Fibromyalgia
Conditions
Keywords
FMS, Fibro, pain, Body pain, tenderness, stiffness, muscular pain, joint pain
Brief summary
The objective of this trial is to evaluate the safety and efficacy of Xyrem® compared to placebo for the treatment of fibromyalgia in a randomized, double blind, placebo controlled, parallel group trial.
Detailed description
The trial is a randomized, double blind, placebo controlled, parallel group trial in subjects diagnosed with fibromyalgia in accordance with the American College of Rheumatology. Total duration is up to twenty-two (22) weeks of trial participation. Subjects will undergo a screening and withdrawal/washout period lasting up to five (5) weeks combined followed by baseline period lasting one (1) week. Total treatment duration will be fourteen (14) weeks followed by a two week safety follow-up post treatment period. During the screening and withdrawal/washout period, no study medication will be given; however rescue medication up to 4 grams per day of acetaminophen (paracetemol) will be allowed.
Interventions
DRUGplacebo
Oral Solution
DRUGXyrem®
two doses
Sponsors
Jazz Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female subjects, 18 years or older who meet the American College of Rheumatology (ACR) diagnostic criteria for fibromyalgia.
Exclusion criteria
* Subjects will be excluded if they have a history of rheumatic disease or other disorders that may compromise reliable representation of subjective symptoms. * Any other condition that will cause a risk to subjects if they participate in the trial is also a reason for exclusion.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Pain VAS (Visual Analog Scale) Response. Percentage of Subjects With a Greater Than or Equal to 30% Reduction in Pain VAS From Baseline (BOCF). | Baseline to Week 14 | 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. |
Countries
France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Placebo Placebo taken as two equally divided nightly doses | 188 |
| Xyrem (Sodium Oxybate) 4.5g Xyrem 4.5g taken as 2 equally divided nightly doses | 195 |
| Xyrem (Sodium Oxybate) 6.0g Xyrem 6.0g taken as 2 equally divided nightly doses | 190 |
| Total | 573 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Adverse Event | 11 | 30 | 40 |
| Overall Study | Lack of Efficacy | 23 | 18 | 19 |
| Overall Study | Lost to Follow-up | 5 | 4 | 4 |
| Overall Study | Physician Decision | 2 | 0 | 0 |
| Overall Study | Pregnancy | 0 | 0 | 1 |
| Overall Study | Protocol Violation | 9 | 4 | 4 |
| Overall Study | Sponsor Decision | 0 | 1 | 0 |
| Overall Study | Subject started night shift work | 1 | 0 | 0 |
| Overall Study | Undergoing tests for Cushings Syndrome | 0 | 0 | 1 |
| Overall Study | Will start exclusionary medications | 0 | 1 | 0 |
| Overall Study | Withdrawal by Subject | 6 | 8 | 5 |
Baseline characteristics
| Characteristic | Total | Xyrem (Sodium Oxybate) 6.0g | Placebo | Xyrem (Sodium Oxybate) 4.5g |
|---|---|---|---|---|
| Age Continuous | 46.6 years STANDARD_DEVIATION 10.72 | 46.4 years STANDARD_DEVIATION 11.62 | 46.8 years STANDARD_DEVIATION 9.73 | 46.6 years STANDARD_DEVIATION 10.76 |
| Age, Customized 18 - 39 years | 141 participants | 53 participants | 43 participants | 45 participants |
| Age, Customized 40 - 49 years | 192 participants | 58 participants | 62 participants | 72 participants |
| Age, Customized 50 - 64 years | 221 participants | 69 participants | 81 participants | 71 participants |
| Age, Customized >= 65 years | 19 participants | 10 participants | 2 participants | 7 participants |
| Race (NIH/OMB) American Indian or Alaska Native | 3 Participants | 1 Participants | 2 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 4 Participants | 1 Participants | 2 Participants | 1 Participants |
| Race (NIH/OMB) Black or African American | 39 Participants | 18 Participants | 10 Participants | 11 Participants |
| Race (NIH/OMB) More than one race | 3 Participants | 1 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 524 Participants | 169 Participants | 173 Participants | 182 Participants |
| Region of Enrollment France | 24 participants | 7 participants | 9 participants | 8 participants |
| Region of Enrollment Germany | 70 participants | 20 participants | 22 participants | 28 participants |
| Region of Enrollment Italy | 10 participants | 4 participants | 2 participants | 4 participants |
| Region of Enrollment Netherlands | 4 participants | 3 participants | 1 participants | 0 participants |
| Region of Enrollment Poland | 22 participants | 6 participants | 8 participants | 8 participants |
| Region of Enrollment Spain | 52 participants | 14 participants | 18 participants | 20 participants |
| Region of Enrollment United Kingdom | 11 participants | 3 participants | 4 participants | 4 participants |
| Region of Enrollment United States | 380 participants | 133 participants | 124 participants | 123 participants |
| Sex: Female, Male Female | 513 Participants | 170 Participants | 168 Participants | 175 Participants |
| Sex: Female, Male Male | 60 Participants | 20 Participants | 20 Participants | 20 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 126 / 188 | 146 / 194 | 156 / 189 |
| serious Total, serious adverse events | 3 / 188 | 2 / 194 | 3 / 189 |
Outcome results
Primary
Pain VAS (Visual Analog Scale) Response. Percentage of Subjects With a Greater Than or Equal to 30% Reduction in Pain VAS From Baseline (BOCF).
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.
Time frame: Baseline to Week 14
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Placebo | 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 |
| Placebo | 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 |
| Xyrem (Sodium Oxybate) 4.5g | 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 | 35.4 Percentage of Participants |
| Xyrem (Sodium Oxybate) 4.5g | 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 | 64.6 Percentage of Participants |
| Xyrem (Sodium Oxybate) 6.0g | 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 | 35.3 Percentage of Participants |
| Xyrem (Sodium Oxybate) 6.0g | 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 | 64.7 Percentage of Participants |
Comparison: Overall Comparisonp-value: 0.001Chi-squared
Comparison: Pairwise comparison of Xyrem 4.5g and placebop-value: <0.001Chi-squared
Comparison: Pairwise comparison of Xyrem 6.0g and placebop-value: 0.001Chi-squared