Tourette Syndrome
Conditions
Brief 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 of TS for pediatric participants who have taken part in a Phase 2 NBI-98854 study.
Interventions
DRUGNBI-98854
vesicular monoamine transporter 2 (VMAT2) inhibitor
Sponsors
Neurocrine Biosciences
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
1. Have a clinical diagnosis of Tourette Syndrome (TS) 2. Have participated in the NBI-98854-TS2004 or NBI 98854-TS2005 Phase 2 studies 3. 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 4. Be in good general health 5. 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
Exclusion criteria
1. Have an active, clinically significant unstable medical condition within 1 month before screening 2. Have a known history of long QT syndrome or cardiac arrhythmia 3. Have a known history of neuroleptic malignant syndrome 4. Have a cancer diagnosis within 3 years prior to screening (some exceptions allowed) 5. Have an allergy, hypersensitivity, or intolerance to VMAT2 inhibitors 6. Have a blood loss ≥250 mL or donated blood within 56 days, or donated plasma within 7 days, before the start of the study 7. Have a known history of substance dependence, substance (drug) or alcohol abuse within 3 months before the start of the study 8. Have a significant risk of suicidal or violent behavior 9. 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 10. Are currently participating in another NBI-98854 clinical study 11. Are pregnant (for females)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | 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. |
Countries
Puerto Rico, United States
Participant flow
Recruitment details
Approximately 240 participants who completed the Phase 2 Study NBI-98854-TS2004 or were to be dosed for at least 16 weeks in Phase 2 Study NBI-98854-TS2005 were planned for this study. However, only 6 participants were enrolled and received study drug because the study was terminated early by the Sponsor.
Participants by arm
| Arm | Count |
|---|---|
| Valbenazine Participants received valbenazine once daily for up to 96 weeks. The starting dose was 20 mg for participants \<50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants \<50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant. | 6 |
| Total | 6 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Study terminated by Sponsor | 6 |
Baseline characteristics
| Characteristic | Valbenazine |
|---|---|
| Age, Continuous | 11.8 years STANDARD_DEVIATION 2.5 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 4 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 5 Participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 6 |
| other Total, other adverse events | 2 / 6 |
| serious Total, serious adverse events | 0 / 6 |
Outcome results
Primary
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.
Time frame: Up to 16 Weeks
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Valbenazine | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | 2 Participants |