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

Neurocrine Biosciences

Status and phase: Terminated; Phase 2

Conditions: Tourette Syndrome

Treatments: Drug: NBI-98854

Study type: Interventional

Funder types: Industry

Identifiers

NCT03732534

NBI-98854-TS2007

Details and patient eligibility

About

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.

Enrollment: 6 patients

Sex: All

Ages: 6 to 18 years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

6 participants in 1 patient group

NBI-98854

Experimental group

Description:

NBI-98854 administered once daily for up to 96 weeks

Treatment:

Drug: NBI-98854