Fibromyalgia
Conditions
Keywords
FMS, Fibro, pain, Body pain, tenderness, joint pain, stiffness, muscular 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-one (21) 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 one (1) week safety follow-up post treatment period. During the screening and withdrawal/washout period, no study medication will be given; however rescue medication acetaminophen (up to 4 grams per day) will be allowed.
Interventions
DRUGXyrem®
two doses
DRUGPlacebo
Oral Solution
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
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Placebo Placebo taken as two equally divided nightly doses | 183 |
| Xyrem 4.5g Xyrem 4.5g taken as two equally divided nightly doses | 182 |
| Xyrem 6.0g Xyrem 6.0g taken as two equally divided nightly doses | 183 |
| Total | 548 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Adverse Event | 20 | 35 | 42 |
| Overall Study | Lack of Efficacy | 30 | 12 | 13 |
| Overall Study | Lost to Follow-up | 6 | 3 | 5 |
| Overall Study | Physician Decision | 1 | 1 | 1 |
| Overall Study | Protocol Violation | 2 | 1 | 1 |
| Overall Study | Sponsor Decision | 2 | 1 | 2 |
| Overall Study | Withdrawal by Subject | 11 | 10 | 15 |
Baseline characteristics
| Characteristic | Xyrem 6.0g | Total | Placebo | Xyrem 4.5g |
|---|---|---|---|---|
| Age Continuous | 47.5 years STANDARD_DEVIATION 10.61 | 47.0 years STANDARD_DEVIATION 11.26 | 46.5 years STANDARD_DEVIATION 11.43 | 47.0 years STANDARD_DEVIATION 11.76 |
| Age, Customized 18 - 39 years | 40 participants | 137 participants | 40 participants | 57 participants |
| Age, Customized 40 - 49 years | 57 participants | 174 participants | 70 participants | 47 participants |
| Age, Customized 50 - 64 years | 78 participants | 211 participants | 64 participants | 69 participants |
| Age, Customized >=65 years | 8 participants | 26 participants | 9 participants | 9 participants |
| Race (NIH/OMB) American Indian or Alaska Native | 2 Participants | 6 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) Asian | 2 Participants | 7 Participants | 3 Participants | 2 Participants |
| Race (NIH/OMB) Black or African American | 12 Participants | 33 Participants | 8 Participants | 13 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 3 Participants | 3 Participants | 0 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 | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) White | 167 Participants | 498 Participants | 167 Participants | 164 Participants |
| Region of Enrollment United States | 183 participants | 548 participants | 183 participants | 182 participants |
| Sex: Female, Male Female | 167 Participants | 500 Participants | 167 Participants | 166 Participants |
| Sex: Female, Male Male | 16 Participants | 48 Participants | 16 Participants | 16 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 | 105 / 183 | 149 / 182 | 145 / 182 |
| serious Total, serious adverse events | 1 / 183 | 0 / 182 | 2 / 182 |
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). | Non - Responder | 72.7 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). | Responder | 27.3 Percentage of Participants |
| Xyrem 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 | 46.2 Percentage of Participants |
| Xyrem 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 | 53.8 Percentage of Participants |
| Xyrem 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 | 39.3 Percentage of Participants |
| Xyrem 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 | 60.7 Percentage of Participants |
Comparison: Overall Comparisonp-value: >0.001Chi-squared
Comparison: Pairwise comparison of placebo and Xyrem 4.5gp-value: <0.001Chi-squared
Comparison: Pairwise comparison of placebo and Xyrem 6.0gp-value: 0.015Chi-squared