A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)
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| ClinicalTrials.gov Identifier: NCT05107128 |
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Recruitment Status :
Recruiting
First Posted : November 4, 2021
Last Update Posted : November 14, 2022
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Sponsor:
Sage Therapeutics
Information provided by (Responsible Party):
Sage Therapeutics
- Study Details
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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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Huntington's Disease | Drug: SAGE-718 Drug: Placebo | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 178 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Triple (Participant, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease |
| Actual Study Start Date : | February 10, 2022 |
| Estimated Primary Completion Date : | December 2024 |
| Estimated Study Completion Date : | December 2024 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Huntington disease
MedlinePlus related topics:
Huntington's Disease
| Arm | Intervention/treatment |
|---|---|
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Experimental: SAGE-718
Participants will receive SAGE-718, once daily for 84 days.
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Drug: SAGE-718
Oral capsules. |
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Placebo Comparator: Placebo
Participants will receive placebo, once daily for 84 days.
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Drug: Placebo
SAGE-718-matching oral capsules. |
Primary Outcome Measures :
- Change From Baseline in the Huntington's Disease Cognitive Assessment Battery (HD-CAB) Composite Score [ Time Frame: Baseline and Day 84 ]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.
Secondary Outcome Measures :
- Change From Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) - Independence Scale [ Time Frame: Baseline and Day 84 ]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.
- Change From Baseline in the UHDRS - Total Motor Score (TMS) [ Time Frame: Baseline and Day 84 ]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.
- Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Up to approximately 112 days ]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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| Ages Eligible for Study: | 25 Years to 65 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
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Meet all the following criteria for HD:
- Genetically confirmed disease with huntingtin gene CAG expansion ≥36.
- UHDRS-Total Functional Capacity (TFC) score >6 and <13.
- No features of juvenile HD.
- Score <26 on the Montreal Cognitive Assessment (MoCA) at screening.
- 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 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.
Exclusion Criteria:
- Have participated in a previous clinical study of SAGE-718, have participated in a previous gene therapy study, or have participated in any other drug, biologic, or device trial within 30 days or 5 half-lives (whichever is longer), unless the patient participated solely in the placebo arm of the study. Additionally, participants who have received treatment with antisense oligonucleotides (ASOs) or an mRNA splicing modifier will be excluded.
- 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.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05107128
Contacts
| Contact: Emily Lefler, PharmD | 619-630-4802 | emily.lefler@sagerx.com |
Locations
| United States, California | |
| Sage Investigational Site | Recruiting |
| La Jolla, California, United States, 92037 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| Sage Investigational Site | Recruiting |
| Los Angeles, California, United States, 90095 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Colorado | |
| Sage Investigational Site | Recruiting |
| Englewood, Colorado, United States, 80113 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, District of Columbia | |
| Sage Investigational Site | Recruiting |
| Washington, District of Columbia, United States, 20007 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Florida | |
| Sage Investigational Site | Recruiting |
| Boca Raton, Florida, United States, 33431 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| Sage Investigational Site | Recruiting |
| Boca Raton, Florida, United States, 33486 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| Sage Investigational Site | Recruiting |
| Tampa, Florida, United States, 33612 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Hawaii | |
| Sage Investigational Site | Recruiting |
| Honolulu, Hawaii, United States, 96817 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Illinois | |
| Sage Investigational Site | Recruiting |
| Chicago, Illinois, United States, 60611 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Indiana | |
| Sage Investigational Site | Recruiting |
| Indianapolis, Indiana, United States, 46202 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Iowa | |
| Sage Investigational Site | Recruiting |
| Iowa City, Iowa, United States, 52242 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Maryland | |
| Sage Investigational Site | Recruiting |
| Baltimore, Maryland, United States, 21287 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Massachusetts | |
| Sage Investigational Site | Recruiting |
| Boston, Massachusetts, United States, 02215 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Michigan | |
| Sage Investigational Site | Recruiting |
| Farmington Hills, Michigan, United States, 48334 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, New York | |
| Sage Investigational Site | Recruiting |
| Williamsville, New York, United States, 14221 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Ohio | |
| Sage Investigational Site | Recruiting |
| Cincinnati, Ohio, United States, 45219 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Pennsylvania | |
| Sage Investigational Site | Recruiting |
| Philadelphia, Pennsylvania, United States, 19107 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, South Carolina | |
| Sage Investigational Site | Recruiting |
| Charleston, South Carolina, United States, 29425 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Tennessee | |
| Sage Investigational Site | Recruiting |
| Memphis, Tennessee, United States, 38137 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Texas | |
| Sage Investigational Site | Recruiting |
| Houston, Texas, United States, 77030 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Virginia | |
| Sage Investigational Site | Recruiting |
| Richmond, Virginia, United States, 23219 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United States, Washington | |
| Sage Investigational Site | Recruiting |
| Kirkland, Washington, United States, 98034 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| Sage Investigational Site | Recruiting |
| Spokane, Washington, United States, 99202 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| Australia, New South Wales | |
| Sage Investigational Site | Recruiting |
| Westmead, New South Wales, Australia, 2145 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| Australia, Western Australia | |
| Sage Investigational Site | Recruiting |
| Nedlands, Western Australia, Australia, 6009 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| Canada, Ontario | |
| Sage Investigational Site | Recruiting |
| North York, Ontario, Canada, M2K 1E1 | |
| Contact clinicaltrialsinquiry@sagerx.com | |
| United Kingdom | |
| Sage Investigational Site | Recruiting |
| Plymouth, United Kingdom, PL6 8BT | |
| Contact clinicaltrialsinquiry@sagerx.com | |
Sponsors and Collaborators
Sage Therapeutics
| Responsible Party: | Sage Therapeutics |
| ClinicalTrials.gov Identifier: | NCT05107128 |
| Other Study ID Numbers: |
718-CIH-201 |
| First Posted: | November 4, 2021 Key Record Dates |
| Last Update Posted: | November 14, 2022 |
| Last Verified: | November 2022 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Plan Description: | Data sharing will be consistent with the results submission policy of ClinicalTrials.gov. |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Additional relevant MeSH terms:
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Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Dementia Chorea Dyskinesias |
Movement Disorders Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Cognition Disorders Neurocognitive Disorders Mental Disorders |