Mivelsiran is named as an intervention in 2 current clinical trials in our USA, UK, Netherlands, Switzerland, Canada, Australia and Spain registers. 1 is recruiting patients now. All registered phases are Phase 2. The lead sponsor is Alnylam Pharmaceuticals. The most-studied condition is Cerebral Amyloid Angiopathy. The EU clinical trials register (CTIS) separately lists 1 authorised or ongoing trial, 1 with a site in Ireland.
| Study | Phase | Status | Lead sponsor | Registers |
|---|---|---|---|---|
|
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
NCT07636811 · ClinicalTrials.gov ↗ · Down Syndrome-Associated Alzheimer's Disease (DS-AD)
|
Phase 2 | Recruiting | Alnylam Pharmaceuticals | USA, Spain |
|
A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy
NCT06393712 · ClinicalTrials.gov ↗ · Cerebral Amyloid Angiopathy
|
Phase 2 | Active, closed | Alnylam Pharmaceuticals | UK, USA, Netherlands, Switzerland, Canada, Australia |
Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Authorised or ongoing trials in the EU Clinical Trials Information System that list a product containing Mivelsiran. Many trials are registered in both CTIS and ClinicalTrials.gov, so these are shown separately rather than added to the count above. Trials with a site in Ireland are listed first.
| Study | Phase | Status | Sponsor | Countries |
|---|---|---|---|---|
|
A Phase 2, Multicenter, Randomized, Double-blind, Placebocontrolled Study, to Evaluate Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered Mivelsiran in Adult Participants with Early-Stage Down Syndrome-Associated Alzheimer’s Disease (DS-AD)
2025-523390-42-00 · Nervous System Diseases
|
Phase 2 | Authorised | Alnylam Pharmaceuticals Inc | Ireland + 4 more |
| Lead sponsor | Trials |
|---|---|
| Alnylam Pharmaceuticals | 2 |
A trial with sites in more than one country appears in each of those registers. CTIS does not publish a recruiting flag; its trials may also appear in the registers above.
2 current trials in our USA, UK, Netherlands, Switzerland, Canada, Australia and Spain registers name Mivelsiran as a drug or biological intervention, as of the registry sync on 28 September 2026. Completed and withdrawn studies are not counted.
Yes. 1 of the 2 trials is recruiting: 1 with sites in USA and 1 with sites in Spain. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
The lead sponsor is Alnylam Pharmaceuticals. The number in brackets is how many trials each organisation leads.
Yes. 1 trial in the EU register (CTIS) list a site in Ireland. Some may be the same study registered in both. See the Irish clinical trials register for sites and contacts.
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Mivelsiran works or whether it is effective, and it is not medical advice.
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Mivelsiran works or whether it is effective. Each trial links to its own record so you can check it yourself.
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Mivelsiran (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 28 Sep 2026.