A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Patients With Huntington's Disease - Part B

Study Overview
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Brief Summary
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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
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This posting addresses Part B

Official Title
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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

Conditions
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Huntington Disease

Intervention / Treatment
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* Drug: SAGE-718


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: SAGE-718<br> | Drug: SAGE-718<br>* SAGE-718<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants with the Incidence of Adverse Events and Serious Adverse Events. |  | 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 | 
| Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS). |  | 21 Days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 |