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

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

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)

Study Overview

• Status: Completed
• Conditions: Huntington Disease
• Intervention / Treatment: Drug: SAGE-718

Detailed Description

This posting addresses Part B

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Long Beach, California, United States, 90806
      • Sage Investigational Site
  • New Jersey
    • Marlton, New Jersey, United States, 08053
      • Sage Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 66 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: SAGE-718	Drug: SAGE-718; SAGE-718

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Supernus Pharmaceuticals, Inc.

Publications and helpful links

General Publications

• Koenig A, Lewis M, Wald J, Li S, Varoglu M, Dai J, Sankoh A, Paumier K, Doherty J, Quirk M. Dalzanemdor (SAGE-718), a novel, investigational N-methyl-D-aspartate receptor positive allosteric modulator: Safety, tolerability, and clinical pharmacology in randomized dose-finding studies in healthy participants and an open-label study in participants with Huntington's disease. Clin Transl Sci. 2024 Jul;17(7):e13852. doi: 10.1111/cts.13852.

Study record dates

Study Major Dates

• Study Start (Actual): February 28, 2019
• Primary Completion (Actual): October 7, 2019
• Study Completion (Actual): October 7, 2019

Study Registration Dates

• First Submitted: December 17, 2018
• First Submitted That Met QC Criteria: December 21, 2018
• First Posted (Actual): December 26, 2018

Study Record Updates

• Last Update Posted (Estimated): September 17, 2025
• Last Update Submitted That Met QC Criteria: September 11, 2025
• Last Verified: January 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Mental Disorders; Genetic Diseases, Inborn; Neurocognitive Disorders; Cognition Disorders; Dementia; Neurodegenerative Diseases; Movement Disorders; Heredodegenerative Disorders, Nervous System; Basal Ganglia Diseases; Dyskinesias; Chorea; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Huntington Disease
• Other Study ID Numbers: 718-CLP-102 B

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Data sharing will be consistent with the results submission policy of ClinicalTrials.gov.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No