A study to evaluate the effect of SAGE-718 on cognitive function in participants with Huntington's Disease
- Conditions
- Huntington's diseaseNervous System Diseases
- Registration Number
- ISRCTN17896603
- Lead Sponsor
- Sage Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Ongoing
- Sex
- All
- Target Recruitment
- 189
Current inclusion criteria as of 20/05/2024:
1. Meet all the following criteria for HD at Screening (Days -28 to -2):
1.1. Genetically confirmed disease with huntingtin gene CAG expansion =36.
1.2. At Screening, UHDRS-Total Functional Capacity (TFC) score >6 and <13
suggesting no more than a moderate level of functional impairment.
1.3. 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.
_____
Previous inclusion criteria as of 25/10/2022:
1. Meet all the following criteria for HD:
1.1. Genetically confirmed disease with huntingtin gene CAG expansion =36.
1.2. UHDRS-Total Functional Capacity (TFC) score >6 and <13.
1.3. No features of juvenile HD.
2. Score <26 on the Montreal Cognitive Assessment (MoCA) at screening.
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.
_____
Previous inclusion criteria:
1. Meet all the following criteria for HD:
1.1. Genetically confirmed disease with huntingtin gene CAG expansion =36.
1.2. UHDRS-Total Functional Capacity (TFC) score >6 and <13.
1.3. No features of juvenile HD.
2. Score <26 on the Montreal Cognitive Assessment (MoCA) at screening.
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; individuals requiring a wheelchair 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.
Current exclusion criteria as of 20/05/2024:
1. Have participated in a previous clinical study of SAGE-718, have previous exposure to gene therapy or 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
_____
Previous exclusion criteria as of 25/10/2022:
1. 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 participant participated solely in the placebo arm of the study. Additionally, participants who have received treatment with antisense oligonucleotides (ASOs) or a messenger ribonucleic acid (mRNA) splicing modifier will 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
_____
Previous exclusion criteria:
1. 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 180 days or 5 half-lives (whichever is longer), unless the patient participated solely in the placebo arm of the study.
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change from baseline to day 84 in the cognitive function assessed using Huntington's Disease Cognitive Assessment Battery (HD-CAB) Composite Score
- Secondary Outcome Measures
Name Time Method 1. Change from baseline to day 84 in the functional capacity assessed using Unified Huntington's Disease Rating Scale (UHDRS) - Independence Scale <br>2. Change from baseline to day 84 in the motor function assessed using UHDRS - Total Motor Score (TMS) <br>3. Percentage of Participants with Treatment-emergent Adverse Events (TEAEs) up to approximately 112 days