Narcolepsy With CataplexyPhase 4NCT02637076

Xyrem and Brain Dopamine in Narcolepsy

A Phase 4 study testing Xyrem in people with Narcolepsy With Cataplexy and Healthy Controls, run by Centre for Addiction and Mental Health at 1 site in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The overall aim of this investigation is to establish whether an action of Xyrem® on the brain dopamine system in patients with narcolepsy, and in a comparison control group, might explain part of the anti-narcoleptic effect of the drug. Read more about this study

Who is it for?: Ages 19 years and olderHealthy volunteers can take part
Conditions the study is for: Narcolepsy With Cataplexy, Healthy Controls
What is being tested?: Xyrem
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 17 participants
Where: 1 siteCanada
Study dates: Jan 2016to 23 Jan 2020
Phase: Phase 4
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 19 years and older
All sexes
Healthy volunteers accepted
Adult, Older adult

Who may be able to join

current diagnosis of narcolepsy with cataplexy OR healthy control

Who cannot join

use of any sedative hypnotics, tranquilizers, anticonvulsants, antihistamines (except non-sedating), benzodiazepines, clonidine or any medication known to affect dopamine at start of baseline period
significant unstable or uncontrolled medical/psychiatric disease
significant history of head trauma/surgery or seizure disorder
radiation exposure exceeding 20mSv in last 12 months
pregnancy
substance abuse/dependence (including alcohol)

Show all 12Show fewer

have sleep apnea, or are shift workers
on a sodium-restricted diet
has ever taken Xyrem / sodium oxybate / GHB at any time
claustrophobia
metal implants / objects in the body that may interfere with MRI
succinic semialdehyde dehydrogenase deficiency

Trial locations and contacts

1 site in 1 country.

Canada1 site

Centre for Addiction and Mental Health (CAMH)
Toronto, Ontario, M5T 1R8

Other trials in Canada

About this study

Official title: Does Xyrem Influence Brain Dopamine in Patients With Narcolepsy? A PET Imaging Investigation

Trial Objective is to establish, using positron emission tomography (PET), in Xyrem®-naïve narcolepsy with cataplexy patients, and in matched controls, whether a single dose of Xyrem® causes changes in striatal binding of 11C-raclopride and 11C-DTBZ that would suggest altered activity of brain dopamine neurones.

Conditions: Narcolepsy With Cataplexy, Healthy Controls
Being tested: Xyrem
Keywords: Xyrem, narcolepsy with cataplexy, positron emission tomography, dopamine
Medical subject headings: Narcolepsy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

XyremDrug
Also called Sodium Oxybate, gamma-hydroxybutyrate
single 3.0 gram dose

Study groups in NCT02637076
Study group	Role	What they receive
narcolepsy with cataplexy	Experimental	patients given single dose of Xyrem Receives: Drug: Xyrem
healthy controls	Experimental	healthy controls given a single dose of Xyrem Receives: Drug: Xyrem

What is being measured

The primary outcome is the main question the study is designed to answer.

[C-11]Raclopride BPND at 1 Hour Post Xyrem
BPND (Binding Potential) of [C-11]raclopride measures 1 hour after taking a single 3g dose of Xyrem.
Measured: 1 hour post Xyrem
% Change in PET [C11]Raclopride Binding From Baseline After Single Dose of Xyrem
[C-11] raclopride is a radioligand that binds to the D2/3 dopamine receptor in the dopamine-rich striatum and which is sensitive to dopamine occupancy.
% change was calculated as follows: (1 hour BPND - baseline BPND)/baseline BPND *100; Mean % change represents the mean of each participant's individual % change within the group.
Measured: 1 hour post Xyrem
[C-11]Raclopride BPND at 7 Hours Post Xyrem
BPND (Binding Potential) of [C-11]raclopride measures 7 hours after taking a single 3g dose of Xyrem.
Measured: 7 hours post Xyrem
% Change in PET [C11]Raclopride Binding From Baseline After Single Dose of Xyrem
[C-11] raclopride is a radioligand that binds to the D2/3 dopamine receptor in the dopamine-rich striatum and which is sensitive to dopamine occupancy.
% change was calculated as follows: (7 hours BPND - baseline BPND)/baseline BPND *100; Mean % change represents the mean of each participant's individual % change within the group.
Measured: 7 hours post Xyrem

Secondary outcomes (5)

[C-11]DTBZ BPND at 5 Hours Post Xyrem
Measurement of [C-11]DTBZ 5 hours after taking a single dose of 3g Xyrem
Measured: 5 hours post single Xyrem dose
% Change in PET [C-11] Dihydrotetrabenazine (DTBZ) Binding From Baseline to Five Hours Post Xyrem
[C-11] DTBZ is a radioligand that binds to the vesicular monoamine transporter and which is sensitive to dopamine occupancy.
% change was calculated as follows: (5 hour BPND - baseline BPND)/baseline BPND *100; Mean % change represents the mean of each participant's individual % change within the group.
Measured: 5 hours post single Xyrem dose
Blood Gamma-hydroxybutyrate (GHB) Concentration (AUC)
The concentration of gammahydroxybutyrate in blood of participants who have received a single dose of Xyrem, as measured by Area Under the Curve.
Measured: multiple time points from 0 to 7 hours post-Xyrem
Blood Gamma-hydroxybutyrate (GHB) Cmax
The concentration of gammahydroxybutyrate in blood of participants who have received a single dose of Xyrem as measured by Cmax.
Measured: multiple time points from 0 to 7 hours post-Xyrem
Duration of Drowsiness
Period of time when the participant was experiencing the sedative action of Xyrem. Data derived from self-report as well as anesthesiologist observation.
Measured: observed after receiving single dose of Xyrem, up to 9 hours

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jan 201623 Jan 2020

First posted22 Dec 2015 (estimated)
Study startJan 2016
Primary completion23 Jan 2020
Study completion23 Jan 2020

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.
Other trials for Narcolepsy With CataplexyStudies of the same condition, open and under way.
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 4
Primary purpose: Basic science
Allocation: Non-randomised
Intervention model: Parallel groups
Model description: Participants will be recruited to fill 2 separate study groups: 1) participants with a diagnosis of narcolepsy with cataplexy; 2) healthy controls.
Masking: None (open label)
Enrolment: 17 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02637076
Sponsor's protocol ID: 017-2014
Status last verified: Feb 2021
First submitted: 17 Dec 2015
Last updated: 23 Mar 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingPhase 2
A Study of TAK-360 in Adults With Narcolepsy Without Cataplexy (NT2)
Narcolepsy Type 2
RecruitingPhase 3
A Study to Evaluate the Efficacy and Safety of ALKS 2680 in Adults With Narcolepsy Type 1
Narcolepsy Type 1
RecruitingPhase 2/3
A Study to Investigate the Effects of Cleminorexton Compared With Placebo in the Treatment of Participants With Central Disorders of Hypersomnolence
Narcolepsy Type 1, Narcolepsy Type 2
RecruitingPhase 3
A Phase 3 Study to Evaluate the Efficacy and Safety of Samelisant in Patients With Narcolepsy (AWAKE)
Narcolepsy

All Narcolepsy With Cataplexy trials

Study at a glance

From the registry record, last updated by the sponsor on 23 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Does Xyrem Influence Brain Dopamine in Patients With Narcolepsy? A PET Imaging Investigation
ClinicalTrials.gov ID: NCT02637076
Acronym / sponsor ID: 017-2014
Conditions: Narcolepsy With Cataplexy, Healthy Controls
Keywords: Xyrem, narcolepsy with cataplexy, positron emission tomography, dopamine
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 17 participants (actual)
Lead sponsor: Centre for Addiction and Mental HealthUniversity, hospital or other
Study start: Jan 2016
Primary completion: 23 Jan 2020 (actual)
Study completion: 23 Jan 2020 (actual)
First posted: 22 Dec 2015 (estimated)
Results: Posted 23 Mar 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Basic science
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2
Model description: Participants will be recruited to fill 2 separate study groups: 1) participants with a diagnosis of narcolepsy with cataplexy; 2) healthy controls.

Arm	Type	Receives
narcolepsy with cataplexy	Experimental	Drug: Xyrempatients given single dose of Xyrem
healthy controls	Experimental	Drug: Xyremhealthy controls given a single dose of Xyrem

Who may qualify

Ages: 19 years and older
Sex: All
Healthy volunteers: Accepted
Age groups: Adult, Older adult

Key inclusion criteria (1 listed)

current diagnosis of narcolepsy with cataplexy OR healthy control

Key exclusion criteria (12 listed)

use of any sedative hypnotics, tranquilizers, anticonvulsants, antihistamines (except non-sedating), benzodiazepines, clonidine or any medication known to affect dopamine at start of baseline period
significant unstable or uncontrolled medical/psychiatric disease
significant history of head trauma/surgery or seizure disorder
radiation exposure exceeding 20mSv in last 12 months
pregnancy

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
XyremAlso: Sodium Oxybate, gamma-hydroxybutyratesingle 3.0 gram dose	Drug	healthy controls, narcolepsy with cataplexy

Primary outcome

[C-11]Raclopride BPND at 1 Hour Post Xyrem
Time frame: 1 hour post Xyrem
BPND (Binding Potential) of [C-11]raclopride measures 1 hour after taking a single 3g dose of Xyrem.
% Change in PET [C11]Raclopride Binding From Baseline After Single Dose of Xyrem
Time frame: 1 hour post Xyrem
[C-11] raclopride is a radioligand that binds to the D2/3 dopamine receptor in the dopamine-rich striatum and which is sensitive to dopamine occupancy. % change was calculated as follows: (1 hour BPND - baseline BPND)/baseline BPND *100; Mean % change represents the mean of each participant's individual % change within the group.
[C-11]Raclopride BPND at 7 Hours Post Xyrem
Time frame: 7 hours post Xyrem
BPND (Binding Potential) of [C-11]raclopride measures 7 hours after taking a single 3g dose of Xyrem.
% Change in PET [C11]Raclopride Binding From Baseline After Single Dose of Xyrem
Time frame: 7 hours post Xyrem
[C-11] raclopride is a radioligand that binds to the D2/3 dopamine receptor in the dopamine-rich striatum and which is sensitive to dopamine occupancy. % change was calculated as follows: (7 hours BPND - baseline BPND)/baseline BPND *100; Mean % change represents the mean of each participant's individual % change within the group.

5 secondary outcomes

[C-11]DTBZ BPND at 5 Hours Post Xyrem
Time frame: 5 hours post single Xyrem dose
% Change in PET [C-11] Dihydrotetrabenazine (DTBZ) Binding From Baseline to Five Hours Post Xyrem
Time frame: 5 hours post single Xyrem dose
Blood Gamma-hydroxybutyrate (GHB) Concentration (AUC)
Time frame: multiple time points from 0 to 7 hours post-Xyrem
Blood Gamma-hydroxybutyrate (GHB) Cmax
Time frame: multiple time points from 0 to 7 hours post-Xyrem
Duration of Drowsiness
Time frame: observed after receiving single dose of Xyrem, up to 9 hours

Results and safety

Results posted to the registry on 23 Mar 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Narcolepsy With Cataplexy	2	1
Healthy Controls	15	10

Primary outcome: [C-11]Raclopride BPND at 1 Hour Post Xyrem

Time frame: 1 hour post Xyrem. Mean (Standard Deviation). Unit: BPND (Binding Potential)

Measure	Narcolepsy With Cataplexy n = 1	Healthy Controls n = 10
Limbic Striatum	2.94	3.01 (0.28)
Associate Striatum	3.80	3.70 (0.33)
Sensorimotor Striatum	3.87	4.11 (0.49)

Healthy Controls: p = 0.31, repeated measures ANOVA

Primary outcome: % Change in PET [C11]Raclopride Binding From Baseline After Single Dose of Xyrem

Time frame: 1 hour post Xyrem. Mean (Standard Deviation). Unit: percentage change from baseline

Measure	Narcolepsy With Cataplexy n = 1	Healthy Controls n = 10
Limbic Stiatum	13.27	6.49 (8.60)
Associative Striatum	23.88	2.11 (8.59)
Sensorimotor Striatum	24.43	1.93 (8.85)

Primary outcome: [C-11]Raclopride BPND at 7 Hours Post Xyrem

Time frame: 7 hours post Xyrem. Mean (Standard Deviation). Unit: BPND (Binding Potential)

Measure	Narcolepsy With Cataplexy n = 1	Healthy Controls n = 10
Limbic Striatum	2.33	2.76 (0.18)
Associate Striatum	2.83	3.62 (0.29)
Sensorimotor Striatum	2.75	4.06 (0.40)

Primary outcome: % Change in PET [C11]Raclopride Binding From Baseline After Single Dose of Xyrem

Time frame: 7 hours post Xyrem. Mean (Standard Deviation). Unit: percentage change from baseline

Measure	Narcolepsy With Cataplexy n = 1	Healthy Controls n = 10
Limbic Striatum	-10.13	-2.07 (8.24)
Associative Striatum	-7.50	0.17 (7.42)
Sensorimotor Striatum	-11.56	0.72 (7.64)

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events will be assessed at each study visit following administration of the first PET radiotracer until the follow-up phone after Xyrem administration (2 visits plus 1 follow-up phone call), an average of 10 days per participant.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Narcolepsy With Cataplexy	0 of 2 (0%)	0 of 2 (0%)	0 of 2
Healthy Controls	0 of 15 (0%)	4 of 15 (26.7%)	0 of 15

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Narcolepsy With Cataplexy

RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingPhase 2
A Study of TAK-360 in Adults With Narcolepsy Without Cataplexy (NT2)
Narcolepsy Type 2
RecruitingPhase 3
A Study to Evaluate the Efficacy and Safety of ALKS 2680 in Adults With Narcolepsy Type 1
Narcolepsy Type 1
RecruitingPhase 2/3
A Study to Investigate the Effects of Cleminorexton Compared With Placebo in the Treatment of Participants With Central Disorders of Hypersomnolence
Narcolepsy Type 1, Narcolepsy Type 2
RecruitingPhase 3
A Phase 3 Study to Evaluate the Efficacy and Safety of Samelisant in Patients With Narcolepsy (AWAKE)
Narcolepsy

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.

Stephen J Kish, Ph.D. principal investigator

Centre for Addiction and Mental Health

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Xyrem in people with Narcolepsy With Cataplexy and Healthy Controls, run by Centre for Addiction and Mental Health at 1 site in Canada.

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 1 inclusion and 12 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 23 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Canada. 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. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

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 23 Mar 2021). 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?

[C-11]Raclopride BPND at 1 Hour Post Xyrem (time frame: 1 hour post Xyrem). The study lists 4 primary outcomes in all. 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 NCT02637076.

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 23 Mar 2021.