Actively Recruiting

Phase 2
Phase 3
Age: 18Years +
All Genders
Healthy Volunteers
ID05552157

A Phase II/III Multicenter Randomized, Double-Blind, Placebo-Controlled, Two-Stage Adaptive Design Platform Trial of Investigational Treatments for Primary Prevention of Disease Progression in Dominantly Inherited Alzheimer's Disease

Led by Washington University School of Medicine · Updated on 2026-02-13

280

Participants Needed

35

Research Sites

21 weeks

Total Duration

On this page

Sponsors

W

Washington University School of Medicine

Lead Sponsor

A

Alzheimer's Association

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating the effects, safety, and tolerability of investigational drugs in people who have a genetic mutation that causes early-onset Alzheimer's disease. The study focuses on whether these treatments can prevent or slow the buildup of amyloid beta plaques in the brain, an important marker of the disease. It also looks at how reducing these plaques early on might affect other Alzheimer's disease markers and slow cognitive decline. This research involves participants who are either known mutation carriers or at risk due to family history, many of whom are still without symptoms. Participants receive one of several study drugs or a matching placebo through subcutaneous injections every 12 weeks. The study has two stages: a 4-year blinded placebo-controlled period where mutation carriers are randomly assigned to active drug or placebo, followed by a 4-year open-label period in which all mutation carriers receive the active drug. Those initially on placebo will start the active drug with dose adjustments and safety monitoring similar to the first stage. Mutation-negative participants receive placebo throughout. The study design includes sharing a placebo group across different drug arms to improve efficiency. During the study, participants undergo regular clinical assessments, cognitive testing, MRI and amyloid imaging scans, and cerebrospinal fluid analysis to track disease markers and cognitive changes. Safety is monitored closely through imaging and clinical evaluations. Researchers measure the primary outcomes including changes in amyloid beta accumulation over the first 4 years and later effects on other Alzheimer's biomarkers over the next 4 years. Participation requires visits and procedures over approximately 8 years, with ongoing monitoring of cognitive and clinical status.

CONDITIONS

Brief Title

A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset AD Caused by a Genetic Mutation

Who Can Participate

Age: 18Years +
All Genders
Healthy Volunteers

Eligibility Criteria

Eligible

You may qualify if you...

  • Provide written informed consent and have a study partner who can assist with assessments.
  • Be at least 18 years old.
  • If of childbearing potential, have a negative pregnancy test at screening and agree to use effective contraception and not breastfeed during the study.
  • Carry a mutation in the APP, PSEN1, or PSEN2 gene linked to dominantly inherited Alzheimer's disease or be at risk due to family mutation.
  • Be within 11 to 25 years before the predicted age of cognitive symptom onset based on mutation or family history.
  • Have normal cognitive status (CDR-SB 0).
  • Be fluent in a language validated for the DIAN-TU cognitive and clinical measures.
  • Have adequate vision and hearing to complete assessments.
  • Be on stable doses of medications for non-excluded conditions for at least 30 days prior to baseline.
  • Agree not to donate blood or blood products during and shortly after the study.
  • Be able and willing to complete all study tests and procedures.
  • Be assessed by the investigator as likely to comply and complete the study.
Not Eligible

You will not qualify if you...

  • Have significant neurological or psychiatric disorders that affect cognition or study participation, except controlled or remote histories unlikely to interfere.
  • Be at high risk for suicide or have major depression as defined by DSM-V.
  • Have history of stroke or significant stroke risk factors interfering with cognition or study participation.
  • Have alcohol or substance use disorder currently or within the past year.
  • Have brain MRI abnormalities such as microhemorrhages, contusions, or aneurysms that are significant.
  • Have implanted devices or metal objects that prevent MRI scanning.
  • Have uncontrolled cardiovascular, hepatic, or renal conditions affecting study participation.
  • Have certain infections like untreated hepatitis C or recent hepatitis B, HIV, or CNS spirochete infections.
  • Have severe drug allergies or hypersensitivity reactions relevant to study drugs or imaging agents.
  • Have recent treatment with immunosuppressive drugs or chemotherapy.
  • Have clinically significant thyroid or vitamin B12 abnormalities.
  • Have unstable or poorly controlled diabetes.
  • Be morbidly obese with conditions preventing MRI.
  • Be on anticoagulants except low dose aspirin.
  • Have recent exposure to monoclonal antibodies targeting amyloid beta or other investigational drugs.
  • Have insufficient venous access or clinically relevant blood abnormalities.
  • Have a history of cancer with high risk of recurrence impacting participation.
  • Have medical conditions that could bias assessments or increase risk.
  • Have participated in another clinical study recently without approval.
  • Have the "Dutch" APP E693Q mutation.
  • Be unable to complete baseline cognitive and clinical assessments for eligibility.

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Up to 4 years

Participants receive subcutaneous injections of either the study drug Remternetug or a matching placebo every 12 weeks during the blinded Stage 1 period to evaluate the ability of the drug to prevent or slow disease progression.

Injections every 12 weeks with scheduled assessments throughout

Treatment

Duration - Up to 4 years

In Stage 2, all mutation carriers receive open-label Remternetug with continued monitoring to assess the effect of the treatment on downstream Alzheimer's disease biomarkers.

Injections every 12 weeks with scheduled assessments throughout

Trial Site Locations

Total: 35 locations

1

University of Alabama in Birmingham

Birmingham, Alabama, United States, 35294

Actively Recruiting

2

University of California San Diego Medical Center

La Jolla, California, United States, 92037

Actively Recruiting

3

Yale University School of Medicine

New Haven, Connecticut, United States, 06510

Actively Recruiting

4

Emory University

Atlanta, Georgia, United States, 30329

Actively Recruiting

5

Advocate Lutheran General Hospital

Park Ridge, Illinois, United States, 60068

Actively Recruiting

6

Indiana University School of Medicine

Indianapolis, Indiana, United States, 46202

Actively Recruiting

7

Washington University in St. Louis

St Louis, Missouri, United States, 63110

Actively Recruiting

8

New York University Medical Center

New York, New York, United States, 10016

Actively Recruiting

9

University of Pittsburgh

Pittsburgh, Pennsylvania, United States, 15213

Actively Recruiting

10

Butler Hospital

Providence, Rhode Island, United States, 02096

Actively Recruiting

11

Kerwin Research and Memory Center

Dallas, Texas, United States, 75231

Actively Recruiting

12

University of Washington

Seattle, Washington, United States, 98195

Actively Recruiting

13

Instituto de Investigaciones Neurologicas Raul Carrea, FLENI

Ciudad Autonoma de Buenos Aire, Argentina, C1428AQK

Actively Recruiting

14

Neuroscience Research Australia

Randwick, New South Wales, Australia, 2031

Actively Recruiting

15

Alzheimer's Research Australia

Melbourne, Victoria, Australia, 3010

Not Yet Recruiting

16

UBC Hospital

Vancouver, British Columbia, Canada, V6T 2B5

Actively Recruiting

17

Sunnybrook Health Sciences Centre

Toronto, Ontario, Canada, M4N 3M5

Actively Recruiting

18

McGill Center for Studies in Aging

Verdun, Quebec, Canada, H4H 1R3

Not Yet Recruiting

19

CHU de Quebec - Hôpital de l' Enfant Jésus

Québec, Canada, G1J 1Z4

Actively Recruiting

20

Grupo de Neurociencias Sede de la Universidad de Antioquia

Medellín, Colombia

Actively Recruiting

21

CHU de Toulouse - Hôpital Purpan

Toulouse, Haute Garonne, France, 31059

Not Yet Recruiting

22

Hopital Roger Salengro - CHU Lille

Lille, Nord, France, 59037

Not Yet Recruiting

23

Groupe Hospitalier Pitie-Salpetriere

Paris, Paris, France, 69677

Not Yet Recruiting

24

Hopital Neurologique Pierre Wertheimer

Bron, Rhone, France, 69677

Not Yet Recruiting

25

CHU de Rouen - Hôpital Charles Nicolle

Rouen, Seine Maritime, France, 76031

Not Yet Recruiting

26

Universitaetsklinikum Tubingen

Tübingen, Baden-Wurttemberg, Germany, 72076

Not Yet Recruiting

27

LMU-Campus Grosshadern

Munich, Bavaria, Germany, 81377

Not Yet Recruiting

28

IRCCS Centro San Giovanni di Dio Fatebenefratelli

Brescia, Italy, 25125

Not Yet Recruiting

29

Azienda Ospedaliera Universitaria Careggi

Florence, Italy, 50134

Not Yet Recruiting

30

Instituto Nacional de Neurologia y Neurocirugia Manuel Velasco Suarez

Mexico City, Mexico City, Mexico, 14269

Not Yet Recruiting

31

Brain Research Center

Amsterdam, Netherlands, 1081 GM

Not Yet Recruiting

32

New Zealand Brain Research Institute

Christchurch, New Zealand, 8011

Not Yet Recruiting

33

University of Puerto Rico, School of Medicine

San Juan, Puerto Rico, 00936

Actively Recruiting

34

Hospital Clínic I Provincial de Barcelona

Barcelona, Spain, 8036

Not Yet Recruiting

35

The National Hospital for Neurology and Neurosurgery

London, Greater London, United Kingdom, WC1B 3BG

Actively Recruiting

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Research Team

J

Jamie Bartzel

E

Ellen Ziegemeier

How is the study designed?

Study Type

INTERVENTIONAL

Masking

QUADRUPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

3

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