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Xyrem

Phase 3

Fibromyalgia | Small molecule | Musculoskeletal |Jazz Pharmaceuticals plc|Last Updated: Mar 30, 2012

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials4
Total Enrollment1,876
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00423605Safety and Efficacy Study of Xyrem® (Sodium Oxybate) in Subjects With Fibromyalgia.PHASE3 COMPLETED 560Dec 1, 2006Jan 1, 2010Mar 30, 201291 United States, France +5
NCT00423813A Safety and Efficacy Study of Xyrem® (Sodium Oxybate) to Treat Fibromyalgia.PHASE3 COMPLETED 573Dec 1, 2006Jun 1, 2009Jan 24, 2012116 United States, France +6
NCT00371137A Safety and Efficacy Study of Xyrem® in Subjects With FibromyalgiaPHASE3 COMPLETED 548Aug 1, 2006Dec 1, 2008Dec 29, 201170 United States
NCT00087555Trial Comparing the Effects of Xyrem (Sodium Oxybate) With Placebo for the Treatment of FibromyalgiaPHASE2 COMPLETED 195Jul 1, 2004Jan 1, 2006Jan 24, 201220 United States
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Study Endpoints
Primary Endpoints
Number of Subjects Reporting Adverse Events
Treatment Period (38 weeks)

Number of subjects reporting adverse events.

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.

The Primary Outcome Measure Was a Composite of Changes From Baseline in Three Co-primary Self Report Measures: Pain Visual Analog Scale (PVAS, Electronic Diaries), Fibromyalgia Impact Questionnaire (FIQ), and Patient Global Impression of Change (PGI-C).
Baseline to week 8

The percentage of participants who met all 3 of the following criteria: Reduction of \>=20% from baseline to week 8 in both PVAS \& FIQ total score and PGI-C response of "very much better" or "much better". Analysis was based on LOCF (Last Observation Carried Forward) data. The PVAS ranges from 0 (no pain) to 100 (worst imaginable pain). The FIQ ranges from 0 (best function) to 100 (worst function). PGI-C is a 7 point likert scale measuring change in the participant's fibromyalgia symptoms that ranges from "very much worse" to "very much better"

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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
1EXPERIMENTAL -
2EXPERIMENTAL -
3PLACEBO_COMPARATORPlacebo (one of two doses matching active treatment by volume).
Interventions
NameTypeDescription
Xyrem®DRUGflexible dosing
placeboDRUGOral Solution
Xyrem (sodium oxybate) oral solutionDRUGXyrem (sodium oxybate) oral solution 4.5 g per day in divided doses, 2.25 g at bedtime and another 2.5 g two and a half to four hours later for 8 weeks.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites91

Inclusion Criteria: * Subject has qualified for and completed either study 06-009 (NCT00423813) or 06-008 (NCT00371137). * Subject is able, in the opinion of the investigator, to take Xyrem® for approximately 9-1/2 months. Exclusion Criteria: * Subject terminated early from either study 06-009 or...

Countries:United StatesFranceGermanyItalyNetherlandsSpainUnited KingdomPoland
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Competitive Landscape -Fibromyalgia 5 trials
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