A Phase 1, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Determine the Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Healthy Adults With an Open-label Cohort of Patients With Huntington's Disease
Overview
- Phase
- Phase 1
- Intervention
- SAGE-718
- Conditions
- Huntington Disease
- Sponsor
- Supernus Pharmaceuticals, Inc.
- Enrollment
- 6
- Locations
- 1
- Primary Endpoint
- Number of Participants with the Incidence of Adverse Events and Serious Adverse Events.
- Status
- Completed
- Last Updated
- 7 months ago
Overview
Brief Summary
This study is a phase 1, double-blind, placebo-controlled, multiple ascending dose study to determine the safety, tolerability, and pharmacokinetics of SAGE-718 oral solution in healthy adults (Part A) with an open-label cohort of patients with Huntington's disease (Part B)
Detailed Description
This posting addresses Part B
Investigators
Eligibility Criteria
Inclusion Criteria
- •Subject is positive for mutant HTT (documented CAG repeats ≥ 40 units).
- •Subject has a body weight ≥50 kg and body mass index ≥18.0 and ≤30.0 kg/m2 at screening.
Exclusion Criteria
- •Subject has any clinically significant abnormal finding on the physical exam at screening or admission.
- •Subject has a history or presence of a neurologic disease or condition (other than Huntington's disease), including but not limited to severe chorea, epilepsy, closed head trauma with clinically significant sequelae, or a prior seizure.
- •Subject has a family history of epilepsy.
- •Subject has a positive screening test for alcohol or drugs of abuse (including marijuana) at screening or admission.
Arms & Interventions
SAGE-718
Intervention: SAGE-718
Outcomes
Primary Outcomes
Number of Participants with the Incidence of Adverse Events and Serious Adverse Events.
Time Frame: 21 Days
Percentage of participants with change from baseline in vital signs.
Time Frame: 21 Days
Percentage of participants with change from baseline in clinical laboratory parameters.
Time Frame: 21 Days
Change from baseline in electrocardiograms (ECGs) including PR interval, QT interval, QTc interval, QTcF, and rhythm abnormalities
Time Frame: 21 Days
Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS).
Time Frame: 21 Days
Secondary Outcomes
- PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by area under the curve [AUC].(17 Days)
- PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by maximum observed concentration [Cmax].(17 Days)
- PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by time of occurrence of Cmax [tmax].(17 Days)