Safety and Tolerability Study of NBI-98854 for the Treatment of Subjects With Tourette Syndrome

Neurocrine Biosciences

Status and phase: Completed; Phase 2

Conditions: Tourette Syndrome

Treatments: Drug: Valbenazine

Study type: Interventional

Funder types: Industry

Identifiers

NCT02879578

NBI-98854-1601

Details and patient eligibility

About

Phase 2, open-label, fixed-dose titration study to evaluate the safety and tolerability of NBI-98854 administered once daily for a total of 24 weeks in children, adolescents, and adults with Tourette Syndrome (TS).

Enrollment: 155 patients

Sex: All

Ages: 6 to 64 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Have participated in and completed the NBI-98854-1501 (T-Force Green) or NBI-98854-1505 (T-Forward) Phase 2 study
• Have a clinical diagnosis of Tourette Syndrome (TS)
• If using maintenance medication(s) for TS or TS spectrum diagnoses (e.g. obsessive-compulsive disorder [OCD], Attention-Deficit Hyperactivity Disorder [ADHD]), be on stable doses
• 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
• Adolescent and adult subjects (12 to 64 years of age) must have a negative urine drug screen for amphetamines, barbiturates, benzodiazepine, phencyclidine, cocaine, opiates, or cannabinoids and a negative alcohol screen. Subjects who are on stable doses of prescribed and supervised (not as needed) benzodiazepines, opiates, or psychostimulants (for subjects with comorbid ADHD) are allowed to participate in the study
• Be in good general health

Exclusion criteria

• Have an active, clinically significant unstable medical condition within 1 month prior to 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 a blood loss ≥250 mL or donated blood within 56 days prior to screening (subjects 6 to 17 years of age); have a blood loss ≥550 mL or donated blood within 30 days prior to screening (subjects 18 to 64 years of age)
• Have a known history of substance dependence, substance (drug) or alcohol abuse
• Have a significant risk of suicidal or violent behavior
• Have initiated Comprehensive Behavioral Intervention for Tics (CBIT) during the screening period or at baseline or plan to initiate CBIT during the study
• Have received an investigational drug within 30 days before screening or plan to use an investigational drug (other than NBI-98854) during the study

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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155 participants in 3 patient groups

Valbenazine (Children)

Experimental group

Description:

Children (6 to 11 years of age) received valbenazine 10 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 20 mg or continue with the participant's current dose for the remainder of the treatment period.

Treatment:

Drug: Valbenazine

Valbenazine (Adolescents)

Experimental group

Description:

Adolescents (12 to 17 years of age) received valbenazine 20 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 40 mg or continue with the participant's current dose for the remainder of the treatment period.

Treatment:

Drug: Valbenazine

Valbenazine (Adults)

Experimental group

Description:

Adults (18 to 64 years of age) received valbenazine 40 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 80 mg or continue with the participant's current dose for the remainder of the treatment period.

Treatment:

Drug: Valbenazine