Clinical trial NCT05107128: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Huntington's Disease
Lead sponsor: Sage Therapeutics
Sponsor class: INDUSTRY
First posted: 2021-11-04
Enrollment: 189
Sex: ALL
Minimum age (years): 25
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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 ≥36.
   2. At screening, UHDRS-Total Functional Capacity (TFC) score \&amp;amp;gt;6 and \&amp;amp;lt;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.

Primary outcomes:
measure: Change From Baseline in the Symbol Digit Modalities Test (SDMT)
timeFrame: Baseline and Day 84
Secondary outcomes:
measure: Change From Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) - Independence Scale
timeFrame: Baseline and Day 84
measure: Change From Baseline in the Trial Making Test Part B
timeFrame: Baseline and Day 84
measure: Change From Baseline in the One Touch Stockings of Cambridge (OTS-Mean Latency until Correct response)
timeFrame: Baseline and Day 84
measure: Change From Baseline in the Paced Tapping Test (PTAP)
timeFrame: Baseline and Day 84
measure: Change From Baseline in the Huntington's Disease Everyday Functioning (Hi-DEF) Home Subdomain Score
timeFrame: Baseline and Day 84
measure: Change From Baseline in the Clinical Global Impression - Severity (CGI-S) Cognitive Status Subdomain Score
timeFrame: Baseline and Day 84
measure: Proportion of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: Up to approximately 112 days
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Sage Investigational Site
state: Arkansas
city: La Jolla
country: United States
facility: Sage Investigational Site
state: California
city: Los Angeles
country: United States
facility: Sage Investigational Site
state: California
city: Sacramento
country: United States
facility: Sage Investigational Site
state: California
city: Englewood
country: United States
facility: Sage Investigational Site
state: Colorado
city: Washington
country: United States
facility: Sage Investigational Site
state: District of Columbia
city: Boca Raton
country: United States
facility: Sage Investigational Site
state: Florida
city: Tampa
country: United States
facility: Sage Investigational Site
state: Florida
city: Honolulu
country: United States
facility: Sage Investigational Site
state: Hawaii
city: Chicago
country: United States
facility: Sage Investigational Site
state: Illinois
city: Chicago
country: United States
facility: Sage Investigational Site
state: Illinois
city: Indianapolis
country: United States
facility: Sage Investigational Site
state: Indiana
city: Iowa City
country: United States
facility: Sage Investigational Site
state: Iowa
city: Kansas City
country: United States
facility: Sage Investigational Site
state: Kansas
city: Baltimore
country: United States
facility: Sage Investigational Site
state: Maryland
city: Boston
country: United States
facility: Sage Investigational Site
state: Massachusetts
city: New York
country: United States
facility: Sage Investigational Site
state: New York
city: Williamsville
country: United States
facility: Sage Investigational Site
state: New York
city: Chapel Hill
country: United States
facility: Sage Investigational Site
state: North Carolina
city: Durham
country: United States
facility: Sage Investigational Site
state: North Carolina
city: Cincinnati
country: United States
facility: Sage Investigational Site
state: Ohio
city: Toledo
country: United States
facility: Sage Investigational Site
state: Ohio
city: Philadelphia
country: United States
facility: Sage Investigational Site
state: Pennsylvania
city: Charleston
country: United States
facility: Sage Investigational Site
state: South Carolina
city: Memphis
country: United States
facility: Sage Investigational Site
state: Tennessee
city: Houston
country: United States
facility: Sage Investigational Site
state: Texas
city: Richmond
country: United States
facility: Sage Investigational Site
state: Virginia
city: Kirkland
country: United States
facility: Sage Investigational Site
state: Washington
city: Spokane
country: United States
facility: Sage Investigational Site
state: Washington
city: Westmead
country: Australia
facility: Sage Investigational Site
state: New South Wales
city: Herston
country: Australia
facility: Sage Investigational Site
state: Queensland
city: Parkdale
country: Australia
facility: Sage Investigational Site
state: Victoria
city: Nedlands
country: Australia
facility: Sage Investigational Site
state: Western Australia
city: Halifax
country: Canada
facility: Sage Investigational Site
state: Nova Scotia
city: North York
country: Canada
facility: Sage Investigational Site
state: Ontario
city: Montréal
country: Canada
facility: Sage Investigational Site
state: Quebec
city: Tooting
country: United Kingdom
facility: Sage Investigational Site
state: London
city: Aberdeen
country: United Kingdom
facility: Sage Investigational Site
city: Birmingham
country: United Kingdom
facility: Sage Investigational Site
city: Cardiff
country: United Kingdom
facility: Sage Investigational Site
city: Leeds
country: United Kingdom
facility: Sage Investigational Site
city: Leeds
country: United Kingdom
facility: Sage Investigational Site
city: Newcastle Upon Tyne
country: United Kingdom
facility: Sage Investigational Site
city: Plymouth
country: United Kingdom
facility: Sage Investigational Site
city: Southampton
country: United Kingdom
facility: Sage Investigational Site
Study officials:
References: