Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The primary purpose of this study is to assess the magnitude of the baseline difference between participants with early Huntington's Disease (HD) and healthy participants (HP) with respect to measures of cognitive performance.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SAGE-718 | Experimental | Participants with HD will receive SAGE-718 1.2 milligrams (mg), orally, once daily for up to 28 days. |
|
| Placebo | Placebo Comparator | Participants with HD will receive SAGE-718-matching placebo, orally, once daily for up to 28 days. |
|
| Healthy Participants | No Intervention | HP enrolled in this study will not receive any investigational product (IP) (SAGE-718 or placebo). |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SAGE-718 | Drug | SAGE-718 oral softgel lipid capsules |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Difference in Huntington's Disease Cognitive Assessment Battery (HD-CAB) Composite Score Between Participants With HD vs HP at Baseline | HD-CAB assesses cognitive function using 6 subtests:Symbol Digit Modalities Test-correctly coded items(0-110); One Touch Stockings of Cambridge(OTS)-mean time to reach correct response(range not defined); Trail Making Test Trail B (TMT-B)-time to complete task(0-240 sec); Hopkins Verbal Learning Test Revised-total correct recall trials(0-48); Paced Tapping Test-reciprocal of standard deviation(SD) of intertap intervals (range not defined); Emotion Recognition Test-negative emotions correctly identified(0-24). Values of OTS & TMT B are multiplied by -1 to represent higher is better direction as other tests. Each of 6 subtests scores was transformed to z-score(range not defined;low negative value represents cognitive impairment), which is calculated by subtracting mean and dividing by SD of HP at baseline. HD-CAB composite=average of 6 z-scores. Negative HD-CAB of HD participants=decline in cognitive function relative to HP. Assessment is relative to reference group(healthy population). | Baseline |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With HD With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | An adverse event (AE) is any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Treatment-emergent adverse events are defined as any adverse events with onset on or after the first dose of IP. A serious TEAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death (a life-threatening event), requires in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability or incapacity, results in a congenital abnormality or birth defect. |
Not provided
Inclusion Criteria:
For all
Agree to refrain from drugs of abuse for the duration of the study and from alcohol during the 48 hours preceding each study visit.
Additional criteria for participants with HD only:
Be ambulatory, 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.
Have:
CAG-Age-Product (CAP) score >70, as calculated using the CAP formula: Age × (CAG - 30) / 6.49.
Score of 15 to 25 (inclusive) on the Montreal Cognitive Assessment (MoCA) at screening, indicating the presence of cognitive impairment.
Additional criteria for HP only:
Exclusion Criteria:
For All
Additional criteria for participants with HD only:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Sage Investigational Site | Birmingham | Alabama | 35233 | United States | ||
| Sage Investigational Site |
Data sharing will be consistent with the results submission policy of ClinicalTrials.gov.
Not provided
Not provided
Not provided
Not provided
A total of 69 adult participants, comprising 29 healthy participants (HP) and 40 participants with Huntington's disease (HD) were enrolled in the study. Participants with HD received either SAGE-718 or placebo. As pre-specified in the protocol, HP enrolled in the study followed the same assessment schedule as participants with HD, but did not receive any investigational product (IP) (SAGE-718 or placebo).
Participants were enrolled at 14 investigative sites in the United States and Canada from 26 May 2022 to 10 April 2024.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants with HD received SAGE-718-matching placebo, orally, once daily for up to 28 days. |
| FG001 | SAGE-718 | Participants with HD received SAGE-718 1.2 milligrams (mg), orally, once daily for up to 28 days. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 6, 2023 | Feb 28, 2025 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Drug |
SAGE-718-matching oral capsules |
|
| Up to Day 42 |
| Number of Participants With HD With Clinically Significant Change From Baseline in Vital Sign Measurements | Vital signs including oral temperature, respiratory rate, heart rate (supine and standing), and blood pressures (supine and standing). Number of participants with clinically significant change in vital signs measurements which were deemed clinically significant by the investigator were reported. | Up to Day 42 |
| Number of Participants With HD With Clinically Significant Change From Baseline in Clinical Laboratory Assessments | Clinical laboratory assessments including hematology, serum chemistry, coagulation, and urinalysis were performed. Number of participants with clinically significant change in laboratory assessments which were deemed clinically significant by the investigator were reported. | Up to Day 42 |
| Los Alamitos |
| California |
| 90720 |
| United States |
| Sage Investigational Site | Englewood | Colorado | 80113 | United States |
| Sage Investigational Site | Washington D.C. | District of Columbia | 20057 | United States |
| Sage Investigational Site | Boca Raton | Florida | 33431 | United States |
| Sage Investigational Site | Tampa | Florida | 33602 | United States |
| Sage Investigational Site | Chicago | Illinois | 60612 | United States |
| Sage Investigational Site | Baltimore | Maryland | 21218 | United States |
| Sage Investigational Site | New York | New York | 10027 | United States |
| Sage Investigational Site | Chapel Hill | North Carolina | 27599 | United States |
| Sage Investigational Site | Cincinnati | Ohio | 45221 | United States |
| Sage Investigational Site | Philadelphia | Pennsylvania | 19104 | United States |
| Sage Investigational Site | Houston | Texas | 77030 | United States |
| Sage Investigational Site | Montreal | H2X 3E4 | Canada |
| FG002 | Healthy Participants | HP enrolled in this study did not receive any IP (SAGE-718 or placebo). |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Full Analysis Set (FAS) included all randomized participants with HD and all HP who met the eligibility criteria and had at least one baseline assessment of Huntington's disease cognitive assessment Battery (HD-CAB) (participants with HD and HP).
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants with HD received SAGE-718-matching placebo, orally, once daily for up to 28 days. |
| BG001 | SAGE-718 | Participants with HD received SAGE-718 1.2 mg, orally, once daily for up to 28 days. |
| BG002 | Healthy Participants | HP enrolled in this study did not receive any IP (SAGE-718 or placebo). |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Difference in Huntington's Disease Cognitive Assessment Battery (HD-CAB) Composite Score Between Participants With HD vs HP at Baseline | HD-CAB assesses cognitive function using 6 subtests:Symbol Digit Modalities Test-correctly coded items(0-110); One Touch Stockings of Cambridge(OTS)-mean time to reach correct response(range not defined); Trail Making Test Trail B (TMT-B)-time to complete task(0-240 sec); Hopkins Verbal Learning Test Revised-total correct recall trials(0-48); Paced Tapping Test-reciprocal of standard deviation(SD) of intertap intervals (range not defined); Emotion Recognition Test-negative emotions correctly identified(0-24). Values of OTS & TMT B are multiplied by -1 to represent higher is better direction as other tests. Each of 6 subtests scores was transformed to z-score(range not defined;low negative value represents cognitive impairment), which is calculated by subtracting mean and dividing by SD of HP at baseline. HD-CAB composite=average of 6 z-scores. Negative HD-CAB of HD participants=decline in cognitive function relative to HP. Assessment is relative to reference group(healthy population). | FAS included all randomized participants with HD & all HP who met eligibility criteria & had at least 1 baseline assessment of HD-CAB (participants with HD & HP). As primary analysis was to estimate baseline differences between participants with HD & HP in HD-CAB composite score, data from SAGE-718 & placebo arms were combined for analysis. Baseline for HD participants=last non-missing measurement prior to first dose of IP. Baseline for HP=last non-missing measurement on or prior to Day 1. | Posted | Mean | Standard Deviation | Z score | Baseline |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With HD With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | An adverse event (AE) is any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Treatment-emergent adverse events are defined as any adverse events with onset on or after the first dose of IP. A serious TEAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death (a life-threatening event), requires in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability or incapacity, results in a congenital abnormality or birth defect. | Safety Set included all participants with HD who were administered IP or HP who met eligibility criteria and was used to describe the safety data. | Posted | Count of Participants | Participants | Up to Day 42 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With HD With Clinically Significant Change From Baseline in Vital Sign Measurements | Vital signs including oral temperature, respiratory rate, heart rate (supine and standing), and blood pressures (supine and standing). Number of participants with clinically significant change in vital signs measurements which were deemed clinically significant by the investigator were reported. | Safety Set included all participants with HD who were administered IP or HP who met eligibility criteria and was used to describe the safety data. | Posted | Count of Participants | Participants | Up to Day 42 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With HD With Clinically Significant Change From Baseline in Clinical Laboratory Assessments | Clinical laboratory assessments including hematology, serum chemistry, coagulation, and urinalysis were performed. Number of participants with clinically significant change in laboratory assessments which were deemed clinically significant by the investigator were reported. | Safety Set included all participants with HD who were administered IP or HP who met eligibility criteria and was used to describe the safety data. | Posted | Count of Participants | Participants | Up to Day 42 |
|
|
Up to Day 42
Safety Set included all participants with HD who were administered IP or HP who met eligibility criteria and will be used to describe the safety data.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Participants with HD received SAGE-718-matching placebo, orally, once daily for up to 28 days. | 0 | 19 | 0 | 19 | 6 | 19 |
| EG001 | SAGE-718 | Participants with HD received SAGE-718 1.2 mg, orally, once daily for up to 28 days. | 0 | 21 | 0 | 21 | 7 | 21 |
| EG002 | Healthy Participants | HP enrolled in this study did not receive any IP (SAGE-718 or placebo). | 0 | 29 | 0 | 29 | 4 | 29 |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Micturition urgency | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Reflexes abnormal | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
The PI can either be a party and subject to the same restrictions as the institution, or if not a party, the restrictions are described on the face of the contract (i.e., PI is a contractor of the institution; PI is part of a larger group of study personnel; institution has contracted with or otherwise bound all study personnel under confidentiality obligations and requirements to vest intellectual property to the institution).
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Amy Bullock | Sage Therapeutics | 617-949-5151 | amy.bullock@sagerx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 22, 2024 | Feb 28, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D006816 | Huntington Disease |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D003704 | Dementia |
| D002819 | Chorea |
| D020820 | Dyskinesias |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D003072 | Cognition Disorders |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Black or African American |
|
| Native Hawaiian or Other Pacific Islander |
|
| White |
|
| Multiple |
|
| Other |
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
|
|