MedPath

A Study to Learn About the Safety of BIIB080 Injections and Whether They Can Improve Symptoms of Participants With Mild Cognitive Impairment Due to Alzheimer's Disease (AD) or Mild AD Dementia Between 50 to 80 Years of Age

Phase 2
Active, not recruiting
Conditions
Alzheimer's Disease Dementia
Mild Cognitive Impairment Due to Alzheimer's Disease
Interventions
Drug: BIIB080-matching placebo
Registration Number
NCT05399888
Lead Sponsor
Biogen
Brief Summary

In this study, researchers will learn more about a study drug called BIIB080. The study will focus on participants with mild cognitive impairment or mild dementia due to AD.

The main question researchers are trying to answer is if BIIB080 can slow the worsening of AD more than placebo. It will focus on what dose of BIIB080 slows worsening of AD the most.

To help answer this question, researchers will use the Clinical Dementia Rating-Sum of Boxes, also known as the CDR-SB.

* Clinicians use the CDR-SB to measure several categories of dementia symptoms.

* The results for each category are added together for a total score. Lower scores are better.

Researchers will also learn more about the safety of BIIB080.

The study will be split into 2 parts. The 1st part is the Placebo-Controlled Period. The 2nd part is the Long-Term Extension (LTE) Period. The 2nd part of the study will help researchers learn about the long-term safety of BIIB080, and how it affects the participant's daily life, thinking, and memory abilities in the longer term.

A description of how the study will be done is given below.

* After screening, participants will first receive either a low dose or high dose of BIIB080, or a placebo, as an injection into the fluid around the spinal cord (cerebrospinal fluid). A placebo looks like the study drug but contains no real medicine.

* Participants will receive BIIB080 or placebo once every 12 weeks or 24 weeks.

* After 76 weeks of treatment in the Placebo-Controlled Period, eligible participants will move onto the Extension Treatment period, which will last 96 weeks.

* In the extension period, participants who received placebo will be switched to high dose BIIB080 every 12 or 24 weeks.

* Participants may be in the study for up to 201 weeks, or about 4 years. This includes the screening and follow-up periods.

* Participants can continue to take certain medications for AD. Participants must be on the same dose of medication for at least 8 weeks before the screening period.

* After the screening period, most participants will visit the clinic every 6 weeks.

Detailed Description

BIIB080 is an investigational antisense therapy designed to target microtubule-associated protein tau (MAPT) messenger ribonucleic acid (mRNA) and prevent production of tau protein.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
416
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Placebo Q12WBIIB080Participants will receive BIIB080-matching placebo, intrathecal (IT) injection, once on Day 1 and then once every 12 weeks (Q12W) for up to 72 weeks, during the placebo-controlled period. Eligible participants will enter the LTE period, during which they will be randomized to receive BIIB080 high dose, IT injection, either Q12W or once every 24 weeks (Q24W) for an additional 96 weeks.
BIIB080 High Dose Q24WBIIB080-matching placeboParticipants will receive a high dose of BIIB080, IT injection, Q24W from Week 1 up to 72 weeks and BIIB080-matching placebo, IT injection, once at Weeks 12, 36, and 60 during the placebo-controlled period. Eligible participants will enter the LTE period, during which they will continue to receive BIIB080 high dose, IT injection, Q24W for an additional 96 weeks.
Placebo Q12WBIIB080-matching placeboParticipants will receive BIIB080-matching placebo, intrathecal (IT) injection, once on Day 1 and then once every 12 weeks (Q12W) for up to 72 weeks, during the placebo-controlled period. Eligible participants will enter the LTE period, during which they will be randomized to receive BIIB080 high dose, IT injection, either Q12W or once every 24 weeks (Q24W) for an additional 96 weeks.
BIIB080 Low Dose Q24WBIIB080-matching placeboParticipants will receive a low dose of BIIB080, IT injection, Q24W from Week 1 up to 72 weeks and BIIB080-matching placebo, IT injection, once at Weeks 12, 36, and 60 during the placebo-controlled period. Eligible participants will enter the LTE period, during which they will continue to receive BIIB080 low dose, IT injection, Q24W for an additional 96 weeks.
BIIB080 High Dose Q12WBIIB080Participants will receive a high dose of BIIB080, IT injection, once on Day 1 and then Q12W for up to 72 weeks during the placebo-controlled period. Eligible participants will enter the LTE period, during which they will continue to receive BIIB080 high dose, IT injection, Q12W for an additional 96 weeks.
BIIB080 Low Dose Q24WBIIB080Participants will receive a low dose of BIIB080, IT injection, Q24W from Week 1 up to 72 weeks and BIIB080-matching placebo, IT injection, once at Weeks 12, 36, and 60 during the placebo-controlled period. Eligible participants will enter the LTE period, during which they will continue to receive BIIB080 low dose, IT injection, Q24W for an additional 96 weeks.
BIIB080 High Dose Q24WBIIB080Participants will receive a high dose of BIIB080, IT injection, Q24W from Week 1 up to 72 weeks and BIIB080-matching placebo, IT injection, once at Weeks 12, 36, and 60 during the placebo-controlled period. Eligible participants will enter the LTE period, during which they will continue to receive BIIB080 high dose, IT injection, Q24W for an additional 96 weeks.
Primary Outcome Measures
NameTimeMethod
Dose response in Change From Baseline to Week 76 on the CDR-SBBaseline to Week 76

The Clinical Dementia Rating (CDR) scale is a clinician-rated dementia staging system that tracks the progression of cognitive impairment in 6 categories (memory, orientation, judgment and problem solving, community affairs, home and hobbies, and personal care). Each category is scored on a 5-point scale in which None=0, Questionable=0.5, Mild=1, Moderate=2, and Severe=3. The global CDR score is established by clinical scoring rules and has values of 0 (no dementia), 0.5, (questionable dementia), 1 (mild dementia), 2 (moderate dementia), and 3 (severe dementia). The CDR-SB is obtained by adding the ratings in each of the 6 categories and ranges from 0 to 18 with higher scores indicative of greater impairment.

Secondary Outcome Measures
NameTimeMethod
Change From Baseline to Week 76 on the Alzheimer's Disease Assessment Scale Cognitive Subscale (ADAS-Cog 13)Baseline to Week 76

ADAS-Cog13 comprises both cognitive tasks and clinical ratings of cognitive performance. The scale items capture word recall, ability to follow commands, the ability to correctly copy or draw an image, naming, the ability to interact with everyday objects, orientation, word recognition, memory, comprehension of spoken language, word-finding, and language ability, with a measure for delayed word recall and concentration/distractibility. The total score ranges from 0 to 85. An increase in score over time indicates increasing cognitive impairment. Positive change from baseline indicates clinical decline.

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)From first dose of study drug up to end of study of placebo-controlled period (up to Week 96)

An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory finding), symptom, or disease temporally associated with use of a medicinal (investigational) product, whether or not related to medicinal (investigational) product. TEAE is any AE that started or worsened on or after the administration of the first dose of study drug through the end of follow-up period. SAE is any untoward medical occurrence that at any dose results in death, in the view of investigator, places the participant at immediate risk of death (life-threatening event), requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in congenital anomaly/birth defect or is medically important event.

Change From Baseline to Week 76 on the CDR-SBBaseline to Week 76

The CDR scale is a clinician-rated dementia staging system that tracks the progression of cognitive impairment in 6 categories (memory, orientation, judgment and problem solving, community affairs, home and hobbies, and personal care). Each category is scored on a 5-point scale in which None=0, Questionable=0.5, Mild=1, Moderate=2, and Severe=3. The global CDR score is established by clinical scoring rules and has values of 0 (no dementia), 0.5, (questionable dementia), 1 (mild dementia), 2 (moderate dementia), and 3 (severe dementia). The CDR-SB is obtained by adding the ratings in each of the 6 categories and ranges from 0 to 18 with higher scores indicative of greater impairment. Positive change from baseline indicates clinical decline.

Change From Baseline to Week 76 on the Alzheimer's Disease Cooperative Study Activities of Daily Living for Mild Cognitive Impairment (ADCS-ADL-MCI)Baseline to Week 76

The ADCS-ADL-MCI consists of 17 instrumental items (e.g., shopping, preparing meals, using household appliances) and 1 basic item (getting dressed). Ratings reflect caregiver observations about the participant's actual functioning and provide an assessment of change in the functional state of the participant over time. The total score ranges from 0 to 53, with lower values over time reflecting functional deterioration. Positive change from baseline indicates clinical improvement.

Change From Baseline to Week 76 on the Modified Integrated Alzheimer's Disease Rating Scale (iADRS)Baseline to Week 76

iADRS is a composite and is calculated as a linear combination of total scores of ADAS-Cog13 and Alzheimer's Disease Cooperative Study Instrumental Activities of Daily Living Inventory (ADCS-iADL) that measures cognition and daily function. ADCS-iADL is calculated from a subset of questions from ADCS-ADL. Range for ADCS-iADL is 0-59 and higher scores reflect better performance. ADAS-Cog13 comprises cognitive tasks and clinical ratings of cognitive performance. Scale items capture word recall, ability to follow commands, ability to correctly copy/draw, naming, ability to interact with everyday objects, orientation, word recognition, memory, spoken language comprehension, word-finding, and language ability, with a measure for delayed word recall and concentration/distractibility. Score ranges from 0 to 85 with higher scores reflecting cognitive impairment. iADRS score range is 0-144 and higher scores indicate greater impairment. Positive change from baseline indicates clinical decline.

Change From Baseline to Week 76 on the Mini Mental State Examination (MMSE)Baseline to Week 76

The MMSE is a widely used performance-based test of global cognitive status. It consists of 11 tasks that assess orientation, word recall, attention and calculation, language abilities, and visuospatial functions. The scores from the 11 tests are combined to obtain the total score, which ranges from 0 to 30, with lower scores over time indicating increasing cognitive impairment. Positive change from baseline indicates clinical improvement.

Change From Baseline to Week 76 on the Alzheimer's Disease Composite Score (ADCOMS)Baseline to Week 76

ADCOMS is a composite score comprised of ADAS-cog (4 items), MMSE (2 items) and CDR-SB (6 items). The total scores on the scale range from 0 to 1.97 with higher scores indicating greater impairment. Positive change from baseline indicates clinical decline.

Trial Locations

Locations (137)

Mary S. Easton Center for Alzheimer's Disease Research, UCLA

🇺🇸

Los Angeles, California, United States

University of California San Diego Medical Center

🇺🇸

San Diego, California, United States

University of California San Francisco (PARENT)

🇺🇸

San Francisco, California, United States

Ålborg Universitets Hospital

🇩🇰

Ålborg, Denmark

Charter Research, LLC

🇺🇸

Winter Park, Florida, United States

Conquest Research

🇺🇸

Winter Park, Florida, United States

Brigham and Women's Hospital Department of Neurology

🇺🇸

Boston, Massachusetts, United States

Beth Israel Deaconess Medical Center

🇺🇸

Boston, Massachusetts, United States

Lahey Clinic Medical Center - Burlington

🇺🇸

Burlington, Massachusetts, United States

Neurological Associates of Albany, PC

🇺🇸

Albany, New York, United States

SUNY Upstate Medical University

🇺🇸

Syracuse, New York, United States

Neurology Consultants of Dallas, PA

🇺🇸

Dallas, Texas, United States

Recherches Neuro-Hippocampe Inc., d/b/a Ottawa Memory Clinic

🇨🇦

Ottawa, Ontario, Canada

Medical Arts Health Research Group

🇨🇦

West Vancouver, British Columbia, Canada

HonorHealth Neurology

🇺🇸

Scottsdale, Arizona, United States

The Medical Arts Health Research Group

🇨🇦

Kamloops, British Columbia, Canada

Neurology Clinic, PC

🇺🇸

Cordova, Tennessee, United States

Jewish General Hospital - NETWORK

🇨🇦

Montreal, Quebec, Canada

AZ Groeninge

🇧🇪

Kortrijk, Belgium

Azienda Ospedaliera Card. G. Panico

🇮🇹

Tricase, Lecce, Italy

Tokyo Metropolitan Institute for Geriatrics and Gerontology

🇯🇵

Itabashi-ku, Tokyo-To, Japan

FORBELI s.r.o.

🇨🇿

Praha 6, Czechia

Universitaetsklinikum Ulm

🇩🇪

Ulm, Baden Wuerttemberg, Germany

Universitaetsmedizin Goettingen

🇩🇪

Goettingen, Niedersachsen, Germany

Hospital Universitari de Santa Maria

🇪🇸

Lleida, Spain

Spitalzentrum Biel

🇨🇭

Biel/Bienne, Switzerland

Kantonsspital St. Gallen

🇨🇭

St. Gallen, Switzerland

NeuroClin Limited

🇬🇧

Motherwell, Strathclyde, United Kingdom

Re Cognition Health Bristol

🇬🇧

Bristol, United Kingdom

Dent Neurologic Institute

🇺🇸

Amherst, New York, United States

Rocky Mountain Movement Disorders Center, PC

🇺🇸

Englewood, Colorado, United States

Duke University

🇺🇸

Durham, North Carolina, United States

Boston Center for Memory

🇺🇸

Newton, Massachusetts, United States

Center for Neurosciences

🇺🇸

Tucson, Arizona, United States

PNS Clinical Research, LLC dba

🇺🇸

Orange, California, United States

Stanford Hospital and Clinics

🇺🇸

Palo Alto, California, United States

New York University Medical Center PRIME

🇺🇸

New York, New York, United States

South Shore Neurologic Associates, P.C.

🇺🇸

Patchogue, New York, United States

Butler Hospital

🇺🇸

Providence, Rhode Island, United States

AMC Research, LLC

🇺🇸

Matthews, North Carolina, United States

NeuroScience Research Center, LLC.

🇺🇸

Canton, Ohio, United States

Kingfisher Cooperative, LLC

🇺🇸

Spokane, Washington, United States

University of Cincinnati Physicians Group, LLC

🇺🇸

Cincinnati, Ohio, United States

University of Texas Health Science Center at San Antonio

🇺🇸

San Antonio, Texas, United States

EvergreenHealth

🇺🇸

Kirkland, Washington, United States

Royal Prince Alfred Hospital

🇦🇺

Camperdown, New South Wales, Australia

St Vincent's Hospital Sydney

🇦🇺

Darlinghurst, New South Wales, Australia

Liverpool Hospital

🇦🇺

Liverpool, New South Wales, Australia

Southern Neurology

🇦🇺

Kogarah, New South Wales, Australia

Mater Hospital Brisbane

🇦🇺

South Brisbane, Queensland, Australia

UZ Brussel

🇧🇪

Brussel, Belgium

Toronto Memory Program (Neurology Research Inc.)

🇨🇦

Toronto, Ontario, Canada

Fakultni nemocnice Hradec Kralove

🇨🇿

Hradec Kralove, Czechia

Fakultni nemocnice u sv. Anny v Brne

🇨🇿

Brno, Czechia

Fakultni nemocnice Ostrava

🇨🇿

Ostrava, Czechia

Fakultni nemocnice v Motole

🇨🇿

Praha 5, Czechia

Vestra Clinics s.r.o.

🇨🇿

Rychnov nad Kneznou, Czechia

Rigshospitalet

🇩🇰

Copenhagen, Denmark

Itä-Suomen yliopisto, Kuopion kampus

🇫🇮

Kuopio, Finland

CHU Strasbourg - Hôpital Hautepierre

🇫🇷

Strasbourg Cedex, Bas Rhin, France

CHU Nantes - Hopital Nord Laënnec

🇫🇷

Saint-Herblain, Loire Atlantique, France

Hopital Purpan

🇫🇷

Toulouse Cedex 9, Haute Garonne, France

Hopital Gui de Chauliac

🇫🇷

Montpellier, Herault, France

Hôpital La Grave

🇫🇷

Toulouse Cedex 9, Haute Garonne, France

Hôpital Lariboisière

🇫🇷

Paris cedex 10, Paris, France

Universitaetsmedizin Mannheim

🇩🇪

Mannheim, Baden Wuerttemberg, Germany

Universitaetsklinikum Tuebingen

🇩🇪

Tuebingen, Baden Wuerttemberg, Germany

Groupe Hospitalier Pitie-Salpetriere

🇫🇷

Paris, France

CHU de Rouen - Hôpital Charles Nicolle

🇫🇷

Rouen Cedex, Seine Maritime, France

Klinikum Bayreuth GmbH- Hohe Warte

🇩🇪

Bayreuth, Bayern, Germany

Klinikum der Universität München

🇩🇪

München, Bayern, Germany

Neuro Centrum Science GmbH

🇩🇪

Erbach, Hessen, Germany

Deutsches Zentrum fuer Neurodegenerative Erkrankungen (DZNE)

🇩🇪

Bonn, Nordrhein Westfalen, Germany

Klinikum Altenburger Land GmbH

🇩🇪

Altenburg, Thueringen, Germany

Universitaetsklinikum Koeln

🇩🇪

Koeln, Nordrhein Westfalen, Germany

Charité - Campus Charité Mitte

🇩🇪

Berlin, Germany

Charité - Universitätsmedizin Berlin

🇩🇪

Berlin, Germany

Katholisches Klinikum Bochum gGmbH

🇩🇪

Bochum, Germany

Fondazione Istituto G.Giglio di Cefalù

🇮🇹

Cefalù, Palermo, Italy

Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (Presidio Spedali Civili)

🇮🇹

Brescia, Italy

Ospedale San Raffaele

🇮🇹

Milano, Italy

Azienda Ospedaliera e Universitaria di Perugia

🇮🇹

Perugia, Italy

Fondazione IRCCS Istituto Neurologico Carlo Besta

🇮🇹

Milano, Italy

Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza

🇮🇹

Roma, Italy

Himeji Central Hospital Clinic

🇯🇵

Himeji-shi, Hyogo-Ken, Japan

Podlaskie Centrum Psychogeriatrii

🇵🇱

Bialystok, Poland

Samodzielny Publiczny Szpital Kliniczny Nr 4 w Lublinie Kliniczny Oddział Neurologii Oddział Udarowy

🇵🇱

Lublin, Poland

Centrum Medyczne Senior

🇵🇱

Sopot, Poland

Clinica Universidad de Navarra

🇪🇸

Pamplona, Navarra, Spain

Hospital de la Santa Creu i Sant Pau

🇪🇸

Barcelona, Spain

Fundacio ACE

🇪🇸

Barcelona, Spain

CAE Oroitu

🇪🇸

Getxo, Vizcaya, Spain

Hospital Clinic de Barcelona

🇪🇸

Barcelona, Spain

Hospital Universitario Reina Sofia

🇪🇸

Cordoba, Spain

Hospital Victoria Eugenia

🇪🇸

Sevilla, Spain

Sahlgrenska Universitetssjukhuset, Mölndal Sjukhus

🇸🇪

Mölndal, Sweden

Hospital Universitario Dr. Peset

🇪🇸

Valencia, Spain

Hospital Universitari i Politecnic La Fe

🇪🇸

Valencia, Spain

Ospedale Regionale di Lugano

🇨🇭

Lugano, Ticino, Switzerland

Karolinska universitetssjukhuset - Huddinge

🇸🇪

Stockholm, Sweden

Re:Cognition Health Ltd (London)

🇬🇧

London, Greater London, United Kingdom

Institute of Psychiatry, Psychology and Neuroscience

🇬🇧

London, Greater London, United Kingdom

The National Hospital for Neurology and Neurosurgery Centre

🇬🇧

London, Greater London, United Kingdom

Warneford Hospital

🇬🇧

Oxford, Oxfordshire, United Kingdom

Royal Hallamshire Hospital

🇬🇧

Sheffield, South Yorkshire, United Kingdom

Re:Cognition Health - Birmingham

🇬🇧

Birmingham, West Midlands, United Kingdom

Xenoscience Inc.

🇺🇸

Phoenix, Arizona, United States

Sutter Institute for Medical Research

🇺🇸

Sacramento, California, United States

K2 Medical Research, LLC

🇺🇸

Orlando, Florida, United States

Advent Health

🇺🇸

Orlando, Florida, United States

UZ Leuven

🇧🇪

Leuven, Belgium

Hopital Roger Salengro - CHU Lille

🇫🇷

Lille Cedex, Nord, France

Cliniques Universitaires Saint-Luc

🇧🇪

Bruxelles, Belgium

Ehime University Hospital

🇯🇵

Toon-shi, Ehime-Ken, Japan

Osaka Metropolitan University Hospital

🇯🇵

Osaka-shi, Osaka-Fu, Japan

Osaka University Hospital

🇯🇵

Suita-shi, Osaka-Fu, Japan

PROMENTE Sp. z o.o.

🇵🇱

Bydgoszcz, Poland

Ospedale di Arzignano

🇮🇹

Arzignano VI, Vicenza, Italy

Yokohama City Minato Red Cross Hospital

🇯🇵

Yokohama-shi, Kanagawa-Ken, Japan

Brain Research Center Amsterdam

🇳🇱

Amsterdam, Netherlands

SPZOZ Centralny Szpital Kliniczny UM w Lodzi

🇵🇱

Lodz, Poland

Banner Sun Health Research Institute

🇺🇸

Sun City, Arizona, United States

Nzoz Novo-Med

🇵🇱

Katowice, Poland

Clinique de la Memoire de l'Outaouais

🇨🇦

Gatineau, Quebec, Canada

Montreal Neurological Institute Clinical Research Unit

🇨🇦

Montréal, Quebec, Canada

CRST, Clinical Research Services Turku

🇫🇮

Turku, Finland

Nippon Medical School Musashi Kosugi Hospital

🇯🇵

Kawasaki-shi, Kanagawa-Ken, Japan

SPZOZ Szpital Uniwersytecki w Krakowie

🇵🇱

Krakow, Poland

Southampton General Hospital

🇬🇧

Southampton, Hampshire, United Kingdom

UBC Hospital

🇨🇦

Vancouver, British Columbia, Canada

Care Clinic Centrum Medyczne

🇵🇱

Katowice, Poland

NeuroProtect Sp. z o.o.

🇵🇱

Warszawa, Poland

Mazowiecki Szpital Wojewódzki w Warszawie Sp z oo

🇵🇱

Warszawa, Poland

Hôpitaux Universitaires de Genève - HUG- Centre de la mémoire, Bâtiment A1 - Morier

🇨🇭

Geneve, Switzerland

Hawaii Pacific Neuroscience

🇺🇸

Honolulu, Hawaii, United States

Charing Cross Hospital

🇬🇧

London, Greater London, United Kingdom

Greater Manchester Mental Health NHS Foundation Trust

🇬🇧

Manchester, Greater Manchester, United Kingdom

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