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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05107128
Recruitment Status : Recruiting
First Posted : November 4, 2021
Last Update Posted : November 14, 2022
Sponsor:
Information provided by (Responsible Party):
Sage Therapeutics

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

Condition or disease Intervention/treatment Phase
Huntington's Disease Drug: SAGE-718 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 178 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease
Actual Study Start Date : February 10, 2022
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: SAGE-718
Participants will receive SAGE-718, once daily for 84 days.
Drug: SAGE-718
Oral capsules.

Placebo Comparator: Placebo
Participants will receive placebo, once daily for 84 days.
Drug: Placebo
SAGE-718-matching oral capsules.




Primary Outcome Measures :
  1. Change From Baseline in the Huntington's Disease Cognitive Assessment Battery (HD-CAB) Composite Score [ Time Frame: Baseline and Day 84 ]
    The HD-CAB evaluates cognitive function via the following subtests: Symbol Digit Modalities Test; One Touch Stockings of Cambridge; Trail Making; Hopkins Verbal Learning Test Revised; Paced Tapping Test; and the Emotion Recognition Test. The HD-CAB composite is derived by transforming individual participants' scores on each of the 6 subtests to z-scores, and averaging z-scores to represent global performance relative to the reference sample. Change from baseline will be computed using baseline mean and standard deviation, with an expected z-score distribution of approximately -3 to +3. A positive HD-CAB composite at follow-up indicates improvement in cognitive function; a negative composite score indicates worsening in cognitive function; and a composite of "0" would reflect no change relative to baseline.


Secondary Outcome Measures :
  1. Change From Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) - Independence Scale [ Time Frame: Baseline and Day 84 ]
    The UHDRS independence scale is a single item of independence rated from 10 to 100, with higher scores on the function scales indicating better functioning than lower scores.

  2. Change From Baseline in the UHDRS - Total Motor Score (TMS) [ Time Frame: Baseline and Day 84 ]
    The motor section of the UHDRS assesses motor features of HD with standardized ratings of oculomotor function, dysarthria, chorea, dystonia, gait, and postural stability. Each item is scored on a scale of 0-4, where 0 is "normal," and 4 is the "highest motor dysfunction." The total motor impairment score is the sum of all the individual motor ratings. The total score range is 0-24, with higher scores indicating more severe motor impairment than lower scores.

  3. Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Up to approximately 112 days ]
    An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product that does not necessarily have a causal relationship with this treatment. A TEAE is defined as an AE with onset after the start of the investigational product, or any worsening of a pre-existing medical condition/AE with onset after the start of the investigational product and throughout the study.



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Ages Eligible for Study:   25 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Meet all the following criteria for HD:

    1. Genetically confirmed disease with huntingtin gene CAG expansion ≥36.
    2. UHDRS-Total Functional Capacity (TFC) score >6 and <13.
    3. No features of juvenile HD.
  2. Score <26 on the Montreal Cognitive Assessment (MoCA) at screening.
  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.

Exclusion Criteria:

  1. Have participated in a previous clinical study of SAGE-718, have participated in a previous gene therapy study, or have participated in any other drug, biologic, or device trial within 30 days or 5 half-lives (whichever is longer), unless the patient participated solely in the placebo arm of the study. Additionally, participants who have received treatment with antisense oligonucleotides (ASOs) or an mRNA splicing modifier will 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05107128


Contacts
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Contact: Emily Lefler, PharmD 619-630-4802 emily.lefler@sagerx.com

Locations
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United States, California
Sage Investigational Site Recruiting
La Jolla, California, United States, 92037
Contact       clinicaltrialsinquiry@sagerx.com   
Sage Investigational Site Recruiting
Los Angeles, California, United States, 90095
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Colorado
Sage Investigational Site Recruiting
Englewood, Colorado, United States, 80113
Contact       clinicaltrialsinquiry@sagerx.com   
United States, District of Columbia
Sage Investigational Site Recruiting
Washington, District of Columbia, United States, 20007
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Florida
Sage Investigational Site Recruiting
Boca Raton, Florida, United States, 33431
Contact       clinicaltrialsinquiry@sagerx.com   
Sage Investigational Site Recruiting
Boca Raton, Florida, United States, 33486
Contact       clinicaltrialsinquiry@sagerx.com   
Sage Investigational Site Recruiting
Tampa, Florida, United States, 33612
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Hawaii
Sage Investigational Site Recruiting
Honolulu, Hawaii, United States, 96817
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Illinois
Sage Investigational Site Recruiting
Chicago, Illinois, United States, 60611
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Indiana
Sage Investigational Site Recruiting
Indianapolis, Indiana, United States, 46202
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Iowa
Sage Investigational Site Recruiting
Iowa City, Iowa, United States, 52242
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Maryland
Sage Investigational Site Recruiting
Baltimore, Maryland, United States, 21287
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Massachusetts
Sage Investigational Site Recruiting
Boston, Massachusetts, United States, 02215
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Michigan
Sage Investigational Site Recruiting
Farmington Hills, Michigan, United States, 48334
Contact       clinicaltrialsinquiry@sagerx.com   
United States, New York
Sage Investigational Site Recruiting
Williamsville, New York, United States, 14221
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Ohio
Sage Investigational Site Recruiting
Cincinnati, Ohio, United States, 45219
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Pennsylvania
Sage Investigational Site Recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact       clinicaltrialsinquiry@sagerx.com   
United States, South Carolina
Sage Investigational Site Recruiting
Charleston, South Carolina, United States, 29425
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Tennessee
Sage Investigational Site Recruiting
Memphis, Tennessee, United States, 38137
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Texas
Sage Investigational Site Recruiting
Houston, Texas, United States, 77030
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Virginia
Sage Investigational Site Recruiting
Richmond, Virginia, United States, 23219
Contact       clinicaltrialsinquiry@sagerx.com   
United States, Washington
Sage Investigational Site Recruiting
Kirkland, Washington, United States, 98034
Contact       clinicaltrialsinquiry@sagerx.com   
Sage Investigational Site Recruiting
Spokane, Washington, United States, 99202
Contact       clinicaltrialsinquiry@sagerx.com   
Australia, New South Wales
Sage Investigational Site Recruiting
Westmead, New South Wales, Australia, 2145
Contact       clinicaltrialsinquiry@sagerx.com   
Australia, Western Australia
Sage Investigational Site Recruiting
Nedlands, Western Australia, Australia, 6009
Contact       clinicaltrialsinquiry@sagerx.com   
Canada, Ontario
Sage Investigational Site Recruiting
North York, Ontario, Canada, M2K 1E1
Contact       clinicaltrialsinquiry@sagerx.com   
United Kingdom
Sage Investigational Site Recruiting
Plymouth, United Kingdom, PL6 8BT
Contact       clinicaltrialsinquiry@sagerx.com   
Sponsors and Collaborators
Sage Therapeutics
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Responsible Party: Sage Therapeutics
ClinicalTrials.gov Identifier: NCT05107128    
Other Study ID Numbers: 718-CIH-201
First Posted: November 4, 2021    Key Record Dates
Last Update Posted: November 14, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Data sharing will be consistent with the results submission policy of ClinicalTrials.gov.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Chorea
Dyskinesias
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Neurocognitive Disorders
Mental Disorders