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RecruitingPhase 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)

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
40 Years to 55 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 39 months

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 39 months."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests mivelsiran in adults with early-stage Alzheimer's disease linked to Down syndrome. It looks at the drug's effects in the body, its safety, and how well people handle treatment. Taking part may last up to 39 months.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Have a diagnosis of Down syndrome caused by an extra copy of chromosome 21.
  • Have a PET brain scan that shows amyloid, a protein linked to Alzheimer's disease.
  • Have stable thinking and memory skills, as judged by the study doctor.
  • Have well-controlled seizures, with no seizures in the 6 months before screening.

Who can't join

  • Have a severe intellectual disability, meaning major limits in learning and daily living skills.
  • Have a history of Down syndrome regression disorder, which causes a loss of skills.
  • Have ALT or AST liver blood test levels more than twice the upper normal limit at screening.
  • Have an eGFR kidney test result below 45 at screening, showing reduced kidney function.
  • Have recently received an experimental drug.
  • Have received antibody treatment that targets amyloid.
  • Have other health problems that are not well controlled, such as breathing pauses during sleep or an underactive thyroid.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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

Study sites (4)

FL

IN

MO