuff Syndrome
|
0.00%
0/188
|
0.00%
0/194
|
0.53%
1/189 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Cervical Spinal Stenosis
|
0.00%
0/188
|
0.00%
0/194
|
0.53%
1/189 • Number of events 1
|
|
Nervous system disorders
Headache
|
0.00%
0/188
|
0.00%
0/194
|
0.53%
1/189 • Number of events 1
|
Other adverse events
| Measure |
Placebo
n=188 participants at risk
Placebo taken as two equally divided nightly doses
|
Xyrem (Sodium Oxybate) 4.5g
n=194 participants at risk
Xyrem 4.5g taken as 2 equally divided nightly doses
|
Xyrem (Sodium Oxybate) 6.0g
n=189 participants at risk
Xyrem 6.0g taken as 2 equally divided nightly doses
|
|---|---|---|---|
|
Cardiac disorders
Palpatations
|
1.1%
2/188
|
1.0%
2/194
|
2.1%
4/189
|
|
Ear and labyrinth disorders
Vertigo
|
2.1%
4/188
|
4.1%
8/194
|
3.7%
7/189
|
|
Eye disorders
Vision Blurred
|
1.1%
2/188
|
2.1%
4/194
|
1.6%
3/189
|
|
Gastrointestinal disorders
Nausea
|
8.5%
16/188
|
19.1%
37/194
|
21.2%
40/189
|
|
Gastrointestinal disorders
Diarrhoea
|
8.0%
15/188
|
7.2%
14/194
|
11.6%
22/189
|
|
Gastrointestinal disorders
Vomiting
|
3.2%
6/188
|
5.2%
10/194
|
8.5%
16/189
|
|
Gastrointestinal disorders
Constipation
|
1.6%
3/188
|
2.6%
5/194
|
2.1%
4/189
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
1.6%
3/188
|
1.0%
2/194
|
2.6%
5/189
|
|
Gastrointestinal disorders
Dry Mouth
|
1.1%
2/188
|
1.0%
2/194
|
3.2%
6/189
|
|
Gastrointestinal disorders
Abdominal Pain
|
1.1%
2/188
|
2.1%
4/194
|
1.6%
3/189
|
|
Gastrointestinal disorders
Dyspepsia
|
0.53%
1/188
|
1.0%
2/194
|
2.1%
4/189
|
|
General disorders
Fatigue
|
2.1%
4/188
|
3.1%
6/194
|
5.8%
11/189
|
|
General disorders
Oedema Peripheral
|
2.1%
4/188
|
1.5%
3/194
|
6.3%
12/189
|
|
General disorders
Pain
|
1.1%
2/188
|
2.1%
4/194
|
1.1%
2/189
|
|
General disorders
Malaise
|
0.00%
0/188
|
0.52%
1/194
|
2.1%
4/189
|
|
Infections and infestations
Nasopharyngitis
|
3.7%
7/188
|
6.7%
13/194
|
6.9%
13/189
|
|
Infections and infestations
Influenza
|
6.4%
12/188
|
2.1%
4/194
|
4.8%
9/189
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
3.2%
6/188
|
3.1%
6/194
|
2.1%
4/189
|
|
Infections and infestations
Sinusitis
|
2.7%
5/188
|
2.6%
5/194
|
0.53%
1/189
|
|
Investigations
Weight Increased
|
0.53%
1/188
|
2.1%
4/194
|
1.6%
3/189
|
|
Investigations
Blood Pressure Increased
|
0.00%
0/188
|
0.52%
1/194
|
2.1%
4/189
|
|
Metabolism and nutrition disorders
Anorexia
|
0.53%
1/188
|
3.6%
7/194
|
2.1%
4/189
|
|
Metabolism and nutrition disorders
Decrease Appetite
|
0.00%
0/188
|
1.0%
2/194
|
2.1%
4/189
|
|
Metabolism and nutrition disorders
Increased Appetite
|
0.00%
0/188
|
0.00%
0/194
|
2.6%
5/189
|
|
Musculoskeletal and connective tissue disorders
Pain In Extremity
|
4.3%
8/188
|
3.6%
7/194
|
3.7%
7/189
|
|
Musculoskeletal and connective tissue disorders
Muscle Spasms
|
1.1%
2/188
|
4.1%
8/194
|
5.3%
10/189
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
2.1%
4/188
|
2.1%
4/194
|
4.8%
9/189
|
|
Musculoskeletal and connective tissue disorders
Fibromyalgia
|
3.2%
6/188
|
2.1%
4/194
|
3.2%
6/189
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
1.6%
3/188
|
1.5%
3/194
|
3.2%
6/189
|
|
Musculoskeletal and connective tissue disorders
Neck Pain
|
0.53%
1/188
|
2.1%
4/194
|
0.53%
1/189
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/188
|
2.6%
5/194
|
0.00%
0/189
|
|
Nervous system disorders
Headache
|
19.1%
36/188
|
21.1%
41/194
|
22.8%
43/189
|
|
Nervous system disorders
Dizziness
|
1.6%
3/188
|
11.9%
23/194
|
13.2%
25/189
|
|
Nervous system disorders
Somnolence
|
1.6%
3/188
|
3.6%
7/194
|
5.8%
11/189
|
|
Nervous system disorders
Paraesthesia
|
2.7%
5/188
|
2.1%
4/194
|
4.8%
9/189
|
|
Nervous system disorders
Migraine
|
3.2%
6/188
|
1.5%
3/194
|
1.1%
2/189
|
|
Nervous system disorders
Tremor
|
0.00%
0/188
|
3.1%
6/194
|
1.6%
3/189
|
|
Nervous system disorders
Disturbance in Attention
|
0.53%
1/188
|
2.1%
4/194
|
1.1%
2/189
|
|
Psychiatric disorders
Insomnia
|
3.2%
6/188
|
7.7%
15/194
|
4.2%
8/189
|
|
Psychiatric disorders
Anxiety
|
1.6%
3/188
|
5.2%
10/194
|
7.9%
15/189
|
|
Psychiatric disorders
Depression
|
0.00%
0/188
|
1.5%
3/194
|
2.6%
5/189
|
|
Psychiatric disorders
Disorientation
|
0.53%
1/188
|
1.0%
2/194
|
2.6%
5/189
|
|
Psychiatric disorders
Confusional State
|
0.00%
0/188
|
0.52%
1/194
|
2.1%
4/189
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
1.1%
2/188
|
1.0%
2/194
|
3.2%
6/189
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal Pain
|
2.1%
4/188
|
0.52%
1/194
|
1.6%
3/189
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/188
|
2.1%
4/194
|
0.00%
0/189
|
|
Skin and subcutaneous tissue disorders
Rash
|
2.1%
4/188
|
0.52%
1/194
|
4.2%
8/189
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
1.1%
2/188
|
0.52%
1/194
|
2.6%
5/189
|
|
Vascular disorders
Hypertension
|
1.6%
3/188
|
0.52%
1/194
|
2.6%
5/189
|
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