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| ID | Type | Description | Link |
|---|---|---|---|
| 2025-523390-42-00 | EU Trial (CTIS) Number |
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The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran. The study will be conducted over 2 periods: a 24-month double-blind period and an optional 12-month open-label treatment extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 39 months.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Mivelsiran (Treatment Group A) | Experimental | Participants will be administered multiple low doses of mivelsiran. |
|
| Mivelsiran (Treatment Group B) | Experimental | Participants will be administered multiple high doses of mivelsiran. |
|
| Placebo (Treatment Group C) | Placebo Comparator | Participants will be administered multiple doses of placebo. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Mivelsiran | Drug | Mivelsiran will be administered intrathecally |
|
| Measure | Description | Time Frame |
|---|---|---|
| Double-Blind Period: Change from baseline in brain amyloid burden measured in centiloids (CLs) | Up to 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Double-Blind Period: Change from baseline in APP protein concentration in cerebrospinal fluid (CSF) | Up to 24 months | |
| Double-Blind Period: Change from baseline in the concentration of amyloid beta proteins in CSF | Up to 24 months |
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Inclusion Criteria:
Exclusion Criteria:
Note: other protocol defined inclusion / exclusion criteria apply
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Alnylam Clinical Trial Information Line | Contact | 1-877-ALNYLAM | clinicaltrials@alnylam.com | |
| Alnylam Clinical Trial Information Line | Contact | 1-877-256-9526 | clinicaltrials@alnylam.com |
| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Alnylam Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Study Site | Maitland | Florida | 32751 | United States | ||
| Clinical Study Site |
Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU.
Access to data may be declined where there is likelihood a patient could be identified or other feasibility issue, where there is a potential conflict of interest, a planned business activities or an actual or potential competitive risk. Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Timeframes for data access may vary and can take up to 6 months or more.
Requests for access to data can be submitted via the website www.vivli.org. Questions can also be directed to datasharing@alnylam.com.
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| Placebo | Drug | Placebo will be administered intrathecally |
|
| Double-Blind Period: Change from baseline in the concentration of tau proteins in plasma | Up to 24 months |
| Double-Blind Period: Change from baseline on a cognitive scale | Up to 24 months |
| Double-blind Period and Open-label Extension (OLE) Period: Frequency of adverse events (AEs) | Up to 36 months |
| Naples |
| Florida |
| 34105 |
| United States |
| ID | Term |
|---|---|
| D004314 | Down Syndrome |
| ID | Term |
|---|---|
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
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