Huntington Disease
Conditions
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
Interventions
DRUGSAGE-718
SAGE-718
Sponsors
Supernus Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Subject is positive for mutant HTT (documented CAG repeats ≥ 40 units). 2. Subject has a body weight ≥50 kg and body mass index ≥18.0 and ≤30.0 kg/m2 at screening.
Exclusion criteria
1. Subject has any clinically significant abnormal finding on the physical exam at screening or admission. 2. 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. 3. Subject has a family history of epilepsy. 4. Subject has a positive screening test for alcohol or drugs of abuse (including marijuana) at screening or admission.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS). | 21 Days |
| Percentage of participants with change from baseline in vital signs. | 21 Days |
| Change from baseline in electrocardiograms (ECGs) including PR interval, QT interval, QTc interval, QTcF, and rhythm abnormalities | 21 Days |
| Percentage of participants with change from baseline in clinical laboratory parameters. | 21 Days |
| Number of Participants with the Incidence of Adverse Events and Serious Adverse Events. | 21 Days |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
| 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 |
Countries
United States
Outcome results
None listed