Trial Review

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05107128




Registration number
NCT05107128
Ethics application status
Date submitted
25/10/2021
Date registered
4/11/2021
Date last updated
12/11/2024

Titles & IDs
Public title
A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)
Scientific title
A Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease
Secondary ID [1] 0 0
2021-005577-16
Secondary ID [2] 0 0
718-CIH-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Huntington's Disease 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - SAGE-718
Treatment: Drugs - Placebo

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

Placebo comparator: Placebo - Participants will receive placebo, once daily for 84 days.


Treatment: Drugs: SAGE-718
Oral capsules.

Treatment: Drugs: Placebo
SAGE-718-matching oral capsules.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in the Symbol Digit Modalities Test (SDMT)
Timepoint [1] 0 0
Baseline and Day 84
Secondary outcome [1] 0 0
Change From Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) - Independence Scale
Timepoint [1] 0 0
Baseline and Day 84
Secondary outcome [2] 0 0
Change From Baseline in the Trial Making Test Part B
Timepoint [2] 0 0
Baseline and Day 84
Secondary outcome [3] 0 0
Change From Baseline in the One Touch Stockings of Cambridge (OTS-Mean Latency until Correct response)
Timepoint [3] 0 0
Baseline and Day 84
Secondary outcome [4] 0 0
Change From Baseline in the Paced Tapping Test (PTAP)
Timepoint [4] 0 0
Baseline and Day 84
Secondary outcome [5] 0 0
Change From Baseline in the Huntington's Disease Everyday Functioning (Hi-DEF) Home Subdomain Score
Timepoint [5] 0 0
Baseline and Day 84
Secondary outcome [6] 0 0
Change From Baseline in the Clinical Global Impression - Severity (CGI-S) Cognitive Status Subdomain Score
Timepoint [6] 0 0
Baseline and Day 84
Secondary outcome [7] 0 0
Proportion of Participants With Treatment-emergent Adverse Events (TEAEs)
Timepoint [7] 0 0
Up to approximately 112 days

Eligibility
Key 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 >6 and <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).
Minimum age
25 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s

The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
10/02/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
3/10/2024
Sample size
Target
Accrual to date
Final
189
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Sage Investigational Site - Westmead
Recruitment hospital [2] 0 0
Sage Investigational Site - Herston
Recruitment hospital [3] 0 0
Sage Investigational Site - Parkdale
Recruitment hospital [4] 0 0
Sage Investigational Site - Nedlands
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4029 - Herston
Recruitment postcode(s) [3] 0 0
3195 - Parkdale
Recruitment postcode(s) [4] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
District of Columbia
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Hawaii
Country [7] 0 0
United States of America
State/province [7] 0 0
Illinois
Country [8] 0 0
United States of America
State/province [8] 0 0
Indiana
Country [9] 0 0
United States of America
State/province [9] 0 0
Iowa
Country [10] 0 0
United States of America
State/province [10] 0 0
Kansas
Country [11] 0 0
United States of America
State/province [11] 0 0
Maryland
Country [12] 0 0
United States of America
State/province [12] 0 0
Massachusetts
Country [13] 0 0
United States of America
State/province [13] 0 0
New York
Country [14] 0 0
United States of America
State/province [14] 0 0
North Carolina
Country [15] 0 0
United States of America
State/province [15] 0 0
Ohio
Country [16] 0 0
United States of America
State/province [16] 0 0
Pennsylvania
Country [17] 0 0
United States of America
State/province [17] 0 0
South Carolina
Country [18] 0 0
United States of America
State/province [18] 0 0
Tennessee
Country [19] 0 0
United States of America
State/province [19] 0 0
Texas
Country [20] 0 0
United States of America
State/province [20] 0 0
Virginia
Country [21] 0 0
United States of America
State/province [21] 0 0
Washington
Country [22] 0 0
Canada
State/province [22] 0 0
Nova Scotia
Country [23] 0 0
Canada
State/province [23] 0 0
Ontario
Country [24] 0 0
Canada
State/province [24] 0 0
Quebec
Country [25] 0 0
United Kingdom
State/province [25] 0 0
London
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Aberdeen
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Birmingham
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Cardiff
Country [29] 0 0
United Kingdom
State/province [29] 0 0
Leeds
Country [30] 0 0
United Kingdom
State/province [30] 0 0
Newcastle Upon Tyne
Country [31] 0 0
United Kingdom
State/province [31] 0 0
Plymouth
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Sage Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT05107128
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Grant Rutledge, PhD
Address 0 0
Country 0 0
Phone 0 0
339-368-8432
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05107128