Tourette SyndromePhase 2NCT03732534
Rollover Study for Continuing NBI-98854 Administration in Pediatric Subjects With Tourette Syndrome
A Phase 2 study testing NBI-98854 in people with Tourette Syndrome, run by Neurocrine Biosciences at 10 sites in 2 countries.
Trial summary
This is an open-label, rollover study to collect long-term safety, tolerability, and investigator- and participant-reported pharmacodynamic (PD) data after chronic administration of NBI-98854 in pediatric participants with Tourette Syndrome (TS), as well as to provide open-label access to NBI-98854 for the treatment… Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Tourette Syndrome
- What is being tested?
- NBI-98854
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 10 sitesin 2 countries
- Study dates
- 17 Oct 2018to 18 Feb 2019
- Phase
- Phase 2
- 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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Have a clinical diagnosis of Tourette Syndrome (TS)
- Have participated in the NBI-98854-TS2004 or NBI 98854-TS2005 Phase 2 studies
- If using maintenance medication(s) for TS and/or TS spectrum diagnoses (e.g. obsessive-compulsive disorder [OCD], Attention-Deficit Hyperactivity Disorder [ADHD]), be on stable doses
- Be in good general health
- Subjects of childbearing potential who do not practice total abstinence must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently during the screening, treatment and follow-up periods of the study
Who cannot join
- Have an active, clinically significant unstable medical condition within 1 month before screening
- Have a known history of long QT syndrome or cardiac arrhythmia
- Have a known history of neuroleptic malignant syndrome
- Have a cancer diagnosis within 3 years prior to screening (some exceptions allowed)
- Have an allergy, hypersensitivity, or intolerance to VMAT2 inhibitors
- Have a blood loss ≥250 mL or donated blood within 56 days, or donated plasma within 7 days, before the start of the study
Show all 11Show fewer
- Have a known history of substance dependence, substance (drug) or alcohol abuse within 3 months before the start of the study
- Have a significant risk of suicidal or violent behavior
- Have received an investigational drug within 30 days before the start of the study or plan to use an investigational drug (other than NBI-98854) during the study
- Are currently participating in another NBI-98854 clinical study
- Are pregnant (for females)
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States9 sites
Neurocrine Clinical Site
Anaheim, California, 92805
Neurocrine Clinical Site
Chicago, Illinois, 60634
Neurocrine Clinical Site
Dallas, Texas, 75243
Neurocrine Clinical Site
Gulf Breeze, Florida, 32561
Neurocrine Clinical Site
Hialeah, Florida, 33013
Neurocrine Clinical Site
Houston, Texas, 77058
Neurocrine Clinical Site
Lincoln, Nebraska, 68526
Neurocrine Clinical Site
Naperville, Illinois, 60563
Neurocrine Clinical Site
San Diego, California, 92108
About this study
Official title: Open-Label Rollover Study for Continuing NBI-98854 Administration for the Treatment of Pediatric Subjects With Tourette Syndrome
This is an open-label, rollover study to collect long-term safety, tolerability, and investigator- and participant-reported pharmacodynamic (PD) data after chronic administration of NBI-98854 in pediatric participants with Tourette Syndrome (TS), as well as to provide open-label access to NBI-98854 for the treatment of TS for pediatric participants who have taken part in a Phase 2 NBI-98854 study.
- Conditions
- Tourette Syndrome
- Being tested
- NBI-98854
- Medical subject headings
- Tourette Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NBI-98854Drug
Also called Valbenazine
vesicular monoamine transporter 2 (VMAT2) inhibitor
| Study group | Role | What they receive |
|---|---|---|
| NBI-98854 | Experimental | NBI-98854 administered once daily for up to 96 weeks Receives: Drug: NBI-98854 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
A TEAE is an adverse event not present prior to the initiation of study drug dosing, or is an already present event that worsens either in intensity or frequency following the initiation of study drug dosing.
Measured: Up to 16 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2018
- Study start17 Oct 2018
- Primary completion18 Feb 2019
- Study completion18 Feb 2019
Why it stopped: Study stopped early due to investigational drug not meeting primary endpoint in the main study T-Force GOLD (NBI-98854-TS2003); no safety concerns identified.
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 Tourette SyndromeStudies of the same condition, open and under way.
- More trials run by Neurocrine BiosciencesThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurocrine Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Development Lead, Study director, Neurocrine Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03732534
- Sponsor's protocol ID
- NBI-98854-TS2007
- Status last verified
- Mar 2022
- First submitted
- 5 Nov 2018
- Last updated
- 19 Apr 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-Label Rollover Study for Continuing NBI-98854 Administration for the Treatment of Pediatric Subjects With Tourette Syndrome
- ClinicalTrials.gov ID
- NCT03732534
- Acronym / sponsor ID
- NBI-98854-TS2007
- Conditions
- Tourette Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study stopped early due to investigational drug not meeting primary endpoint in the main study T-Force GOLD (NBI-98854-TS2003); no safety concerns identified.
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Neurocrine BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 17 Oct 2018 (actual)
- Primary completion
- 18 Feb 2019 (actual)
- Study completion
- 18 Feb 2019 (actual)
- First posted
- 6 Nov 2018 (actual)
- Results
- Posted 19 Apr 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label, rollover study to collect long-term safety, tolerability, and investigator- and participant-reported pharmacodynamic (PD) data after chronic administration of NBI-98854 in pediatric participants with Tourette Syndrome (TS), as well as to provide open-label access to NBI-98854 for the treatment of TS for pediatric participants who have taken part in a Phase 2 NBI-98854 study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NBI-98854 | Experimental | Drug: NBI-98854NBI-98854 administered once daily for up to 96 weeks |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Have a clinical diagnosis of Tourette Syndrome (TS)
- Have participated in the NBI-98854-TS2004 or NBI 98854-TS2005 Phase 2 studies
- If using maintenance medication(s) for TS and/or TS spectrum diagnoses (e.g. obsessive-compulsive disorder [OCD], Attention-Deficit Hyperactivity Disorder [ADHD]), be on stable doses
- Be in good general health
- Subjects of childbearing potential who do not practice total abstinence must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently during the screening, treatment and follow-up periods of the study
Key exclusion criteria (11 listed)
- Have an active, clinically significant unstable medical condition within 1 month before screening
- Have a known history of long QT syndrome or cardiac arrhythmia
- Have a known history of neuroleptic malignant syndrome
- Have a cancer diagnosis within 3 years prior to screening (some exceptions allowed)
- Have an allergy, hypersensitivity, or intolerance to VMAT2 inhibitors
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 |
|---|---|---|
| NBI-98854Also: Valbenazinevesicular monoamine transporter 2 (VMAT2) inhibitor | Drug | NBI-98854 |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: Up to 16 Weeks
A TEAE is an adverse event not present prior to the initiation of study drug dosing, or is an already present event that worsens either in intensity or frequency following the initiation of study drug dosing.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Apr 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Clinical Development Lead study director
Neurocrine Biosciences
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NBI-98854 in people with Tourette Syndrome, run by Neurocrine Biosciences at 10 sites in 2 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 5 inclusion and 11 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: stopped early, as of the sponsor's last update on 19 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Puerto Rico. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 19 Apr 2022). 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?
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (time frame: Up to 16 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 19 Apr 2022.