Trial · NCT02221869

A Multicenter Study of the Efficacy and Safety of Xyrem With an Open- Label Pharmacokinetic Evaluation and Safety Extension in Pediatric Subjects With Narcolepsy With Cataplexy

NCT02221869, Phase 3, Completed, Jazz Pharmaceuticals; results first posted 2019-04-30

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Multicenter Study of the Efficacy and Safety of Xyrem With an Open- Label Pharmacokinetic Evaluation and Safety Extension in Pediatric Subjects With Narcolepsy With Cataplexy. Trials, record NCT02221869. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02221869

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Multicenter Study of the Efficacy and Safety of Xyrem With an Open- Label Pharmacokinetic Evaluation and Safety Extension in Pediatric Subjects With Narcolepsy With Cataplexy
NCT number: NCT02221869
Status: Completed
Phases: Phase 3
Enrollment: 106 participants, actual
Lead sponsor: Jazz Pharmaceuticals
Conditions: Narcolepsy With Cataplexy
Interventions: Xyrem
First posted: 2014-08-21
Last updated: 2019-04-30
Results first posted: 2019-04-30

Source ClinicalTrials.gov

Registered outcomes5

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change in Weekly Number of Cataplexy Attacks	From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)	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.
Secondary	Clinical Global Impression of Change (CGIc) for Cataplexy Severity	From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)	CGIc for cataplexy severity from the end of the Stable Dose Period to the end of the Double-blind Treatment Period. The CGIc is a 7-point scale ranging from "very much improved" to "very much worse." A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.
Secondary	Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score	From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)	Change in the ESS (CHAD) score from the end of the Stable Dose Period to the end of the Double-blind Treatment Period. The ESS is a self-administered questionnaire with 8 questions. It provides a measure of a person's general level of daytime sleepiness, or their average sleep propensity in daily life. In the ESS for children and adolescents (CHAD), certain activities were modified. Each activity is scored on a scale ranging from 0-3, with 0 = would never fall asleep, and 3 = high chance of falling asleep. The total score ranges from 0-24, with a higher number representing an increased propensity for sleepiness.
Secondary	CGIc for Narcolepsy Overall	From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)	CGIc for narcolepsy overall from the end of the Stable Dose Period to the end of the Double-blind Treatment Period. The CGIc is a 7-point scale ranging from "very much improved" to "very much worse." A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.
Secondary	Change in Quality of Life (QoL; SF-10 Physical and Psychosocial Summary Score) From the End of the Stable Dose Period to the End of the Double-blind Treatment Period	From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)	The SF-10 Health Survey for Children is a parent-completed survey that contains 10 questions adapted from the Child Health Questionnaire. The SF-10 is intended to produce physical and psychosocial health summary measures. Each of the 10 questions responses is scored with a point value from 1 to 6 (1 is the worst possible condition and 6 is the best possible condition). The SF-10 physical and psychosocial measures are scored such that higher scores indicate more favorable functioning. The questions and associated point values are separated into the Physical Health (PHS-10 domain) and Psychosocial Health (PSS-10 domain). The sums of the scores in each domain are standardized using the mean and standard deviation from a normal population (2006 sample). The standardized scores are transformed to norm based scoring (NBS) metric. Through NBS, scale scores are standardized to a mean of 50 and SD of 10 in the combined U.S. general population and clinical samples. NBS scores are reported

Source ClinicalTrials.gov

Outcomes5

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Change in Weekly Number of Cataplexy Attacks	From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)	number of attacks	MEDIAN
SECONDARY	Clinical Global Impression of Change (CGIc) for Cataplexy Severity	From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)	score on a scale	MEAN
SECONDARY	Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score	From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)	score on a scale	MEDIAN
SECONDARY	CGIc for Narcolepsy Overall	From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)	score on a scale	MEAN
SECONDARY	Change in Quality of Life (QoL; SF-10 Physical and Psychosocial Summary Score) From the End of the Stable Dose Period to the End of the Double-blind Treatment Period	From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)	score on a scale	MEDIAN

Source ClinicalTrials.gov

Results by group12

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change in Weekly Number of Cataplexy Attacks	Xyrem		0.27	Inter-Quartile Range -1.00 to 2.50	number of attacks
Change in Weekly Number of Cataplexy Attacks	Xyrem Placebo		12.71	Inter-Quartile Range 3.44 to 19.77	number of attacks
Clinical Global Impression of Change (CGIc) for Cataplexy Severity	Xyrem		-0.4	Standard Deviation 1.12	score on a scale
Clinical Global Impression of Change (CGIc) for Cataplexy Severity	Xyrem Placebo		-1.5	Standard Deviation 1.19	score on a scale
Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score	Xyrem		0.0	Inter-Quartile Range -1.0 to 2.0	score on a scale
Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score	Xyrem Placebo		3.0	Inter-Quartile Range 1.0 to 5.0	score on a scale
CGIc for Narcolepsy Overall	Xyrem		-0.4	Standard Deviation 0.95	score on a scale
CGIc for Narcolepsy Overall	Xyrem Placebo		-1.4	Standard Deviation 1.13	score on a scale
Change in Quality of Life (QoL; SF-10 Physical and Psychosocial Summary Score) From the End of the Stable Dose Period to the End of the Double-blind Treatment Period	Xyrem	SF-10 Physical Summary Score	0	Inter-Quartile Range -2.730 to 2.500	score on a scale
Change in Quality of Life (QoL; SF-10 Physical and Psychosocial Summary Score) From the End of the Stable Dose Period to the End of the Double-blind Treatment Period	Xyrem Placebo	SF-10 Physical Summary Score	0	Inter-Quartile Range -8.420 to 2.730	score on a scale

1 to 10 of 12Source ClinicalTrials.gov

See all 12 results by group

Participant flow9

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Randomized to Xyrem	31
Overall Study	STARTED	Randomized to Xyrem Placebo	32
Overall Study	STARTED	Open-label Xyrem	43
Overall Study	COMPLETED	Randomized to Xyrem	30
Overall Study	COMPLETED	Randomized to Xyrem Placebo	32
Overall Study	COMPLETED	Open-label Xyrem	33
Overall Study	NOT COMPLETED	Randomized to Xyrem	1
Overall Study	NOT COMPLETED	Randomized to Xyrem Placebo	0
Overall Study	NOT COMPLETED	Open-label Xyrem	10

Source ClinicalTrials.gov

Adverse events28

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Enuresis	Renal and urinary disorders	Safety Population	20	104
other	Enuresis	Renal and urinary disorders	Randomized Placebo	1	32
other	Nausea	Gastrointestinal disorders	Safety Population	20	104
other	Nausea	Gastrointestinal disorders	Randomized Placebo	0	32
other	Vomiting	Gastrointestinal disorders	Safety Population	19	104
other	Vomiting	Gastrointestinal disorders	Randomized Placebo	0	32
other	Headache	Nervous system disorders	Safety Population	18	104
other	Headache	Nervous system disorders	Randomized Placebo	0	32
other	Weight decreased	Investigations	Safety Population	12	104
other	Weight decreased	Investigations	Randomized Placebo	1	32

1 to 10 of 28Source ClinicalTrials.gov

See all 28 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Safety Population	0	104
Total, all-cause mortality	Randomized Placebo	0	32
Total, other adverse events	Safety Population	80	104
Total, other adverse events	Randomized Placebo	10	32
Total, serious adverse events	Safety Population	2	104
Total, serious adverse events	Randomized Placebo	0	32

Source ClinicalTrials.gov

Papers4

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
30169321	DERIVED	PubMed
34920345	DERIVED	PubMed
35689598	DERIVED	PubMed
37942930	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Safety Population vs Randomized Placebo: deaths from any cause	Trial comparisons
Safety Population vs Randomized Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT02221869

GET https://conductscience.org/api/trials/v1/NCT02221869/events?page=2

API reference For AI assistants