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NCT Number: NCT03787758

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

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)

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Key information

Conditions

Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Chorea Cognition Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dementia Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Mental Disorders Movement Disorders Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Sage Investigational Site, Long Beach, California, United States

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About this study

This posting addresses Part B

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

SAGE-718

Drug

SAGE-718

Primary outcomes

  1. Number of Participants with the Incidence of Adverse Events and Serious Adverse Events.

    Time frame: 21 Days

  2. Percentage of participants with change from baseline in vital signs.

    Time frame: 21 Days

  3. Change from baseline in electrocardiograms (ECGs) including PR interval, QT interval, QTc interval, QTcF, and rhythm abnormalities

    Time frame: 21 Days

  4. Percentage of participants with change from baseline in clinical laboratory parameters.

    Time frame: 21 Days

  5. Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS).

    Time frame: 21 Days

Secondary outcomes

  1. PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by area under the curve [AUC].

    Time frame: 17 Days

  2. PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by maximum observed concentration [Cmax].

    Time frame: 17 Days

  3. PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by time of occurrence of Cmax [tmax].

    Time frame: 17 Days

Sponsors and collaborators

Lead sponsor

Supernus Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2019
Study completion
2019
First posted
Dec 26, 2018
Registry last updated
Sep 17, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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