A Study to Evaluate the Safety and Tolerability of SAGE-718 in Participants With Huntington's Disease
NCT ID: NCT05655520
Last Updated: 2025-12-17
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
153 participants
INTERVENTIONAL
2022-12-14
2025-01-20
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Cohort 1 (Direct Rollover)
Participants from the studies 718-CIH-201 (NCT05107128) and 718-CIH-202 (NCT05358821) who will sign the informed consent for study 718-CIH-301 ≤7 days after the last day of the corresponding parent study will be enrolled in this cohort. Participants will receive Sage-718, 0.9 milligrams (mg), orally once daily from Day 1 onwards.
SAGE-718
Oral softgel lipid capsules
Cohort 2 (Gap Rollover)
Participants from the studies 718-CIH-201 (NCT05107128) and 718-CIH-202 (NCT05358821) who will sign the informed consent for study 718-CIH-301 after a gap of \>7 days after the last day of the corresponding parent study will be enrolled in this cohort. Participants will receive Sage-718 0.9 mg, orally once daily from Day 1 onwards.
SAGE-718
Oral softgel lipid capsules
Cohort 3 (De Novo)
Participants who were not previously included in any SAGE-718 clinical study. Participants will receive Sage-718 from Day 1 onwards.
SAGE-718
Oral softgel lipid capsules
Interventions
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SAGE-718
Oral softgel lipid capsules
Eligibility Criteria
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Inclusion Criteria
* Completed 718-CIH-201 (NCT05107128) or 718-CIH-202 (NCT05358821) studies or meet eligibility criteria for the de novo cohort.
* Agree to refrain from drugs of abuse for the duration of the study and from alcohol during the 48 hours preceding each study visit.
* 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.
* Be able to travel to the study center, and, 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.
* Be at least 25 years old, but not older than 65 years of age at Screening.
* Genetically confirmed disease with cytosine-adenine-guanine (CAG) expansion ≥40
* No features of juvenile HD
* CAG-Age-Product (CAP) score ≥90, as calculated using the CAP formula: AGE × (CAG - 30) / 6.49.
* At screening, scores of either: a) Unified Huntington's Disease Rating Scale (UHDRS) -Total Functional Capacity (TFC)=13 and Montreal Cognitive Assessment (MoCA) ≤25 score, or b) UHDRS-TFC ≤12 and MoCA \>25
Exclusion Criteria
* 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.
* Had gastric bypass surgery, has a gastric sleeve or lap band, or has had any related procedures that interfere with gastrointestinal transit.
* Is known to be allergic to any of SAGE-718 excipients, including soy lecithin.
* Receive any prohibited medications within 30 days of screening and during participation in the study.
* Have previous exposure to gene therapy, or have participated in any other 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). Additionally, participants who have received treatment with antisense oligonucleotides or a messenger ribonucleic acid (mRNA) splicing modifier will be excluded.
Note: Participants with confirmation of enrolment in the placebo arm of these investigational trials would not be excluded.
* Have one or more ongoing serious adverse events (SAEs) from the parent study.
* Have ongoing, unresolved AE(s), which in the opinion of the investigator or sponsor, is likely to interfere with study conduct or compliance.
25 Years
65 Years
ALL
No
Sponsors
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Supernus Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Sage Investigational Site
Birmingham, Alabama, United States
Sage Investigational Site
Little Rock, Arkansas, United States
Sage Investigational Site
La Jolla, California, United States
Sage Investigational Site
Los Alamitos, California, United States
Sage Investigational Site
Los Angeles, California, United States
Sage Investigational Site
Sacramento, California, United States
Sage Investigational Site
Englewood, Colorado, United States
Sage Investigational Site
Washington D.C., District of Columbia, United States
Sage Investigational Site
Boca Raton, Florida, United States
Sage Investigational Site
Tampa, Florida, United States
Sage Investigational Site
Chicago, Illinois, United States
Sage Investigational Site
Chicago, Illinois, United States
Sage Investigational Site
Indianapolis, Indiana, United States
Sage Investigational Site
Iowa City, Iowa, United States
Sage Investigational Site
Kansas City, Kansas, United States
Sage Investigational Site
Baltimore, Maryland, United States
Sage Investigational Site
Boston, Massachusetts, United States
Sage Investigational Site
New York, New York, United States
Sage Investigational Site
Williamsville, New York, United States
Sage Investigational Site
Chapel Hill, North Carolina, United States
Sage Investigational Site
Durham, North Carolina, United States
Sage Investigational Site
Cincinnati, Ohio, United States
Sage Investigational Site
Toledo, Ohio, United States
Sage Investigational Site
Philadelphia, Pennsylvania, United States
Sage Investigational Site
Charleston, South Carolina, United States
Sage Investigational Site
Memphis, Tennessee, United States
Sage Investigational Site
Houston, Texas, United States
Sage Investigational Site
Richmond, Virginia, United States
Sage Investigational Site
Spokane, Washington, United States
Sage Investigational Site
Westmead, New South Wales, Australia
Sage Investigational Site
Caulfield South, Victoria, Australia
Sage Investigational Site
Nedlands, Western Australia, Australia
Sage Investigational Site
Halifax, Nova Scotia, Canada
Sage Investigational Site
Toronto, Ontario, Canada
Sage Investigational Site
Montreal, Quebec, Canada
Sage Investigational Site
Plymouth, Derriford, United Kingdom
Sage Investigational Site
Southampton, England, United Kingdom
Sage Investigational Site
Tooting, London, United Kingdom
Sage Investigational Site
Aberdeen, Scotland, United Kingdom
Sage Investigational Site
Newcastle, Upon Tyne, United Kingdom
Sage Investigational Site
Cardiff, Wales, United Kingdom
Sage Investigational Site
Birmingham, West Midlands, United Kingdom
Sage Investigational Site
Leeds, West Yorkshire, United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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718-CIH-301
Identifier Type: -
Identifier Source: org_study_id