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

• ClinicalTrials.gov Identifier: NCT03732534
• Recruitment Status: Terminated
• First Posted: November 6, 2018
• Last Update Posted: February 17, 2020
• Sponsor: Neurocrine Biosciences
• Information provided by (Responsible Party): Neurocrine Biosciences

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Tourette Syndrome	Drug: NBI-98854	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 6 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Open-Label Rollover Study for Continuing NBI-98854 Administration for the Treatment of Pediatric Subjects With Tourette Syndrome
• Actual Study Start Date: October 17, 2018
• Actual Primary Completion Date: February 18, 2019
• Actual Study Completion Date: February 18, 2019

Arms and Interventions

Arm	Intervention/treatment
Experimental: NBI-98854; NBI-98854 administered once daily for up to 96 weeks	Drug: NBI-98854; vesicular monoamine transporter 2 (VMAT2) inhibitor; Other Names: Ingrezza; Valbenazine

Outcome Measures

Primary Outcome Measures :

1. Number of Participants with Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: Up to 96 Weeks ]

Secondary Outcome Measures :

1. Severity of Tic Symptoms as Assessed by Clinical Global Impression of Tics-Severity scale (CGI-Tics-Severity) [ Time Frame: Up to 96 Weeks ]
   The CGI-Tics Severity scale assesses overall severity on a 7-point scale (range; 1=normal, not at all ill to 7=among the most extremely ill subjects).
2. Improvement of Tic Symptoms as Assessed by Clinical Global Impression of Tourette Syndrome-Improvement scale (CGI TS-Improvement) [ Time Frame: Up to 96 Weeks ]
   The CGI TS-Improvement scale assesses overall improvement since the initiation of study drug dosing on a 7-point scale (range; 1=very much improved to 7=very much worse).
3. Improvement of quality of life as assessed by Gilles de la Tourette Syndrome-Quality of Life for Children and Adolescents (C&A GTS QOL) [ Time Frame: Up to 96 Weeks ]
   The C&A GTS QOL consists of 27 items and 4 subscales (psychological, physical, obsessive-compulsive, and cognitive). Each item is rated across 5 response options: "Never," "Rarely," "Sometimes," "Often," and "Always."

Eligibility Criteria

• Ages Eligible for Study: 6 Years to 18 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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)

Contacts and Locations

Locations

United States, California

Neurocrine Clinical Site

Anaheim, California, United States, 92805

Neurocrine Clinical Site

San Diego, California, United States, 92108

United States, Florida

Neurocrine Clinical Site

Gulf Breeze, Florida, United States, 32561

Neurocrine Clinical Site

Hialeah, Florida, United States, 33013

United States, Illinois

Neurocrine Clinical Site

Chicago, Illinois, United States, 60634

Neurocrine Clinical Site

Naperville, Illinois, United States, 60563

United States, Nebraska

Neurocrine Clinical Site

Lincoln, Nebraska, United States, 68526

United States, Texas

Neurocrine Clinical Site

Dallas, Texas, United States, 75243

Neurocrine Clinical Site

Houston, Texas, United States, 77058

Puerto Rico

Neurocrine Clinical Site

San Juan, Puerto Rico, 00926

Sponsors and Collaborators

Neurocrine Biosciences

Investigators

• Study Director: Chief Medical Officer; Chief Medical Officer

More Information

• Responsible Party: Neurocrine Biosciences
• ClinicalTrials.gov Identifier: NCT03732534
• Other Study ID Numbers: NBI-98854-TS2007
• First Posted: November 6, 2018
• Last Update Posted: February 17, 2020
• Last Verified: February 2020

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Tourette Syndrome; Syndrome; Disease; Pathologic Processes; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tic Disorders; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Neurodevelopmental Disorders; Mental Disorders