Trial Outcomes & Findings for Rollover Study for Continuing NBI-98854 Administration in Pediatric Subjects With Tourette Syndrome (NCT NCT03732534)

Last Updated: 2022-04-19

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

6 participants

Primary outcome timeframe

Up to 16 Weeks

Results posted on

2022-04-19

Participant Flow

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.

Participant milestones

Participant milestones
Measure	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.
Overall Study STARTED	6
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Reasons for withdrawal
Measure	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.
Overall Study Study terminated by Sponsor	6

Baseline Characteristics

Rollover Study for Continuing NBI-98854 Administration in Pediatric Subjects With Tourette Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Valbenazine n=6 Participants 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.
Age, Continuous	11.8 years STANDARD_DEVIATION 2.5 • n=39 Participants
Sex: Female, Male Female	1 Participants n=39 Participants
Sex: Female, Male Male	5 Participants n=39 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=39 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=39 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=39 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=39 Participants
Race (NIH/OMB) Asian	0 Participants n=39 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=39 Participants
Race (NIH/OMB) Black or African American	1 Participants n=39 Participants
Race (NIH/OMB) White	5 Participants n=39 Participants
Race (NIH/OMB) More than one race	0 Participants n=39 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=39 Participants

PRIMARY outcome

Timeframe: Up to 16 Weeks

Outcome measures

Outcome measures
Measure	Valbenazine n=6 Participants 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.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	2 Participants

Adverse Events

Valbenazine

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Valbenazine n=6 participants at risk 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.
Nervous system disorders Somnolence	33.3% 2/6 • Up to 16 weeks
Infections and infestations Nasopharyngitis	16.7% 1/6 • Up to 16 weeks

Additional Information

Neurocrine Medical Information

Neurocrine Biosciences

Phone: 877-641-3461

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Generally, the PI has the right to publish results provided such publication does not violate confidentiality or IP provisions within the contract with the Sponsor. Prior to submission for publication or presentation of results, the PI must provide the Sponsor time for review. The Sponsor can request the PI to withhold or remove information from all publications. For a multi-center study, any publication of results by the PI shall not be made before the first multi-center publication.
Publication restrictions are in place

Restriction type: OTHER