A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)

NCT ID: NCT05107128

Last Updated: 2025-09-15

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

189 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-01-26

Study Completion Date

2024-10-03

Brief Summary

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The primary purpose of this study is to evaluate the effect of SAGE-718 on cognitive performance and functioning in participants with HD.

Detailed Description

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Conditions

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Huntington's Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Placebo

Participants will receive SAGE-718-matching placebo, orally, once daily (QD) for up to Day 84.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

SAGE-718-matching oral capsules.

SAGE-718

Participants will receive SAGE-718, 1.2 milligrams (mg), capsules, orally, QD for Days 1 to 27, followed by 0.9 mg for the remainder of the treatment period up to Day 84.

Group Type EXPERIMENTAL

SAGE-718

Intervention Type DRUG

Oral capsules.

Interventions

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SAGE-718

Oral capsules.

Intervention Type DRUG

Placebo

SAGE-718-matching oral capsules.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Meet all the following criteria for HD at Screening (Days -28 to -2):

1. Genetically confirmed disease with huntingtin gene CAG expansion greater than or equal to (≥) 36.
2. At screening, Unified Huntington's Disease Rating Scale (UHDRS)-Total Functional Capacity (TFC) score greater than (\>) 6 and less than (\<)13, suggesting no more than a moderate level of functional impairment.
3. No features of juvenile HD.
2. Score of 15 to 25 (inclusive) on the Montreal Cognitive Assessment (MoCA) at screening indicating the presence of cognitive impairment.
3. Be willing to invite a study partner, if available, who is reliable, competent, and at least 18 years of age to participate in the study.
4. Be ambulatory (use of assistance devices such as a walker or cane is acceptable, as is occasional use of wheelchair, as judged by the investigator. Individuals requiring a wheelchair on a regular basis are excluded), able to travel to the study center, and, as judged by the investigator, is likely to be able to continue to travel to the study center to complete study visits for the duration of the study.
5. Completion of Huntington's Disease Cognitive Assessment Battery (HD-CAB) Trail Making-B Test in less than 240 seconds at Screening (Days -28 to -2).

Exclusion Criteria

1. Have participated in a previous clinical study of SAGE-718, have previous exposure to gene therapy, have participated in any HD investigational drug, biologic, or device trial within 180 days, or a non-HD drug, biologic, or device trial within 30 days or 5 half-lives (whichever is longer).

(Note: Participants with confirmation of enrollment in the placebo arm of these trials would not be excluded.)
2. Have a diagnosis of an ongoing neurodegenerative condition other than HD, including but not limited to Alzheimer's Disease, vascular dementia, dementia with Lewy bodies, or Parkinson's Disease.
Minimum Eligible Age

25 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Supernus Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Sage Investigational Site

Little Rock, Arkansas, United States

Site Status

Sage Investigational Site

La Jolla, California, United States

Site Status

Sage Investigational Site

Los Angeles, California, United States

Site Status

Sage Investigational Site

Sacramento, California, United States

Site Status

Sage Investigational Site

Englewood, Colorado, United States

Site Status

Sage Investigational Site

Washington D.C., District of Columbia, United States

Site Status

Sage Investigational Site

Boca Raton, Florida, United States

Site Status

Sage Investigational Site

Boca Raton, Florida, United States

Site Status

Sage Investigational Site

Tampa, Florida, United States

Site Status

Sage Investigational Site

Honolulu, Hawaii, United States

Site Status

Sage Investigational Site

Chicago, Illinois, United States

Site Status

Sage Investigational Site

Chicago, Illinois, United States

Site Status

Sage Investigational Site

Indianapolis, Indiana, United States

Site Status

Sage Investigational Site

Iowa City, Iowa, United States

Site Status

Sage Investigational Site

Kansas City, Kansas, United States

Site Status

Sage Investigational Site

Baltimore, Maryland, United States

Site Status

Sage Investigational Site

Boston, Massachusetts, United States

Site Status

Sage Investigational Site

Boston, Massachusetts, United States

Site Status

Sage Investigational Site

Farmington Hills, Michigan, United States

Site Status

Sage Investigational Site

Buffalo, New York, United States

Site Status

Sage Investigational Site

New York, New York, United States

Site Status

Sage Investigational Site

Chapel Hill, North Carolina, United States

Site Status

Sage Investigational Site

Durham, North Carolina, United States

Site Status

Sage Investigational Site

Cincinnati, Ohio, United States

Site Status

Sage Investigational Site

Toledo, Ohio, United States

Site Status

Sage Investigational Site

Philadelphia, Pennsylvania, United States

Site Status

Sage Investigational Site

Charleston, South Carolina, United States

Site Status

Sage Investigational Site

Memphis, Tennessee, United States

Site Status

Sage Investigational Site

Nashville, Tennessee, United States

Site Status

Sage Investigational Site

Houston, Texas, United States

Site Status

Sage Investigational Site

Richmond, Virginia, United States

Site Status

Sage Investigational Site

Kirkland, Washington, United States

Site Status

Sage Investigational Site

Spokane, Washington, United States

Site Status

Sage Investigational Site

Madison, Wisconsin, United States

Site Status

Sage Investigational Site

Westmead, New South Wales, Australia

Site Status

Sage Investigational Site

Herston, Queensland, Australia

Site Status

Sage Investigational Site

Bethlehem, Victoria, Australia

Site Status

Sage Investigational Site

Nedlands, Western Australia, Australia

Site Status

Sage Investigational Site

Halifax, Nova Scotia, Canada

Site Status

Sage Investigational Site

North York, Ontario, Canada

Site Status

Sage Investigational Site

Montreal, Quebec, Canada

Site Status

Sage Investigational Site

Tooting, London, United Kingdom

Site Status

Sage Investigational Site

Aberdeen, , United Kingdom

Site Status

Sage Investigational Site

Birmingham, , United Kingdom

Site Status

Sage Investigational Site

Cardiff, , United Kingdom

Site Status

Sage Investigational Site

Leeds, , United Kingdom

Site Status

Sage Investigational Site

Newcastle upon Tyne, , United Kingdom

Site Status

Sage Investigational Site

Plymouth, , United Kingdom

Site Status

Sage Investigational Site

Southampton, , United Kingdom

Site Status

Countries

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United States Australia Canada United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2021-005577-16

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

718-CIH-201

Identifier Type: -

Identifier Source: org_study_id

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