Trial Outcomes & Findings for Use of Xyrem to Improve Sleep in Chronic Fatigue Syndrome (NCT NCT00498485)

NCT ID: NCT00498485

Last Updated: 2016-05-19

Results Overview

A count was made of subject responses on a Patient Assessment of Change questionnaire where scores went from +2 \[much better\] thru 0 \[no change\] to -2 \[much worse\]

• Recruitment status: TERMINATED
• Study phase: PHASE4
• Target enrollment: 17 participants
• Primary outcome timeframe: 6 weeks
• Results posted on: 2016-05-19

Participant Flow

Participant milestones

Measure	Placebo Xyrem: Volunteers will complete questionnaires about their symptoms. They will be randomly assigned to one of two groups, placebo or drug. Volunteers will know what group they are in at end of the study. Only the drug group will receive the medication. The placebo group will receive a substance that looks like the real medicine but with no active ingredients. The medication comes as a liquid and patients will start taking an initial dose about 30 min before they expect to sleep. If subjects awaken after less than 5 hrs of sleep, they will take a second dose. If they sleep more than 5 hrs, they will skip the second dose. We will call patients weekly to see how they are doing . If they have tolerable side effects or report significant improvement, we will maintain the dose. But if patients report no effect, the dosage will be incremented by 1 ml per week until good sleep is achieved or a predeterm	Xyrem Xyrem: Same as for Placebo
Overall Study STARTED	7	10
Overall Study COMPLETED	6	5
Overall Study NOT COMPLETED	1	5

Reasons for withdrawal

Measure	Placebo Xyrem: Volunteers will complete questionnaires about their symptoms. They will be randomly assigned to one of two groups, placebo or drug. Volunteers will know what group they are in at end of the study. Only the drug group will receive the medication. The placebo group will receive a substance that looks like the real medicine but with no active ingredients. The medication comes as a liquid and patients will start taking an initial dose about 30 min before they expect to sleep. If subjects awaken after less than 5 hrs of sleep, they will take a second dose. If they sleep more than 5 hrs, they will skip the second dose. We will call patients weekly to see how they are doing . If they have tolerable side effects or report significant improvement, we will maintain the dose. But if patients report no effect, the dosage will be incremented by 1 ml per week until good sleep is achieved or a predeterm	Xyrem Xyrem: Same as for Placebo
Overall Study Adverse Event	1	5

Baseline Characteristics

Use of Xyrem to Improve Sleep in Chronic Fatigue Syndrome

Baseline characteristics by cohort

Measure	Placebo n=7 Participants Xyrem: Volunteers will complete questionnaires about their symptoms and a computerized test to assess their degree of brain fog. They will be randomly assigned to one of two groups, placebo or drug. Volunteers will know what group they are in at end of the study. Only the drug group will receive the medication. The placebo group will receive a substance that looks like the real medicine but with no active ingredients. The medication comes as a liquid and patients will start taking an initial dose about 30 min before they expect to sleep. If subjects awaken after less than 5 hrs of sleep, they will take a second dose. If they sleep more than 5 hrs, they will skip the second dose. We will call patients weekly to see how they are doing . If they have tolerable side effects or report significant improvement, we will maintain the dose. But if patients report no effect, the dosage will be incremented by 1 ml per week until good sleep is achieved or a predeterm	Xyrem n=10 Participants Xyrem: Same as for Placebo	Total n=17 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=5 Participants	10 Participants n=7 Participants	17 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	10 Participants n=7 Participants	17 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	7 participants n=5 Participants	10 participants n=7 Participants	17 participants n=5 Participants
Initial illness severity rating	7 participants n=5 Participants	10 participants n=7 Participants	17 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 weeks

Population: Had we completed the study, the analysis would have been a comparison of counts of those reporting being very much improved across the two treatment conditions

A count was made of subject responses on a Patient Assessment of Change questionnaire where scores went from +2 [much better] thru 0 [no change] to -2 [much worse]

Outcome measures

Measure	Placebo n=6 Participants Xyrem: Volunteers will complete questionnaires. They will be randomly assigned to one of two groups, placebo or drug. Volunteers will know what group they are in at end of the study. Only the drug group will receive the medication. The placebo group will receive a substance that looks like the real medicine but with no active ingredients. The medication comes as a liquid and patients will start taking an initial dose about 30 min before they expect to sleep. If subjects awaken after less than 5 hrs of sleep, they will take a second dose. If they sleep more than 5 hrs, they will skip the second dose. We will call patients weekly to see how they are doing . If they have tolerable side effects or report significant improvement, we will maintain the dose. But if patients report no effect, the dosage will be incremented by 1 ml per week until good sleep is achieved.	Drug Treated n=5 Participants Xyrem: Volunteers will complete questionnaires. They will be randomly assigned to one of two groups, placebo or drug. Volunteers will know what group they are in at end of the study. Only the drug group will receive the medication. The placebo group will receive a substance that looks like the real medicine but with no active ingredients. The medication comes as a liquid and patients will start taking an initial dose about 30 min before they expect to sleep. If subjects awaken after less than 5 hrs of sleep, they will take a second dose. If they sleep more than 5 hrs, they will skip the second dose. We will call patients weekly to see how they are doing . If they have tolerable side effects or report significant improvement, we will maintain the dose. But if patients report no effect, the dosage will be incremented by 1 ml per week until good sleep is achieved.
Global Assessment of Change	6 participants	5 participants

PRIMARY outcome

Timeframe: Assessed at week 6

Population: While data were collected, these data were never analyzed because the study was terminated prematurely by the sponsor and were lost when PI moved from UMDNJ to Beth Israel in NYC in 2008.

Subjects were asked to provide their input as to the quality of their sleep over the past week

Outcome measures

Outcome data not reported

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Xyrem

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Placebo n=7 participants at risk Xyrem: Volunteers will complete questionnaires about their symptoms and a computerized test to assess their degree of brain fog. They will be randomly assigned to one of two groups, placebo or drug. Volunteers will know what group they are in at end of the study. Only the drug group will receive the medication. The placebo group will receive a substance that looks like the real medicine but with no active ingredients. The medication comes as a liquid and patients will start taking an initial dose about 30 min before they expect to sleep. If subjects awaken after less than 5 hrs of sleep, they will take a second dose. If they sleep more than 5 hrs, they will skip the second dose. We will call patients weekly to see how they are doing . If they have tolerable side effects or report significant improvement, we will maintain the dose. But if patients report no effect, the dosage will be incremented by 1 ml per week until good sleep is achieved or a predeterm	Xyrem n=10 participants at risk Xyrem: Same as for Placebo
Gastrointestinal disorders Gastric upset	14.3% 1/7	50.0% 5/10

Additional Information

Dr Benjamin Natelson

Rutgers

Phone: 973-2236370

Email: bnatelson@chpnet.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place