Tourette SyndromePhase 1T-FORCENCT02256475
Safety, Pharmacokinetics, and Pharmacodynamics of NBI-98854 in Children and Adolescents With Tourette Syndrome
A Phase 1 study testing NBI-98854 in people with Tourette Syndrome, run by Neurocrine Biosciences at 10 sites in United States.
Trial summary
This is a Phase 1b, open-label, multiple-dose study of the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-98854 in a total of 36 pediatric subjects with Tourette syndrome (TS). 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
- 36 participants
- Where
- 10 sitesUnited States
- Study dates
- Sep 2014to Dec 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 documentation of written informed consent, or written and witnessed assent from the subject and written informed consent from the subject's parent or legal guardian.
- Be in good general health.
- Have a Diagnostic and Statistical Manual of Mental Disorders diagnosis of Tourette Syndrome (DSM-IV or -V).
- Have TS symptoms that impair school, occupational, and/or social function.
- If medications are being used to treat TS symptoms, must be on stable doses of these medications for a minimum of 30 days before baseline (Day -1), and the medication regimen is expected to remain stable throughout the study period. The use of tetrabenazine to treat TS symptoms is prohibited.
- Subjects of childbearing potential who do not practice total abstinence must be instructed on the proper use of barrier methods of contraception and agree to use hormonal or two forms of nonhormonal contraception consistently from screening until 30 days after the last dose of study drug.
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- Adolescent subjects (12 to 18 years of age) must have a negative urine drug screen for amphetamines, barbiturates, benzodiazepine, phencyclidine, cocaine, opiates, or cannabinoids. Subjects who are on stable doses of prescribed benzodiazepines, opiates, or psychostimulants are allowed to participate in the study.
- Adolescent subjects (12 to 18 years of age) must have a negative alcohol breath test.
- Be willing and able to adhere to the study regimen and study procedures described in the protocol and informed consent/assent forms, including all requirements at the study center and return for the follow-up visit.
Who cannot join
- Have an unstable medical condition or chronic disease.
- Had a medically significant illness within 30 days of screening.
- Excessive use of tobacco and/or nicotine-containing products.
- Have a history of substance (drug) dependence or substance or alcohol abuse.
- Are currently pregnant or lactating.
- Have a known history of neuroleptic malignant syndrome.
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- Have a known history of long QT syndrome or cardiac tachy-arrhythmia.
- Have a cancer diagnosis within 3 years prior to screening, with the exception of localized skin cancer or carcinoma in situ of the cervix.
- Have received an investigational drug within 30 days before screening or plan to use an investigational drug (other than NBI-98854) during the study.
- Have a significant risk of suicidal or violent behavior.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Study site
Kirkland, Washington
Study site
Kissimmee, Florida
Study site
Lincoln, Nebraska
Study site
Naperville, Illinois
Study site
New York, New York
Study site
Petersburg, Virginia
Study site
Rochester, New York
Study site
San Diego, California
Study site
St Louis, Missouri
Study site
St. Petersburg, Florida
About this study
Official title: A Phase 1b, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-98854 in Children and Adolescents With Tourette Syndrome
This is a Phase 1b, open-label, multiple-dose study of the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-98854 in a total of 36 pediatric subjects with Tourette syndrome (TS). The study will be conducted in approximately 18 male and female children (6 to 11 years of age) and approximately 18 male and female adolescents (12 to 18 years of age). Both age groups will be divided equally into 3 dosing cohorts with 6 subjects each. Ascending doses will be evaluated as part of a staggered-cohort design. Study drug will be administered in each cohort for 14 consecutive days.
- 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
| Study group | Role | What they receive |
|---|---|---|
| Adolescents Dose Group 1 | Experimental | Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Receives: Drug: NBI-98854 |
| Adolescents Dose Group 2 | Experimental | Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the adolescent dose group 1 have been reviewed to ensure there are no safety concerns and that maximum tolerated dose (MTD) has not been reached. Receives: Drug: NBI-98854 |
| Adolescents Dose Group 3 | Experimental | Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the adolescent dose group 2 have been reviewed to ensure there are no safety concerns and that MTD has not been reached. Receives: Drug: NBI-98854 |
| Children Dose Group 1 | Experimental | Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the adolescent dose group 1 have been reviewed to ensure there are no safety concerns and that MTD has not been reached. Receives: Drug: NBI-98854 |
| Children Dose Group 2 | Experimental | Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the children dose group 1 have been reviewed to ensure there are no safety concerns and that MTD has not been reached. Receives: Drug: NBI-98854 |
| Children Dose Group 3 | Experimental | Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the children dose group 2 have been reviewed to ensure there are no safety concerns and that MTD has not been reached. 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 adverse events following dosing with NBI-98854
Measured: Up to 21 days
Area Under Curve (AUC) of NBI-98854 and its metabolites following repeated daily doses of NBI-98854
Measured: Day 1: predose, 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose; Day 14: predose, 0.5, 1, 2, 4, 6, 8, 12, 24, 72, 120, 168 hours postdose
Assessment of tic behaviors associated with TS using the Yale Global Tic Severity Scale (YGTSS)
Measured: Days 1, 7, 14, and 21
Secondary outcomes (4)
Assessment of tic behaviors associated with TS using the Rush Video-based Tic Rating Scale (RTRS)
Measured: Days 1, 7, 14, and 21
Quantification of the premonitory urge phenomena associated with tics using the Premonitory Urge for Tics Scale (PUTS)
Measured: Days 1, 7, 14, and 21
Clinical Global Impression of Tourette Syndrome (CGI-TS)
Measured: Days 1, 7, 14, and 21
CogState computerized test to assess cognitive function
Measured: Day 1 at predose, 2.5 and 8 hours postdose; Day 14 at 8 hours postdose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2014 (estimated)
- Study startSep 2014
- Primary completionNov 2015
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 1
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurocrine Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chris O'Brien, MD, Principal investigator, Neurocrine Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02256475
- Sponsor's protocol ID
- NBI-98854-1403
- Acronym
- T-FORCE
- Status last verified
- Jan 2016
- First submitted
- 23 Sep 2014
- Last updated
- 7 Jan 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-98854 in Children and Adolescents With Tourette Syndrome
- ClinicalTrials.gov ID
- NCT02256475
- Acronym / sponsor ID
- T-FORCE / NBI-98854-1403
- Conditions
- Tourette Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Neurocrine BiosciencesIndustry (pharma, biotech, devices)
- Study start
- Sep 2014
- Primary completion
- Nov 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 3 Oct 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 1b, open-label, multiple-dose study of the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-98854 in a total of 36 pediatric subjects with Tourette syndrome (TS). The study will be conducted in approximately 18 male and female children (6 to 11 years of age) and approximately 18 male and female adolescents (12 to 18 years of age). Both age groups will be divided equally into 3 dosing cohorts with 6 subjects each. Ascending doses will be evaluated as part of a staggered-cohort design. Study drug will be administered in each cohort for 14 consecutive days.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Adolescents Dose Group 1 | Experimental | Drug: NBI-98854Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. |
| Adolescents Dose Group 2 | Experimental | Drug: NBI-98854Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the adolescent dose group 1 have been reviewed to ensure there are no safety concerns and that maximum tolerated dose (MTD) has not been reached. |
| Adolescents Dose Group 3 | Experimental | Drug: NBI-98854Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the adolescent dose group 2 have been reviewed to ensure there are no safety concerns and that MTD has not been reached. |
| Children Dose Group 1 | Experimental | Drug: NBI-98854Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the adolescent dose group 1 have been reviewed to ensure there are no safety concerns and that MTD has not been reached. |
| Children Dose Group 2 | Experimental | Drug: NBI-98854Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the children dose group 1 have been reviewed to ensure there are no safety concerns and that MTD has not been reached. |
| Children Dose Group 3 | Experimental | Drug: NBI-98854Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the children dose group 2 have been reviewed to ensure there are no safety concerns and that MTD has not been reached. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Have documentation of written informed consent, or written and witnessed assent from the subject and written informed consent from the subject's parent or legal guardian.
- Be in good general health.
- Have a Diagnostic and Statistical Manual of Mental Disorders diagnosis of Tourette Syndrome (DSM-IV or -V).
- Have TS symptoms that impair school, occupational, and/or social function.
- If medications are being used to treat TS symptoms, must be on stable doses of these medications for a minimum of 30 days before baseline (Day -1), and the medication regimen is expected to remain stable throughout the study period. The use of tetrabenazine to treat TS symptoms is prohibited.
Key exclusion criteria (10 listed)
- Have an unstable medical condition or chronic disease.
- Had a medically significant illness within 30 days of screening.
- Excessive use of tobacco and/or nicotine-containing products.
- Have a history of substance (drug) dependence or substance or alcohol abuse.
- Are currently pregnant or lactating.
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-98854 | Drug | Adolescents Dose Group 1, Adolescents Dose Group 2, Adolescents Dose Group 3, Children Dose Group 1, Children Dose Group 2, Children Dose Group 3 |
Primary outcome
Number of participants with adverse events following dosing with NBI-98854
Time frame: Up to 21 days
Area Under Curve (AUC) of NBI-98854 and its metabolites following repeated daily doses of NBI-98854
Time frame: Day 1: predose, 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose; Day 14: predose, 0.5, 1, 2, 4, 6, 8, 12, 24, 72, 120, 168 hours postdose
Assessment of tic behaviors associated with TS using the Yale Global Tic Severity Scale (YGTSS)
Time frame: Days 1, 7, 14, and 21
4 secondary outcomes
Assessment of tic behaviors associated with TS using the Rush Video-based Tic Rating Scale (RTRS)
Time frame: Days 1, 7, 14, and 21
Quantification of the premonitory urge phenomena associated with tics using the Premonitory Urge for Tics Scale (PUTS)
Time frame: Days 1, 7, 14, and 21
Clinical Global Impression of Tourette Syndrome (CGI-TS)
Time frame: Days 1, 7, 14, and 21
CogState computerized test to assess cognitive function
Time frame: Day 1 at predose, 2.5 and 8 hours postdose; Day 14 at 8 hours postdose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jan 2016.
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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.
Chris O'Brien, MD principal investigator
Neurocrine Biosciences
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing NBI-98854 in people with Tourette Syndrome, run by Neurocrine Biosciences at 10 sites in United States.
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 10 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 7 Jan 2016. 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 adverse events following dosing with NBI-98854 (time frame: Up to 21 days). The study lists 3 primary outcomes in all. 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 7 Jan 2016.