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A Study of LY3002813 in Participants With Early Symptomatic Alzheimer's Disease (TRAILBLAZER-ALZ)

Phase 2
Completed
Conditions
Alzheimer Disease
Interventions
Registration Number
NCT03367403
Lead Sponsor
Eli Lilly and Company
Brief Summary

The purpose of this study is to evaluate the safety, tolerability and efficacy of LY3002813 in early symptomatic Alzheimer's disease.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
272
Inclusion Criteria
  • Gradual and progressive change in memory function reported by participants or informants for ≥ 6 months.
  • MMSE score of 20 to 28 (inclusive) at baseline or an acceptable historical flortaucipir PET scan within 6 months prior to baseline that meets the central read criteria.
  • Meet 18F flortaucipir PET scan eligibility criteria.
  • Meet 18F florbetapir PET scan (central read) eligibility criteria.
Exclusion Criteria
  • Have a history of long QT syndrome.
  • Have received treatment with a stable dose of an acetylcholinesterase inhibitor (AChEI) and/or memantine for less than 2 months before randomization.
  • Contraindication to MRI.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboParticipants received placebo IV Q4W for up to 72 weeks.
Donanemab in Combination With LY3202626 (Donanemab-C)LY3202626Participants received 700 mg donanemab IV Q4W x 3 doses, then 1400 mg donanemab IV Q4W in combination with 12 mg of LY3202626 orally for up to 72 weeks. As per protocol amendment (d) approved on Oct 9, 2018, donanemab in combination with LY3202626 (donanemab-C) arm discontinued as there was a low probability of identifying a statistically significant effect of 12mg of LY3202626 slowing cognitive decline.
Donanemab Monotherapy (Donanemab-M)DonanemabParticipants received 700 milligram (mg) donanemab intravenously (IV) every 4 weeks (Q4W) x 3 doses, then 1400 mg donanemab IV Q4W for up to 72 weeks.
Donanemab in Combination With LY3202626 (Donanemab-C)DonanemabParticipants received 700 mg donanemab IV Q4W x 3 doses, then 1400 mg donanemab IV Q4W in combination with 12 mg of LY3202626 orally for up to 72 weeks. As per protocol amendment (d) approved on Oct 9, 2018, donanemab in combination with LY3202626 (donanemab-C) arm discontinued as there was a low probability of identifying a statistically significant effect of 12mg of LY3202626 slowing cognitive decline.
Primary Outcome Measures
NameTimeMethod
Change From Baseline in the Integrated Alzheimer's Disease Rating Scale (iADRS) ScoreBaseline, 76 Weeks

Integrated Alzheimer's Disease Rating Scale is used to assess whether donanemab slows down the cognitive and functional decline associated with AD compared with placebo. iADRS is a simple linear combination of 13-item alzheimer's disease assessment scale-cognitive subscale (ADAS-Cog13) and the Alzheimer's disease cooperative study-instrumental activities of daily living scale (ADCS-iADL). The scale ranges from 0 to 144, where lower scores indicate worse performance and higher score indicates better performance. Least Squares (LS) Mean value was controlled for fixed, categorical effects of treatment, visit and treatment-by-visit interaction, pooled investigator, acetylcholinesterase inhibitor (AChEI) and/or memantine use at baseline, as well as the continuous, fixed covariates of baseline, baseline-by-visit, and age at baseline.

Secondary Outcome Measures
NameTimeMethod
Change From Baseline in Brain Tau Deposition as Measured by Flortaucipir F18 PET ScanBaseline, 76 Weeks

Flortaucipir PET imaging was used as a quantitative tau biomarker. Flortaucipir PET scans at baseline and at 76 weeks after the first treatment were used to quantitatively estimate change in aggregated tau neurofibrillary tangles (NFTs). Quantitative tau burden was formalized using two measures: weighted average Standardized Uptake Value Ratio (MUBADA SUVR) in the brain relative to the cerebellum gray as a reference region and the global tau load (TauL) generated using a TauIQ method. Larger weighted average SUVR reflects the larger cortical tau burden relative to cerebellum gray. The TauIQ method quantifies the spatiotemporal accumulation pattern of tau and larger TauL value reflects the larger global tau level in the brain as determined using a TauIQ mathematical framework. Least Squares mean change was controlled for baseline + age + treatment (Type III sum of squares).

Change From Baseline in the 13-item Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog13) ScoreBaseline, 76 Weeks

The ADAS is a rater administered instrument that was designed to assess the severity of the dysfunction in the cognitive and noncognitive behaviors characteristic of persons with AD. The cognitive subscale of the ADAS consists of 13 items assessing areas of cognitive function most typically impaired in AD: orientation, verbal memory, language, praxis, delayed free recall, digit cancellation. The ADAS--Cog13 scale ranges from 0 to 85. Higher scores indicate greater disease severity. LS Mean value was controlled for fixed, categorical effects of treatment, visit and treatment-by-visit interaction, pooled investigator, AChEI and/or memantine use at baseline, as well as the continuous, fixed covariates of baseline, baseline-by-visit, and age at baseline.

Change From Baseline in the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB) ScoreBaseline, 76 Weeks

CDR-SB is a semi-structured interview of participants and their caregivers. Participant's cognitive status is rated across 6 domains of functioning, including memory, orientation, judgment/problem solving, community affairs, home/hobbies, and personal care. Severity score assigned for each of 6 domains; Total score (SB) ranges from 0 to 18. Higher scores indicate greater disease severity. LS Mean value was controlled for fixed, categorical effects of treatment, visit and treatment-by-visit interaction, pooled investigator, AChEI and/or memantine use at baseline, as well as the continuous, fixed covariates of baseline, baseline-by-visit, and age at baseline.

Change From Baseline in the Mini Mental State Examination (MMSE) ScoreBaseline, 76 Weeks

MMSE is a brief screening instrument used to assess cognitive function (orientation, memory, attention, ability to name objects, follow verbal/written commands, write a sentence, and copy figures). Total score ranges from 0 to 30; lower score indicates greater disease severity. LS Mean value was controlled for fixed, categorical effects of treatment, visit and treatment-by-visit interaction, pooled investigator, AChEI and/or memantine use at baseline, as well as the continuous, fixed covariates of baseline, baseline-by-visit, and age at baseline.

Change From Baseline in Alzheimer's Disease Cooperative Study-Instrumental Activities of Daily Living Scale (ADCS-iADL) ScoreBaseline, 76 Weeks

The ADCS-ADL is a 23-item inventory developed as a rater-administered questionnaire answered by the participant's caregiver. The ADCS-ADL measures both basic and instrumental activities (instrumental activity items 6a, 7-23) of daily living by participants. The range for the ADCS-iADL is 0-59 with higher scores reflecting better performance. LS Mean value was controlled for fixed, categorical effects of treatment, visit and treatment-by-visit interaction, pooled investigator, AChEI and/or memantine use at baseline, as well as the continuous, fixed covariates of baseline, baseline-by-visit, and age at baseline.

Change From Baseline in Brain Amyloid Plaque Deposition as Measured by Florbetapir F18 Positron Emission Tomography (PET) ScanBaseline, 76 Weeks

Florbetapir PET imaging was used as a quantitative amyloid biomarker. Florbetapir PET scans at baseline and at 76 weeks after the first treatment were used to quantitatively estimate change in amyloid plaques. Quantitative amyloid burden was first formalized as the average Standardized Uptake Value Ratio (SUVR) in six predetermined cortical areas of the brain relative to the cerebellum as a reference region. Larger SUVR reflects the larger cortical amyloid burden relative to cerebellum. SUVR values were further calibrated to a centiloid (CL) scale. The Centiloid scale anchor points are 0 and 100, where 0 represents a high-certainty amyloid negative scan and 100 represents the amount of global amyloid deposition found in a typical AD scan. Least Squares mean change was controlled for fixed, categorical effects of treatment, visit and treatment-by-visit interaction, as well as the continuous, fixed covariates of baseline, baseline-by-visit, and age at baseline.

Change From Baseline in Brain Volume as Measured by Volumetric Magnetic Resonance Imaging (vMRI)Baseline, 76 Weeks

MRI scans at baseline and at 76 weeks after the first treatment were used to quantitatively estimate change in brain atrophy. Volumetric MRI (vMRI) parameters were measured in 14 brain regions: bilateral cortical, bilateral entorhinal cortex, bilateral hippocampus, bilateral inferior parietal lobe, bilateral isthmus cingulate, bilateral lateral parietal lobe, bilateral medial temporal lobe, bilateral precuneus, bilateral prefrontal lobe, bilateral superior temporal lobe, bilateral ventricles, bilateral whole brain, bilateral whole temporal lobe, and bilateral white matter. The atrophy was assessed by tensor-based morphometry, which captures volume changes within the deformation map. LS Mean value was controlled for fixed, categorical effects of treatment, visit and treatment-by-visit interaction, fixed covariates of baseline, and age at baseline.

Trial Locations

Locations (60)

Toronto Memory Program

🇨🇦

Toronto, Ontario, Canada

Texas Neurology, PA

🇺🇸

Dallas, Texas, United States

Bradenton Research Center

🇺🇸

Bradenton, Florida, United States

University of Kansas Hospital

🇺🇸

Fairway, Kansas, United States

Ohio State University Medical Center

🇺🇸

Columbus, Ohio, United States

Miami Jewish Health Systems

🇺🇸

Miami, Florida, United States

Indiana University School of Medicine

🇺🇸

Indianapolis, Indiana, United States

Josephson Wallack Munshower Neurology

🇺🇸

Indianapolis, Indiana, United States

Houston Methodist

🇺🇸

Houston, Texas, United States

Pacific Research Network Inc

🇺🇸

San Diego, California, United States

Sharp Mesa Vista Hospital

🇺🇸

San Diego, California, United States

Banner Alzheimer's Institute

🇺🇸

Phoenix, Arizona, United States

Las Vegas Medical Research

🇺🇸

Las Vegas, Nevada, United States

Guilford Neurologic Associates

🇺🇸

Greensboro, North Carolina, United States

Neurology Center of North Orange County

🇺🇸

Fullerton, California, United States

Banner Sun Health Research Institute

🇺🇸

Sun City, Arizona, United States

Irvine Clinical Research Center

🇺🇸

Irvine, California, United States

Syrentis Clinical Research

🇺🇸

Santa Ana, California, United States

Associated Neurologists, PC - Danbury

🇺🇸

Danbury, Connecticut, United States

KI Health Partners, LLC d/b/a NE Inst. for Clin. Res.

🇺🇸

Stamford, Connecticut, United States

Pharmacology Research Institute

🇺🇸

Newport Beach, California, United States

Infinity Clinical Research, LLC

🇺🇸

Hollywood, Florida, United States

Brain Matters Research

🇺🇸

Delray Beach, Florida, United States

Pharmax Research Clinic

🇺🇸

Miami, Florida, United States

Merritt Island Medical Research LLC

🇺🇸

Merritt Island, Florida, United States

Jacksonville Center for Clinical Research

🇺🇸

Jacksonville, Florida, United States

Suncoast Clinical Research

🇺🇸

New Port Richey, Florida, United States

Quantum Laboratories

🇺🇸

Pompano Beach, Florida, United States

Palm Beach Neurological Group

🇺🇸

Palm Beach Gardens, Florida, United States

Compass Research

🇺🇸

The Villages, Florida, United States

Great Lakes Clinical Trials

🇺🇸

Chicago, Illinois, United States

Alexian Brothers Medical Center

🇺🇸

Elk Grove Village, Illinois, United States

Cotton O'Neil Clinic

🇺🇸

Topeka, Kansas, United States

Washington University

🇺🇸

Saint Louis, Missouri, United States

Donald S Marks

🇺🇸

Plymouth, Massachusetts, United States

ActivMed Practices & Research, Inc

🇺🇸

Methuen, Massachusetts, United States

Boston Center for Memory

🇺🇸

Newton, Massachusetts, United States

Behavioral Health Center Research

🇺🇸

Charlotte, North Carolina, United States

Advanced Memory Research Institute of New Jersey

🇺🇸

Toms River, New Jersey, United States

Insight Clinical Trials

🇺🇸

Beachwood, Ohio, United States

Raleigh Neurology Associates

🇺🇸

Raleigh, North Carolina, United States

Neurology Diagnostics, Inc.

🇺🇸

Dayton, Ohio, United States

Butler Hospital

🇺🇸

Providence, Rhode Island, United States

Abington Neurological Associates

🇺🇸

Willow Grove, Pennsylvania, United States

Cognition Health

🇺🇸

Fairfax, Virginia, United States

The Memory Clinic

🇺🇸

Bennington, Vermont, United States

Bruyere Research Institute

🇨🇦

Ottawa, Ontario, Canada

Kawartha Regional Memory Clinic

🇨🇦

Peterborough, Ontario, Canada

Clinique de la Memoire de l'Outaouais

🇨🇦

Gatineau, Quebec, Canada

DIEX Recherche Sherbrooke, Inc

🇨🇦

Sherbrooke, Quebec, Canada

Institute for Memory Impairment & Neurological Disorders

🇺🇸

Irvine, California, United States

Intercoastal Medical Group

🇺🇸

Sarasota, Florida, United States

JEM Research Institute

🇺🇸

Atlantis, Florida, United States

Progressive Medical Research

🇺🇸

Port Orange, Florida, United States

McLean Hospital

🇺🇸

Belmont, Massachusetts, United States

Stedman Clinical Trials

🇺🇸

Tampa, Florida, United States

Axiom Research

🇺🇸

Tampa, Florida, United States

Piedmont Medical Research

🇺🇸

Winston-Salem, North Carolina, United States

National Clinical Research - Richmond

🇺🇸

Richmond, Virginia, United States

Rhode Island Hospital

🇺🇸

Providence, Rhode Island, United States

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