A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Al… (NCT07636811) | Clinical Trial Compass
Not Yet RecruitingPhase 2
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
United States58 participantsStarted 2026-07-01
Plain-language summary
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.
Who can participate
Age range
40 Years – 55 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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:
* Has severe intellectual disability (ID)
* Has a history of DS regression disorder
* 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
* Has recently received an investigational agent
* Has had treatment with amyloid-targeting antibody
* Comorbidities such as obstructive sleep apnea and hypothyroidism that are not well controlled
Note: other protocol defined inclusion / exclusion criteria apply
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Mivelsiran is being studied in a Phase 2 trial, which means researchers are still working out safety and dosing details — given that, what is currently known about the risks of this drug, and how does that weigh against my options right now?
2This trial focuses on people with early-stage Down Syndrome-associated Alzheimer's disease and measures changes in brain amyloid levels — based on where I am in my diagnosis, do you think I would even be a candidate to discuss this study when it opens to enrollment?
3The trial isn't recruiting yet, so there could be a wait before it starts — given the early stage of my condition, does the timing of this trial fit with where my disease is likely to be by the time it opens?
4The main thing this trial is measuring is amyloid burden in the brain using a centiloid scale — can you help me understand what that means for real-world outcomes, and whether reducing amyloid has been shown to slow symptoms in people with Down Syndrome-associated Alzheimer's?
5Are there any currently available treatments or other studies I should consider now, rather than waiting for this Phase 2 trial to begin recruiting?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Double-Blind Period: Change from baseline in brain amyloid burden measured in centiloids (CLs)