NCT07636811RecruitingPhase 2
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled 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)
Source: ClinicalTrials.gov · Last updated 13 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 58 (estimated)
- First submitted
- 4 Jun 2026
- Start date
- 17 Aug 2026
- Primary completion date
- 18 May 2030
- Lead sponsor
- Alnylam Pharmaceuticals
- Diseases
- Alzheimer disease
- NCT number
- NCT07636811
- EU CT number
- 2025-523390-42-00
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 58
- From 17 Aug 2026 to the primary completion date, 18 May 2030
- 16 sites in 6 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Has recently received an investigational agent
- Has had treatment with amyloid-targeting antibody
Other illness
- Has a history of DS regression disorder
Lab values
- Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2×upper limit of normal (ULN) at Screening
- Has estimated glomerular filtration rate (eGFR) \<45 mL/min/1.73m\^2 at Screening
Other
- Has severe intellectual disability (ID)
- Comorbidities such as obstructive sleep apnea and hypothyroidism that are not well controlled
- Note: other protocol defined inclusion / exclusion criteria apply
Who the study is looking for
- 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
Age in the registry: 40 years to 55 years · Sex: All · Does not accept healthy volunteers
Sites (16)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Gesundheit Nord gGmbH Klinikverbund Bremen Coordinator of this site? Confirm its status | Bremen | Germany | Not yet recruiting |
| LMU Klinikum Muenchen AöR Coordinator of this site? Confirm its status | Munich | Germany | Not yet recruiting |
| Hospital de la Santa Creu i Sant Pau Coordinator of this site? Confirm its status | Barcelona | Spain | Recruiting |
| Gestion Hospitalaria Del Sur S.A. Coordinator of this site? Confirm its status | Dos Hermanas | Spain | Recruiting |
| Hospital Clínico San Carlos Coordinator of this site? Confirm its status | Madrid | Spain | Recruiting |
| Hospital Universitario Regional de Málaga Coordinator of this site? Confirm its status | Málaga | Spain | Recruiting |
| Clínica Universidad de navarra Coordinator of this site? Confirm its status | Pamplona | Spain | Recruiting |
| Hospital Universitario Dr. Peset Aleixandre Coordinator of this site? Confirm its status | Valencia | Spain | Recruiting |
| Tallaght University Hospital Coordinator of this site? Confirm its status | Dublin 24 | Ireland | Not yet recruiting |
| Ospedale Isola Tiberina - Gemelli Isola Coordinator of this site? Confirm its status | Rome | Italy | Not yet recruiting |
| Fondazione Policlinico Universitario Campus Bio-medico In Forma A Bbreviata Fon Coordinator of this site? Confirm its status | Rome | Italy | Not yet recruiting |
| Amsterdam UMC Stichting Coordinator of this site? Confirm its status | Amsterdam | Netherlands | Not yet recruiting |
| - Coordinator of this site? Confirm its status | Indianapolis | United States | Recruiting |
| - Coordinator of this site? Confirm its status | Maitland | United States | Recruiting |
| - Coordinator of this site? Confirm its status | Naples | United States | Recruiting |
| - Coordinator of this site? Confirm its status | St Louis | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.