Clinical trial NCT05655520: A Study to Evaluate the Safety and Tolerability of SAGE-718 in Participants With Huntington's Disease
Official 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Huntington's Disease
Keywords: Huntington's Disease
SAGE-718
Lead sponsor: Sage Therapeutics
Sponsor class: INDUSTRY
First posted: 2022-12-19
Enrollment: 153
Sex: ALL
Minimum age (years): 25
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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)


Eligibility criteria: 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.
* At screening, scores of either: a) Unified Huntington's Disease Rating Scale (UHDRS) -Total Functional Capacity (TFC)=13 and Montreal Cognitive Assessment (MoCA) ≤25 score, or b) UHDRS-TFC ≤12 and MoCA \>25

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 one or more ongoing serious adverse events (SAEs) 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.

Primary outcomes:
measure: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) and Severity of TEAEs
timeFrame: Up to 49 months
measure: Number of Participants Who Withdrew Due to Adverse Events (AEs)
timeFrame: Up to 49 months
measure: Percentage of Participants with Change From Baseline in Vital Signs, Clinical Laboratory Parameters and Electrocardiograms (ECGs) Parameters
timeFrame: Up to 49 months
measure: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) Responses
timeFrame: Up to 49 months
Secondary outcomes:
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: Sage Investigational Site
state: California
city: Los Alamitos
country: United States
facility: Sage Investigational Site
state: California
city: Los Angeles
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: Chicago
country: United States
facility: Sage Investigational Site
state: Illinois
city: Iowa City
country: United States
facility: Sage Investigational Site
state: Iowa
city: Williamsville
country: United States
facility: Sage Investigational Site
state: New York
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: Richmond
country: United States
facility: Sage Investigational Site
state: Virginia
city: Spokane
country: United States
facility: Sage Investigational Site
state: Washington
city: Toronto
country: Canada
facility: Sage Investigational Site
state: Ontario
Study officials:
References: