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Multiple Dose Study of Aducanumab (BIIB037) (Recombinant, Fully Human Anti-Aβ IgG1 mAb) in Participants With Prodromal or Mild Alzheimer's Disease

Phase 1
Terminated
Conditions
Alzheimer's Disease
Interventions
Registration Number
NCT01677572
Lead Sponsor
Biogen
Brief Summary

The primary objective of this study is to evaluate the safety and tolerability of multiple doses of Aducanumab (recombinant, fully human anti-Aβ IgG1 mAb) in participants with prodromal or mild Alzheimer's Disease (AD). The secondary objectives of this study are to assess the effect on cerebral amyloid plaque content as measured by florbetapir-fluorine-18 (18F-AV-45F-AV-45) positron emission tomography (PET) imaging, to assess the multiple dose serum concentrations of Aducanumab and to evaluate the immunogenicity of Aducanumab after multiple dose administration in this population.

Detailed Description

The study consists of a placebo-controlled period to study week 54, followed by a long-term extension to study week 518. The placebo-controlled period is conducted with a staggered, parallel group design, with the first 3 treatment arms conducted in parallel, 2 further treatment arms subsequently beginning in parallel, 2 additional treatment arms beginning in parallel, and the last 2 treatment arms subsequently beginning in parallel. Qualifying participants can enter the long-term extension period for up to 42 additional doses of active drug for the first 3 years of LTE. Furthermore, up until the last participant in Arms 8 and 9 has had his or her last dose in the fifth year of the LTE, eligible participants will be able to continue treatment beyond the third year of the LTE.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
197
Inclusion Criteria
  • Participants must be ambulatory.
  • Participants must meet the following core clinical criteria as determined by the Investigator:

Prodromal Alzheimer's Disease (AD) (all of the criteria must apply):

  • Mini Mental State Examination (MMSE) scores between 24-30 (inclusive)
  • a spontaneous memory complaint
  • objective memory loss defined as a free recall score of ≤27 on the Free and Cued Selective Reminding Test (FCSRT)
  • a global Clinical Dementia Rating Scale (CDR) score of 0.5
  • absence of significant levels of impairment in other cognitive domains
  • essentially preserved activities of daily living, and an absence of dementia. OR

Mild Alzheimer's Disease (AD) criteria (all criteria must apply):

  • Mini Mental State Examination (MMSE) scores between 20-26 (inclusive)
  • a global Clinical Dementia Rating Scale (CDR) of 0.5 or 1.0
  • meeting the National Institute on Aging-Alzheimer's Association core clinical criteria for probable AD.
  • Participants must have a positive florbetapir positron emission tomography (PET) amyloid scan.
  • Participants must consent to apolipoprotein E (ApoE) genotyping.
  • Apart from clinical diagnosis of Alzheimer's Disease (AD), participant must be in good health.
  • Must have a reliable informant or caregiver.

Key

Exclusion Criteria
  • Any medical or neurological condition (other than Alzheimer's Disease) that might be a contributing cause of the participant's cognitive impairment.
  • Have had a stroke or Transient Ischemic Attack (TIA) or unexplained loss of consciousness in the past 1 year.
  • Clinically significant psychiatric illness in past 6 months.
  • Seizure in the past 3 years.
  • Poorly controlled diabetes mellitus.
  • History of unstable angina, myocardial infarction, chronic heart failure, or clinical significant conduction abnormalities within 1 year prior to Screening.
  • Indication of impaired renal or liver function.
  • Have human immunodeficiency virus (HIV) infection.
  • Have a significant systematic illness or infection in past 30 days.
  • Brain MRI showing evidence of acute or sub-acute micro or macrohemorrhage, greater than 4 microhemorrhages, cortical infarct or greater than one 1 lunar infarct.
  • Any contraindications to brain MRI or positron emission tomography (PET) scans.
  • Negative positron emission tomography (PET) scan with any amyloid-targeting ligand within 48 weeks of Screening.
  • Clinically significant 12-lead electrocardiogram (ECG) abnormalities.
  • Alcohol or substance abuse in past 1 year.
  • Taking blood thinners (except for aspirin at a prophylactic dose or less)
  • Have changes in medications or doses of medication in past 4 weeks.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Low-dose #1 AducanumabAducanumab (recombinant, fully human anti-Aβ IgG1 mAb)Intravenous doses of low-dose level #1 Aducanumab administered approximately 4 weeks apart over approximately 52 weeks (a total of 14 doses). Qualifying participants can continue into the long-term extension at a dose administered approximately 4 weeks apart for up to an additional 112 doses.
Low-dose #2 AducanumabAducanumab (recombinant, fully human anti-Aβ IgG1 mAb)Intravenous doses of low-dose level #2 Aducanumab administered approximately 4 weeks apart over approximately 52 weeks (a total of 14 doses). Qualifying participants can continue into the long-term extension at a dose administered approximately 4 weeks apart for up to an additional 112 doses.
Placebo (low dose group)Aducanumab (recombinant, fully human anti-Aβ IgG1 mAb)Intravenous doses of placebo administered approximately 4 weeks apart over approximately 52 weeks (a total of 14 doses). Qualifying participants can continue into the long-term extension at a dose approximately 4 weeks apart for up to an additional 112 doses.
Mid-dose AducanumabAducanumab (recombinant, fully human anti-Aβ IgG1 mAb)Intravenous doses of mid-dose Aducanumab administered approximately 4 weeks apart over approximately 52 weeks (a total of 14 doses). Qualifying participants can continue into the long-term extension at a dose administered approximately 4 weeks apart for up to an additional 112 doses.
Placebo (mid dose group)Aducanumab (recombinant, fully human anti-Aβ IgG1 mAb)Intravenous doses of placebo administered approximately 4 weeks apart over approximately 52 weeks (a total of 14 doses). Qualifying participants can continue into the long-term extension at a dose administered approximately 4 weeks apart for up to an additional 112 doses.
High-dose AducanumabAducanumab (recombinant, fully human anti-Aβ IgG1 mAb)Intravenous doses of high-dose Aducanumab administered approximately 4 weeks apart over approximately 52 weeks (a total of 14 doses). Qualifying participants can continue into the long-term extension at a dose administered approximately 4 weeks apart for up to an additional 112 doses.
Placebo (high dose group)Aducanumab (recombinant, fully human anti-Aβ IgG1 mAb)Intravenous doses of placebo administered approximately 4 weeks apart over approximately 52 weeks (a total of 14 doses). Qualifying participants can continue into the long-term extension at a dose administered approximately 4 weeks apart for up to an additional 112 doses.
Aducanumab TitrationAducanumab (recombinant, fully human anti-Aβ IgG1 mAb)Intravenous doses of Aducanumab administered approximately 4 weeks apart over approximately 52 weeks (a total of 14 doses). Qualifying participants can continue into the long-term extension at a dose approximately 4 weeks apart for up to an additional 112 doses.
Placebo (mid dose group)PlaceboIntravenous doses of placebo administered approximately 4 weeks apart over approximately 52 weeks (a total of 14 doses). Qualifying participants can continue into the long-term extension at a dose administered approximately 4 weeks apart for up to an additional 112 doses.
Placebo (low dose group)PlaceboIntravenous doses of placebo administered approximately 4 weeks apart over approximately 52 weeks (a total of 14 doses). Qualifying participants can continue into the long-term extension at a dose approximately 4 weeks apart for up to an additional 112 doses.
Placebo (high dose group)PlaceboIntravenous doses of placebo administered approximately 4 weeks apart over approximately 52 weeks (a total of 14 doses). Qualifying participants can continue into the long-term extension at a dose administered approximately 4 weeks apart for up to an additional 112 doses.
Placebo (Titration Group)PlaceboIntravenous doses of placebo administered approximately 4 weeks apart over approximately 52 weeks (a total of 14 doses). Qualifying participants can continue into the long-term extension at a dose approximately 4 weeks apart for up to an additional 112 doses.
Primary Outcome Measures
NameTimeMethod
Number of Participants with Adverse EventsBaseline to week 518
Secondary Outcome Measures
NameTimeMethod
Change from Baseline in Incidence of Anti-Aducanumab Antibodies in Serum.Up to week 518
Multiple dose pharmacokinetic (PK) serum concentrations of AducanumabUp to week 518
Change from baseline in florbetapir-fluorine-18 (18F-AV-45F-AV-45) positron emission tomography (PET) imaging in certain brain areas.Day 1, Weeks 26, 54, End of year 2, 3, and 4

Trial Locations

Locations (32)

Infinity Clinical Research, Inc.

🇺🇸

Sunrise, Florida, United States

Senior Clinical Trials, Inc.

🇺🇸

Laguna Hills, California, United States

University of California, Los Angeles

🇺🇸

Los Angeles, California, United States

Brain Matters Research, Inc.

🇺🇸

Delray Beach, Florida, United States

Neuropsychiatric Research Center of Southwest Florida

🇺🇸

Fort Myers, Florida, United States

Miami Jewish Health Systems

🇺🇸

Miami, Florida, United States

Pacific Research Network, Inc.

🇺🇸

San Diego, California, United States

San Francisco Clinical Research Center

🇺🇸

San Francisco, California, United States

Summit Research Network (Oregon) Inc.

🇺🇸

Portland, Oregon, United States

Axiom Clinical Research of Florida

🇺🇸

Tampa, Florida, United States

Stedman Clinical Trials, LLC

🇺🇸

Tampa, Florida, United States

Collaborative Neuroscience Network, LLC

🇺🇸

Long Beach, California, United States

Torrance Clinical Research Institute, Inc.

🇺🇸

Lomita, California, United States

NNS Clinical Research, LLC

🇺🇸

Tucson, Arizona, United States

Stanford University Medical Center

🇺🇸

Stanford, California, United States

Pacific Neuroscience Medical Group

🇺🇸

Oxnard, California, United States

Georgetown University Hospital

🇺🇸

Washington, District of Columbia, United States

MD Clinical Trials, Inc.

🇺🇸

Hallandale Beach, Florida, United States

Galiz Research, LLC

🇺🇸

Miami Springs, Florida, United States

Alexian Brothers Neurosciences Institute

🇺🇸

Elk Grove Village, Illinois, United States

Neurostudies.net, LLC

🇺🇸

Decatur, Georgia, United States

Indiana University School of Medicine

🇺🇸

Indianapolis, Indiana, United States

St. Louis Clinical Trials, LLC

🇺🇸

Saint Louis, Missouri, United States

Memory Enhancement Center of America, Inc.

🇺🇸

Eatontown, New Jersey, United States

Advanced Memory Research Institute of NJ

🇺🇸

Toms River, New Jersey, United States

CRI Lifetree

🇺🇸

Marlton, New Jersey, United States

Empire Neurology, PC

🇺🇸

Latham, New York, United States

Insight Clinical Trials LLC

🇺🇸

Beachwood, Ohio, United States

Brown Hospital

🇺🇸

East Providence, Rhode Island, United States

Alzheimer's Disease Research Unit, Yale University

🇺🇸

New Haven, Connecticut, United States

Compass Research, LLC

🇺🇸

Orlando, Florida, United States

Rhode Island Hospital

🇺🇸

Providence, Rhode Island, United States

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