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Also known as ALN-APP
Mivelsiran · 3 trials · 3 indications
| 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. |
| ALN-APP | EXPERIMENTAL | Participants will be administered multiple doses of ALN-APP during the double-blind treatment period and optional open-label extension period. |
| Placebo/ALN-APP | PLACEBO_COMPARATOR | Participants will be administered multiple doses of placebo during the double-blind treatment period. Participants who continue into the optional open-label extension period will be administered multiple doses of ALN-APP. |
| Part A: ALN-APP | EXPERIMENTAL | Participants will be administered a single dose of ALN-APP. |
| Part A: Placebo | PLACEBO_COMPARATOR | Participants will be administered a single dose of placebo. |
| Part B: | EXPERIMENTAL | Participants will be administered multiple doses of ALN-APP. |
| Name | Type | Description |
|---|---|---|
| Mivelsiran | DRUG | Mivelsiran will be administered intrathecally |
| Placebo | DRUG | Placebo will be administered intrathecally |
| ALN-APP | DRUG | ALN-APP 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 RNA therapy being developed by Alnylam Pharmaceuticals, Inc. (ticker ALNY) for Down Syndrome-Associated Alzheimer's Disease, or DS-AD. It is in Phase 2 clinical development and has not been approved by the FDA. It is a small interfering RNA (siRNA) therapy, part of Alnylam's -siran naming class.
Mivelsiran is being studied for the treatment of early-stage Down Syndrome-Associated Alzheimer's Disease (DS-AD) in adults. The Phase 2 trial enrolls participants aged 40 years and older with this condition. It is investigational and has not been approved for any indication.
Mivelsiran is a small interfering RNA (siRNA) therapy, a class of RNA therapeutics that uses RNA interference to reduce the production of a specific disease-related protein. Alnylam Pharmaceuticals develops this -siran class of siRNA medicines. The specific molecular target of mivelsiran in DS-AD has not been publicly disclosed.
Mivelsiran is being developed by Alnylam Pharmaceuticals, Inc., which trades under the ticker ALNY. Alnylam is the sponsor of the Phase 2 clinical trial evaluating mivelsiran in adults with early-stage Down Syndrome-Associated Alzheimer's Disease.
Mivelsiran is in Phase 2 clinical development. It is investigational and has not been approved by the FDA for any indication. The ongoing Phase 2 study is currently recruiting participants.
Mivelsiran is being evaluated in one active clinical trial: NCT07636811, titled "A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)." This Phase 2 trial is randomized, double-blind, placebo-controlled, and enrolling 58 participants across the United States and Spain.