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Taking peoplePhase 2

Mivelsiran Tested in Early Down Syndrome-Associated Alzheimer's Disease

Tests mivelsiran against placebo in adults with early Down syndrome-associated Alzheimer's disease.

Key facts

Who runs it
Alnylam Pharmaceuticals
Where
Rome, ItalySee the 2 sitesAlso in 5 other countries, 14 sites
Registry title
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
Status
Recruiting
Phase
Phase 2
People taking part
58 people, planned
Started
17 Aug 2026
Expected to end
30 Jul 2031
Type of study
Interventional, 3 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • Mivelsiran
  • Placebo
Record updated
13 October 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for adults aged 40 to 55 with Down syndrome and early-stage Alzheimer's disease. It tests a drug called mivelsiran against placebo.

Age
40 Years to 55 Years
Sex
All sexes can join.
Disease stage
Early-stage Alzheimer's disease linked to Down syndrome (trisomy 21). Thinking skills must be stable in the investigator's opinion.
Earlier treatment
People who recently received an investigational agent or had treatment with an amyloid-targeting antibody cannot join.
Biomarker or test result
A positive amyloid PET scan.

Main reasons for not taking part

  • Severe intellectual disability
  • History of Down syndrome regression disorder
  • ALT or AST above 2 times the upper limit of normal at screening
  • eGFR below 45 mL/min/1.73m^2 at screening
  • Seizures in the 6 months before screening, or poorly controlled sleep apnea or hypothyroidism

This trial only enrols participants with Down syndrome. The registry sentence this rests on: Clinical diagnosis of Down-Syndrome (DS) associated with trisomy 21

The registry criteria, word for word

Show all 11 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • Has recently received an investigational agent
  • Has had treatment with amyloid-targeting antibody
  • 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 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

Check if you might fit

Answer for yourself. This is not a decision: only the study team can say whether you can take part.

  • Are you between 40 and 55 years old?

Where in Italy

Also in 5 other countries, 14 sites

  • Fondazione Policlinico Universitario Campus Bio-medico In Forma A Bbreviata FonRome, ItalyNot yet recruiting
  • Ospedale Isola Tiberina - Gemelli IsolaRome, ItalyNot yet recruiting

About the trial

What the trial wants to find out

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.

The 3 groups in the trial

  • Mivelsiran (Treatment Group A)Treatment being tested

    Participants will be administered multiple low doses of mivelsiran.

    Treatment: Mivelsiran

  • Mivelsiran (Treatment Group B)Treatment being tested

    Participants will be administered multiple high doses of mivelsiran.

    Treatment: Mivelsiran

  • Placebo (Treatment Group C)Placebo, for comparison

    Participants will be administered multiple doses of placebo.

    Treatment: Placebo

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
Official title
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)
Plain-language title
Mivelsiran Tested in Early Down Syndrome-Associated Alzheimer's DiseaseGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 13 November 2026.
Fit questions
Built from NCT07636811, who can take part
Record updated
13 October 2026