Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05655520




Registration number
NCT05655520
Ethics application status
Date submitted
9/12/2022
Date registered
19/12/2022
Date last updated
2/10/2023

Titles & IDs
Public title
A Study to Evaluate the Safety and Tolerability of SAGE-718 in Participants With Huntington's Disease
Scientific title
A Phase 3, Multicenter, Open-label Safety Study to Evaluate the Long-term Safety and Tolerability of SAGE-718 in Participants With Huntington's Disease
Secondary ID [1] 0 0
718-CIH-301
Universal Trial Number (UTN)
Trial acronym
Linked study record

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

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

Experimental: Cohort 1 (Direct Rollover) - Participants from studies 718-CIH-201 (NCT05107128)/202 (NCT05358821) who will sign the informed consent for study 718-CIH-301 =7 days after the last day of the corresponding parent study. Participants will receive Sage-718 from Day 1 up to Day 365.

Experimental: Cohort 2 (Gap Rollover) - Participants from studies 718-CIH-201 (NCT05107128)/202 (NCT05358821) who will sign the informed consent for study 718-CIH-301 after a gap of >7 days after the last day of the corresponding parent study. Participants will receive Sage-718 from Day 1 up to Day 365.

Experimental: Cohort 3 (De Novo) - Participants who were not previously included in any SAGE-718 clinical study. Participants will receive Sage-718 from Day 1 up to Day 365.


Treatment: Drugs: SAGE-718
Oral softgel lipid capsules
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) and Severity of TEAEs
Timepoint [1] 0 0
Up to 13 months
Primary outcome [2] 0 0
Number of Participants Who Withdrew Due to Adverse Events (AEs)
Timepoint [2] 0 0
Up to 13 months
Primary outcome [3] 0 0
Percentage of Participants with Change From Baseline in Vital Signs, Clinical Laboratory Parameters and Electrocardiograms (ECGs) Parameters
Timepoint [3] 0 0
Up to 13 months
Primary outcome [4] 0 0
Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) Responses
Timepoint [4] 0 0
Up to 13 months

Eligibility
Key inclusion criteria
For all participants:

- Completed 718-CIH-201 (NCT05107128) or 718-CIH-202 (NCT05358821) studies or meet
eligibility criteria for the de novo cohort.

- Agree to refrain from drugs of abuse for the duration of the study and from alcohol
during the 48 hours preceding each study visit.

- 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 able to travel to the study center, and, 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.

Additional inclusion criteria for the de novo cohort (Cohort 3):

- Be at least 25 years old, but not older than 65 years of age at Screening.

- Genetically confirmed disease with cytosine-adenine-guanine (CAG) expansion =40

- No features of juvenile HD

- CAG-Age-Product (CAP) score =90, as calculated using the CAP formula: AGE × (CAG - 30)
/ 6.49.

- Either Unified Huntington's Disease Rating Scale (UHDRS) -Total Functional Capacity
(TFC)=13 or Montreal Cognitive Assessment (MoCA) >25 score at Screening (one or the
other; not both)
Minimum age
25 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
For all participants

- 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.

- Had gastric bypass surgery, has a gastric sleeve or lap band, or has had any related
procedures that interfere with gastrointestinal transit.

- Is known to be allergic to any of SAGE-718 excipients, including soy lecithin.

- Receive any prohibited medications within 30 days of screening and during
participation in the study.

Additional exclusion criteria for the de novo cohort (Cohort 3):

- Have previous exposure to gene therapy, or have participated in any other 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).
Additionally, participants who have received treatment with antisense oligonucleotides
or a messenger ribonucleic acid (mRNA) splicing modifier will be excluded.

Note: Participants with confirmation of enrolment in the placebo arm of these
investigational trials would not be excluded.

Additional exclusion criteria for 718-CIH-201/202 completers (Cohorts 1 and 2):

- Have ongoing serious adverse events from the parent study.

- Have ongoing, unresolved AE(s), which in the opinion of the investigator or sponsor,
is likely to interfere with study conduct or compliance.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/12/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2025
Actual
Sample size
Target
300
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Iowa
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
South Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Tennessee
Country [12] 0 0
United States of America
State/province [12] 0 0
Virginia
Country [13] 0 0
United States of America
State/province [13] 0 0
Washington
Country [14] 0 0
Canada
State/province [14] 0 0
Ontario

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 the study is to evaluate the safety and tolerability of SAGE-718
softgel lipid capsule in participants with Huntington's Disease (HD)
Trial website
https://clinicaltrials.gov/ct2/show/NCT05655520
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

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
grant.rutledge@sagerx.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05655520