Amyloid Lowering for Alzheimer's in Down's With Donanemab Investigation
Trial statusNot yet recruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age35-50
SponsorMichael Rafii, MD, PhD
About this trial
The goal of this clinical trial is to learn if donanemab can reduce levels of amyloid in the brain, and if donanemab is safe and well-tolerated in participants with Down syndrome.
The main questions it aims to answer are: Does donanemab reduce amyloid in the brain? Is donanemab safe and well-tolerated in people with Down syndrome? Researchers will compare donanemab to a placebo (a look-alike substance that contains no drug) to see if donanemab works to reduce levels of amyloid in the brain.
Participants in the study will be 35-50 years old and will be in the study for 12 months. Participants will then stay in the study for an additional 12 months in an long-term extension where all participants will receive donanemab. Participants who had a reduction in amyloid (measured by amyloid brain scan) by the end of the first 12 months will receive placebo for the long-term extension, while participants who did not have an amyloid reduction will receive study donanemab for the long-term extension. Everyone (participants and study staff) will remain blinded to treatment for the duration of the study.
Participants will:
* Have intravenous (IV) infusions of donanemab (or placebo) every 4 weeks * Visit the clinic once every other month for checkups and tests. These tests will include brain scans (magnetic resonance imaging \[MRI\] and positron emission tomography \[PET\] ), blood draws and memory tests. * Have a study partner who who can provide information about the participant and can join participant for some of the study visits.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Documentation of the participant's informed consent to study procedures
Ages 35-50 years (inclusive).
Plasma Phosphorylated tau (pTau) 217 levels at screening consistent with amyloid PET eligibility.
Diagnosis of Down syndrome (including trisomy 21, mosaic trisomy 21, Robertsonian translocation trisomy 21, or partial trisomy 21) as confirmed by medical record review or Karyotype genetic testing.
Disqualifiers
Females who are lactating or pregnant (as confirmed by a urine pregnancy test) during screening, or plan to become pregnant during the study.
Females of childbearing potential or males with a female partner of childbearing potential who did not use a highly effective method of contraception within 28 days of screening alongside with a suitable barrier method (e.g., male condom) and/or are not willing to use both methods for the duration of their participation in the study and for 3 months after the last dose of study drug.
Weight less than 40kg at screening.
Lack of good venous access such that intravenous (IV) drug delivery or multiple blood draws would be precluded.
Trial design
Design model
Parallel
Treatments tested in this trial
Donanemab
DrugDonanemab will be administered as an IV infusion every 4 weeks using an up-titration regimen that will allow participants to reach a target dose of 1400 mg within 24 weeks.
Placebo
DrugThe control product (placebo) will be supplied in vials containing 350 mg/20 mL of donanemab Placebo for donanemab is supplied to the clinical site to match the active study intervention and contains only inactive ingredients. Normal sterile saline solution (0.9% sodium chloride) will be used for dilution.
Treatment groups
60 Participants
are divided into 2 treatment groupsGroup A: donanemabExperimental treatment 1 intervention
Group B: placeboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Change from baseline on brain amyloid levels
Change in brain amyloid measured by amyloid PET imaging (amyloid centiloid levels) from baseline to 12 months. An increase indicates a worsening.
Time frame
12 monthsSecondary outcomes
1
Change from baseline to 12 months in the Modified Cued Recall Test (mCRT)
The Modified Cued Recall Test (mCRT) is a cognitive assessment tool designed to evaluate memory and has been modified from a version developed for adults from the typical population. 12 items are presented for learning, 4 at a time, with each item accompanied by a unique category cue. The testing phase consists of 3 trials. Each trial begins with free recall of the test items; following free recall, a category cue is provided for those items not spontaneously recalled. Two scores are generated from the test, a Free Recall Score and a Total Score. A higher score indicates a better outcome.
Time frame
From baseline to 12 months2
Change from baseline in whole brain volume
Change in whole brain volume as measured by MRI. A decrease indicates a worsening.
Time frame
From baseline to 12 months3
Change from baseline in brain hippocampal volume
Change in brain hippocampal volume as measured by MRI. A decrease indicates a worsening.
Time frame
From baseline to 12 monthsLocations
Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Michael Rafii, MD, PhD
Lead sponsor
University of Southern California
Sponsor institution
National Institute on Aging (NIA)
Collaborator
Eli Lilly and Company
Collaborator
Alzheimer's Clinical Trials Consortium
Collaborator
Alzheimer's Therapeutic Research Institute
Collaborator
Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: