Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Xyrem · 7 trials · 3 indications
Double-blind comparison of the change in weekly number of cataplexy attacks from the last 2 weeks of the Stable Dose Period to the 2 weeks of the Double-blind Treatment Period.
Number of subjects reporting adverse events.
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.
Number of Subjects with treatment-emergent adverse events.
Average Respiratory Rate at 1 year.
The Maintenance of Wakefulness Test consisted of four 20 minute tests of the patient's ability to remain awake in soporific conditions. The Mean change from baseline to week 8 in the average MWT number of minutes until sleep onset was the primary endpoint.
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"
| Arm | Type | Description |
|---|---|---|
| Xyrem | EXPERIMENTAL | Active Xyrem at a dose ≤9 g/night |
| Xyrem Placebo | PLACEBO_COMPARATOR | Xyrem placebo at a volume and regimen equivalent to the stable dose of Xyrem. |
| 1 | EXPERIMENTAL | - |
| 2 | EXPERIMENTAL | - |
| Group 1. | EXPERIMENTAL | Xyrem + Modafinil Placebo |
| Group 2: | PLACEBO_COMPARATOR | Xyrem Placebo + Modafinil Placebo |
| Group 3 | ACTIVE_COMPARATOR | Xyrem Placebo + Modafinil at established dose |
| Group 4: | EXPERIMENTAL | Xyrem + Modafinil at established dose |
| 3 | PLACEBO_COMPARATOR | Placebo (one of two doses matching active treatment by volume). |
| Name | Type | Description |
|---|---|---|
| Xyrem | DRUG | - |
| Xyrem® | DRUG | flexible dosing |
| placebo | DRUG | Oral Solution |
| Xyrem (sodium oxybate) oral solution | DRUG | Xyrem (sodium oxybate) oral solution |
| Xyrem Placebo | DRUG | Xyrem Placebo oral solution 12 ml per day for 4 weeks and 18 ml per day for another 4 weeks. |
| Modafinil at established dose | DRUG | Modafinil oral capsules at 200 to 600 mg per day for 8 weeks. |
| Modafinil (Placebo) | DRUG | Modafinil Placebo oral capsules 1 to 3 capsules per day for 8 weeks. |
Inclusion Criteria: 1. Male or female subjects aged 7-16 years at Visit 2 for subjects on Xyrem at study entry and at Visit 1.1 for Xyrem-naïve subjects (to ensure subjects are \<18 years of age at the end of the study) 2. Have a primary diagnosis of narcolepsy with cataplexy that meets Internation...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Axsome Therapeutics, Inc. | AXSM | 1 | PHASE3 | AXS-14 |
| Grace Therapeutics, Inc. | GRCE | 1 | - | Undisclosed |
Xyrem is used for narcolepsy with cataplexy, fibromyalgia, and narcolepsy. It is a small molecule developed by Jazz Pharmaceuticals plc. Clinical trials have evaluated it in fibromyalgia patients, with completed studies enrolling adults aged 18 years and older.
Xyrem is developed by Jazz Pharmaceuticals plc, a company traded under the ticker JAZZ. The drug is a small molecule being studied for narcolepsy with cataplexy, fibromyalgia, and narcolepsy.
Xyrem is in Phase 3 clinical development. It has completed four trials, including three Phase 3 studies in fibromyalgia. The drug remains investigational and is not approved for the studied indications based on the available data.
Xyrem has completed four clinical trials, including NCT00087555, a Phase 2 study in fibromyalgia with 195 participants, and NCT00371137, NCT00423605, and NCT00423813, all Phase 3 studies in fibromyalgia with enrollments of 548, 560, and 573 participants respectively.
Xyrem is the brand name for sodium oxybate. Clinical trial titles refer to Xyrem (Sodium Oxybate), confirming they are the same drug. Jazz Pharmaceuticals plc develops this small molecule for narcolepsy with cataplexy, fibromyalgia, and narcolepsy.