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

Primary Purpose

Huntington's Disease

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
SAGE-718
Placebo
Sponsored by
Sage Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Huntington's Disease

Eligibility Criteria

25 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
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  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
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  • Sage Investigational SiteRecruiting
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  • Sage Investigational SiteRecruiting
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  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting
  • Sage Investigational SiteRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

SAGE-718

Placebo

Arm Description

Participants will receive SAGE-718, once daily for 84 days.

Participants will receive placebo, once daily for 84 days.

Outcomes

Primary Outcome Measures

Change From Baseline in the Huntington's Disease Cognitive Assessment Battery (HD-CAB) Composite Score
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

Change From Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) - Independence Scale
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.
Change From Baseline in the UHDRS - Total Motor Score (TMS)
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.
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
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.

Full Information

First Posted
October 25, 2021
Last Updated
September 26, 2023
Sponsor
Sage Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT05107128
Brief Title
A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)
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
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
February 10, 2022 (Actual)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
December 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sage Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Huntington's Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
178 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
SAGE-718
Arm Type
Experimental
Arm Description
Participants will receive SAGE-718, once daily for 84 days.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants will receive placebo, once daily for 84 days.
Intervention Type
Drug
Intervention Name(s)
SAGE-718
Intervention Description
Oral capsules.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
SAGE-718-matching oral capsules.
Primary Outcome Measure Information:
Title
Change From Baseline in the Huntington's Disease Cognitive Assessment Battery (HD-CAB) Composite Score
Description
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.
Time Frame
Baseline and Day 84
Secondary Outcome Measure Information:
Title
Change From Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) - Independence Scale
Description
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.
Time Frame
Baseline and Day 84
Title
Change From Baseline in the UHDRS - Total Motor Score (TMS)
Description
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.
Time Frame
Baseline and Day 84
Title
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
Description
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.
Time Frame
Up to approximately 112 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
25 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Meet all the following criteria for HD: Genetically confirmed disease with huntingtin gene CAG expansion ≥36. UHDRS-Total Functional Capacity (TFC) score >6 and <13. No features of juvenile HD. Score <26 on the Montreal Cognitive Assessment (MoCA) at screening. 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 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: 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. 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Grant Rutledge, PhD
Phone
339-368-8432
Email
grant.rutledge@sagerx.com
Facility Information:
Facility Name
Sage Investigational Site
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72205
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Englewood
State/Province
Colorado
ZIP/Postal Code
80113
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Boca Raton
State/Province
Florida
ZIP/Postal Code
33431
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Honolulu
State/Province
Hawaii
ZIP/Postal Code
96817
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Williamsville
State/Province
New York
ZIP/Postal Code
14221
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagex.com
Facility Name
Sage Investigational Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45219
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43614
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38157
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Kirkland
State/Province
Washington
ZIP/Postal Code
98034
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Spokane
State/Province
Washington
ZIP/Postal Code
99202
Country
United States
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Westmead
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Herston
State/Province
Queensland
ZIP/Postal Code
4029
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Parkdale
State/Province
Victoria
ZIP/Postal Code
3195
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Nedlands
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Halifax
State/Province
Nova Scotia
ZIP/Postal Code
B3S 1N2
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
North York
State/Province
Ontario
ZIP/Postal Code
M2K 1E1
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H2X 0A9
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Tooting
State/Province
London
ZIP/Postal Code
SW17 0QT
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Aberdeen
ZIP/Postal Code
AB25 2ZA
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Birmingham
ZIP/Postal Code
B15 2FG
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Cardiff
ZIP/Postal Code
CF10 3AX
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Leeds
ZIP/Postal Code
LS1 3EX
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Leeds
ZIP/Postal Code
LS7 4SA
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Newcastle Upon Tyne
ZIP/Postal Code
NE6 4QD
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Plymouth
ZIP/Postal Code
PL6 8BT
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com
Facility Name
Sage Investigational Site
City
Southampton
ZIP/Postal Code
SO16 6YD
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
Email
clinicaltrialsinquiry@sagerx.com

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
Data sharing will be consistent with the results submission policy of ClinicalTrials.gov.

Learn more about this trial

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

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