Actively Recruiting

Phase 2
Phase 3
Age: 18Years +
All Genders
Healthy Volunteers
NCT05552157

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

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

280

Participants Needed

35

Research Sites

509 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

The purpose is to evaluate the biomarker effect, safety, and tolerability of investigational study drugs in participants who are known to have an Alzheimer's disease (AD)-causing mutation. Stage 1 will determine if treatment with the study drug prevents or slows the rate of amyloid beta (Aβ) pathological disease accumulation demonstrated by Aβ positron emission tomography (PET) imaging. Stage 2 will evaluate the effect of early Aβ plaque reduction/prevention on disease progression by assessing downstream non-Aβ biomarkers of AD (e.g., CSF total tau, p-tau, NfL) compared to an external control group from the DIAN-OBS natural history study and the DIAN-TU-001 placebo-treated participants.

CONDITIONS

Official 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 signed by the participant and study partner or legally authorized representative
  • Participant is at least 18 years old
  • People who can become pregnant must have a negative pregnancy test at screening
  • Agree not to become pregnant or breastfeed during the study until 5 half-lives after the last dose
  • Agree to use effective contraception if applicable from screening until 5 half-lives after last dose
  • Carrier of APP, PSEN1, or PSEN2 mutation linked to DIAD or at direct risk through family mutation
  • Within 25 to 11 years before predicted age of cognitive symptom onset based on mutation or family history
  • Normal cognitive status (CDR-SB 0)
  • Fluent in DIAN-TU approved language with adequate premorbid intellectual functioning
  • Adequate visual and hearing abilities for assessments
  • Stable medication doses for non-excluded conditions for at least 30 days before baseline except episodic medications
  • Has a study partner able to provide accurate cognitive and functional information and signs consent
  • Agree not to donate blood or blood products during the study and for 5 half-lives after last dose
  • Likely to comply and complete the study
  • Able and willing to complete all study testing and procedures
Not Eligible

You will not qualify if you...

  • Significant neurologic or psychiatric diseases that affect cognition or study participation
  • High risk for suicide or recent major depression
  • History of stroke or significant stroke risk factors impacting cognition or study ability
  • Alcohol or substance use disorder within past year
  • Brain MRI showing significant abnormalities such as microhemorrhages or aneurysms
  • Implanted medical devices incompatible with MRI
  • Cardiovascular conditions interfering with study participation
  • Liver or kidney problems interfering with study participation
  • History of HIV, recent Hepatitis B or untreated Hepatitis C, or CNS infections
  • Severe drug allergies or hypersensitivity reactions related to study drugs or imaging agents
  • Recent immunosuppressive or chemotherapy treatments
  • Thyroid abnormalities or significant vitamin B12 deficiency
  • Unstable or poorly controlled diabetes
  • Morbid obesity affecting MRI imaging
  • Current use of anticoagulants except low dose aspirin
  • Recent exposure to monoclonal antibodies targeting amyloid beta
  • Recent use of other investigational drugs
  • Insufficient venous access
  • Abnormal hematology, coagulation, or clinical chemistry
  • History of cancer with high risk of recurrence impacting participation
  • Any other medical condition that may bias assessments or increase risk
  • Recent participation in other clinical studies without approval
  • Participants with the "Dutch" APP E693Q mutation
  • Unable to complete baseline procedures with required cognitive and clinical scores

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

1
2
3
+1

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

Loading map...

Research Team

J

Jamie Bartzel

CONTACT

E

Ellen Ziegemeier

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

QUADRUPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

3

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here