Narcolepsy With CataplexyPhase 3NCT02221869
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
A Phase 3 study testing Xyrem in people with Narcolepsy With Cataplexy, run by Jazz Pharmaceuticals at 20 sites in 4 countries.
Trial summary
The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics subjects with narcolepsy that includes cataplexy. Read more about this study
- Who is it for?
- Ages 7 to 17 years
- Conditions the study is for
- Narcolepsy With Cataplexy
- What is being tested?
- Xyrem
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 106 participants
- Where
- 20 sitesin 4 countries
- Study dates
- 1 Oct 2014to 25 Jan 2019
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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)
- Have a primary diagnosis of narcolepsy with cataplexy that meets International Classification of Sleep Disorders (ICSD)-2 or ICSD-3 criteria, whichever was in effect at the time of the diagnosis or, with the permission of the Medical Monitor, completes a Multiple Sleep Latency Test (MSLT) during Screening to confirm the diagnosis of Type 1 narcolepsy by ICSD-3 criteria (i.e., the subject meets all other ICSD-3 criteria for Type 1 narcolepsy)
- Have given documented assent indicating that he/she was aware of the investigational nature of the study and the required procedures and restrictions before participation in any protocol-related activities
- Have parent(s)/guardian(s) who have given informed consent for his/her/their child's participation in the study
- Be willing to spend the required number of nights (2 to 3) in a sleep laboratory for PSG evaluations
- If currently treated with Xyrem, must have been taking unchanged doses (twice nightly dosing no higher than 9 g/night) of Xyrem, and stimulants, if applicable, for the treatment of narcolepsy symptoms for at least 2 months prior to screening
Show all 9Show fewer
- In addition to the above inclusion criteria, subjects participating in the PK evaluation must meet the following inclusion criteria:
- 7. Be willing to spend 2 additional nights in the clinic for PK evaluation
- -
Who cannot join
- Inability to understand assent or follow study instructions for any reason, in the opinion of the Investigator
- Parent(s) or guardian(s) unable to comply with the requirements of the study for any reason, in the opinion of the Investigator
- Other documented clinically significant condition (including an unstable medical condition, chronic disease other than narcolepsy with cataplexy, or history or presence of another neurological disorder) that might affect the subject's safety and/or interfere with the conduct of the study in the opinion of the Investigator
- Treatment with benzodiazepines, non-benzodiazepine anxiolytics/ hypnotics/sedatives, neuroleptics, opioids, barbiturates, diclofenac, valproate, phenytoin, ethosuximide within 2 weeks prior to enrollment (discontinuation for the purpose of study enrollment is permitted only if considered safe by the Investigator and approved by the Medical Monitor)
- Treatment with any other medications that have anticataplectic effect (e.g., serotonin-norepinephrine reuptake inhibitors [SNRIs], selective serotonin reuptake inhibitors [SSRIs], or tricyclic antidepressants [TCAs]) within 1 month before Screening
- Unsafe for the subject to receive placebo treatment for 2 weeks, in the opinion of the Investigator
- In addition to the above exclusion criteria, subjects participating in the PK evaluation must not demonstrate the following:
- -
Trial locations and contacts
20 sites in 4 countries.
20 sites in 4 countries
United States17 sites
The U-M Sleep Disorders Center
Ann Arbor, Michigan, 48109
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
SleepMed of SC
Columbia, South Carolina, 29201
Nationwide Children's Hospital
Columbus, Ohio, 43205
Duke Children's Hospital
Durham, North Carolina, 27710
Greenville Health System
Greenville, South Carolina, 29615
Todd Swick, MD, PA
Houston, Texas, 77063
ARSM Research, LLC
Huntersville, North Carolina, 28078
Miller Children's Hospital - Long Beach
Long Beach, California, 90806
UT/LeBonheur Neuroscience Institute
Memphis, Tennessee, 38105
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507
SDS Clinical Trials, Inc.
Orange, California, 92868
Stanford Sleep Medicine Center
Redwood City, California, 94063
Seattle Children's Hospital
Seattle, Washington, 98105
Montefiore Medical Center
The Bronx, New York, 10467
Netherlands1 site
Sleep Wake Center SEIN Heemstede
Heemstede, North Holland, 2103 SW
About this study
The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics subjects with narcolepsy that includes cataplexy.
- Conditions
- Narcolepsy With Cataplexy
- Being tested
- Xyrem
- Medical subject headings
- Narcolepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
XyremDrug
| Study group | Role | What they receive |
|---|---|---|
| Xyrem | Experimental | Active Xyrem at a dose ≤9 g/night Receives: Drug: Xyrem |
| Xyrem Placebo | Placebo comparator | Xyrem placebo at a volume and regimen equivalent to the stable dose of Xyrem. Receives: Drug: Xyrem |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Weekly Number of Cataplexy Attacks
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.
Measured: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
Secondary outcomes (4)
Clinical Global Impression of Change (CGIc) for Cataplexy Severity
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.
Measured: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score
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.
Measured: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
CGIc for Narcolepsy Overall
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.
Measured: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
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
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
Measured: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2014 (estimated)
- Study start1 Oct 2014
- Primary completionFeb 2017
- Study completion25 Jan 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Narcolepsy With CataplexyStudies of the same condition, open and under way.
- More trials run by Jazz PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 106 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02221869
- Sponsor's protocol ID
- 13-005
- Status last verified
- Apr 2019
- First submitted
- 28 Jul 2014
- Last updated
- 30 Apr 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Dauvilliers Y, Lammers GJ, Lecendreux M, Maski K, Kansagra S, Black J, Parvataneni R, Chen A, Wang YG, Plazzi G. Effect of sodium oxybate on body mass index in pediatric patients with narcolepsy. J Clin Sleep Med. 2024 Mar 1;20(3):445-454. doi: 10.5664/jcsm.10912.
PubMed 37942930Lecendreux M, Plazzi G, Dauvilliers Y, Rosen CL, Ruoff C, Black J, Parvataneni R, Guinta D, Wang YG, Mignot E. Long-term safety and maintenance of efficacy of sodium oxybate in the treatment of narcolepsy with cataplexy in pediatric patients. J Clin Sleep Med. 2022 Sep 1;18(9):2217-2227. doi: 10.5664/jcsm.10090.
PubMed 35689598Wang YG, Menno D, Chen A, Steininger TL, Morris S, Black J, Profant J, Johns MW. Validation of the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) questionnaire in pediatric patients with narcolepsy with cataplexy aged 7-16 years. Sleep Med. 2022 Jan;89:78-84. doi: 10.1016/j.sleep.2021.11.003. Epub 2021 Nov 11.
PubMed 34920345Plazzi G, Ruoff C, Lecendreux M, Dauvilliers Y, Rosen CL, Black J, Parvataneni R, Guinta D, Wang YG, Mignot E. Treatment of paediatric narcolepsy with sodium oxybate: a double-blind, placebo-controlled, randomised-withdrawal multicentre study and open-label investigation. Lancet Child Adolesc Health. 2018 Jul;2(7):483-494. doi: 10.1016/S2352-4642(18)30133-0. Epub 2018 May 21.
PubMed 30169321
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT02221869
- Acronym / sponsor ID
- 13-005
- Conditions
- Narcolepsy With Cataplexy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 106 participants (actual)
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 1 Oct 2014 (actual)
- Primary completion
- Feb 2017 (actual)
- Study completion
- 25 Jan 2019 (actual)
- First posted
- 21 Aug 2014 (estimated)
- Results
- Posted 30 Apr 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics subjects with narcolepsy that includes cataplexy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Xyrem | Experimental | Drug: XyremActive Xyrem at a dose ≤9 g/night |
| Xyrem Placebo | Placebo comparator | Drug: XyremXyrem placebo at a volume and regimen equivalent to the stable dose of Xyrem. |
Who may qualify
- Ages
- 7 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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)
- Have a primary diagnosis of narcolepsy with cataplexy that meets International Classification of Sleep Disorders (ICSD)-2 or ICSD-3 criteria, whichever was in effect at the time of the diagnosis or, with the permission of the Medical Monitor, completes a Multiple Sleep Latency Test (MSLT) during Screening to confirm the diagnosis of Type 1 narcolepsy by ICSD-3 criteria (i.e., the subject meets all other ICSD-3 criteria for Type 1 narcolepsy)
- Have given documented assent indicating that he/she was aware of the investigational nature of the study and the required procedures and restrictions before participation in any protocol-related activities
- Have parent(s)/guardian(s) who have given informed consent for his/her/their child's participation in the study
- Be willing to spend the required number of nights (2 to 3) in a sleep laboratory for PSG evaluations
Key exclusion criteria (8 listed)
- Inability to understand assent or follow study instructions for any reason, in the opinion of the Investigator
- Parent(s) or guardian(s) unable to comply with the requirements of the study for any reason, in the opinion of the Investigator
- Other documented clinically significant condition (including an unstable medical condition, chronic disease other than narcolepsy with cataplexy, or history or presence of another neurological disorder) that might affect the subject's safety and/or interfere with the conduct of the study in the opinion of the Investigator
- Treatment with benzodiazepines, non-benzodiazepine anxiolytics/ hypnotics/sedatives, neuroleptics, opioids, barbiturates, diclofenac, valproate, phenytoin, ethosuximide within 2 weeks prior to enrollment (discontinuation for the purpose of study enrollment is permitted only if considered safe by the Investigator and approved by the Medical Monitor)
- Treatment with any other medications that have anticataplectic effect (e.g., serotonin-norepinephrine reuptake inhibitors [SNRIs], selective serotonin reuptake inhibitors [SSRIs], or tricyclic antidepressants [TCAs]) within 1 month before Screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Xyrem | Drug | Xyrem, Xyrem Placebo |
Primary outcome
Change in Weekly Number of Cataplexy Attacks
Time frame: 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.
4 secondary outcomes
Clinical Global Impression of Change (CGIc) for Cataplexy Severity
Time frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score
Time frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
CGIc for Narcolepsy Overall
Time frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
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
Time frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37942930Dauvilliers Y, Lammers GJ, Lecendreux M, Maski K, Kansagra S, Black J, Parvataneni R, Chen A, Wang YG, Plazzi G. Effect of sodium oxybate on body mass index in pediatric patients with narcolepsy. J Clin Sleep Med. 2024 Mar 1;20(3):445-454. doi: 10.5664/jcsm.10912.
- Derived from this study: PubMed 35689598Lecendreux M, Plazzi G, Dauvilliers Y, Rosen CL, Ruoff C, Black J, Parvataneni R, Guinta D, Wang YG, Mignot E. Long-term safety and maintenance of efficacy of sodium oxybate in the treatment of narcolepsy with cataplexy in pediatric patients. J Clin Sleep Med. 2022 Sep 1;18(9):2217-2227. doi: 10.5664/jcsm.10090.
- Derived from this study: PubMed 34920345Wang YG, Menno D, Chen A, Steininger TL, Morris S, Black J, Profant J, Johns MW. Validation of the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD) questionnaire in pediatric patients with narcolepsy with cataplexy aged 7-16 years. Sleep Med. 2022 Jan;89:78-84. doi: 10.1016/j.sleep.2021.11.003. Epub 2021 Nov 11.
- Derived from this study: PubMed 30169321Plazzi G, Ruoff C, Lecendreux M, Dauvilliers Y, Rosen CL, Black J, Parvataneni R, Guinta D, Wang YG, Mignot E. Treatment of paediatric narcolepsy with sodium oxybate: a double-blind, placebo-controlled, randomised-withdrawal multicentre study and open-label investigation. Lancet Child Adolesc Health. 2018 Jul;2(7):483-494. doi: 10.1016/S2352-4642(18)30133-0. Epub 2018 May 21.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
20 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Xyrem in people with Narcolepsy With Cataplexy, run by Jazz Pharmaceuticals at 20 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States, France, Italy, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Apr 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Weekly Number of Cataplexy Attacks (time frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02221869, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Apr 2019.