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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
Titles & IDs
Public title
A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)
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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
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Secondary ID [1]
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2021-005577-16
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Secondary ID [2]
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718-CIH-201
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Huntington's Disease
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Condition category
Condition code
Intervention/exposure
Study type
Interventional
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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.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline in the Huntington's Disease Cognitive Assessment Battery (HD-CAB) Composite Score
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline and Day 84
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Secondary outcome [1]
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Change From Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) - Independence Scale
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline and Day 84
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Secondary outcome [2]
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Change From Baseline in the UHDRS - Total Motor Score (TMS)
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Assessment method [2]
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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.
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Timepoint [2]
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Baseline and Day 84
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Secondary outcome [3]
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Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
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Assessment method [3]
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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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Timepoint [3]
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Up to approximately 112 days
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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.
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Minimum age
25
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Maximum age
65
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
10/02/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/12/2024
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Actual
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Sample size
Target
178
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
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Recruitment hospital [1]
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Sage Investigational Site - Westmead
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Recruitment hospital [2]
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Sage Investigational Site - Herston
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Recruitment hospital [3]
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Sage Investigational Site - Parkdale
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Recruitment hospital [4]
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Sage Investigational Site - Nedlands
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment postcode(s) [2]
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4029 - Herston
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Recruitment postcode(s) [3]
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3195 - Parkdale
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Recruitment postcode(s) [4]
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6009 - Nedlands
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Recruitment outside Australia
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United States of America
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Arkansas
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California
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Colorado
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London
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Aberdeen
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Birmingham
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Cardiff
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Newcastle Upon Tyne
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Plymouth
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United Kingdom
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Southampton
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Sage Therapeutics
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Address
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Ethics approval
Ethics application status
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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.
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Trial website
https://clinicaltrials.gov/study/NCT05107128
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Address
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Grant Rutledge, PhD
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Address
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Phone
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339-368-8432
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
Data sharing will be consistent with the results submission policy of ClinicalTrials.gov.
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT05107128