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Mivelsiran · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| 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 |
|---|---|---|
| Mivelsiran | DRUG | Mivelsiran will be administered intrathecally |
| Placebo | DRUG | Placebo will be administered intrathecally |
Inclusion Criteria: * Clinical diagnosis of Down-Syndrome (DS) associated with trisomy 21 * Positive amyloid PET scan * Cognitively stable in the opinion of the investigator * Seizures must be well controlled with no occurrence of seizures in the 6 months prior to screening Exclusion Criteria: * ...
Mivelsiran is an investigational small molecule being studied for the treatment of Down Syndrome-Associated Alzheimer's Disease (DS-AD) in adults. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
Mivelsiran is a small interfering RNA (siRNA) therapeutic, as indicated by its '-siran' suffix. It is designed to target and silence specific genes involved in the pathology of Down Syndrome-Associated Alzheimer's Disease, though the exact molecular target has not been disclosed.
Mivelsiran is being developed by Alnylam Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol ALNY. Alnylam is a leader in RNA interference therapeutics and is conducting the clinical development program for this asset.
Mivelsiran is currently in Phase 2 clinical development. It is being evaluated in a randomized, double-blind, placebo-controlled study in adult participants with early-stage Down Syndrome-Associated Alzheimer's Disease. The drug is investigational and has not received FDA approval.
Mivelsiran is being studied in a Phase 2 clinical trial with the identifier NCT07636811. This trial is actively recruiting participants in the United States and aims to enroll 58 adults aged 40 years and older with early-stage Down Syndrome-Associated Alzheimer's Disease.
Mivelsiran is a distinct investigational drug developed by Alnylam Pharmaceuticals. No alternative names have been reported for this asset, and it is not known to be identical to any other approved or investigational medication.