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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05399888




Registration number
NCT05399888
Ethics application status
Date submitted
27/05/2022
Date registered
1/06/2022
Date last updated
20/11/2024

Titles & IDs
Public title
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
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, and Tolerability of BIIB080 in Subjects With Mild Cognitive Impairment Due to Alzheimer's Disease or Mild Alzheimer's Disease Dementia
Secondary ID [1] 0 0
2022-501644-15
Secondary ID [2] 0 0
247AD201
Universal Trial Number (UTN)
Trial acronym
CELIA
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mild Cognitive Impairment Due to Alzheimer's Disease 0 0
Alzheimer's Disease Dementia 0 0
Condition category
Condition code
Neurological 0 0 0 0
Alzheimer's disease
Neurological 0 0 0 0
Dementias
Mental Health 0 0 0 0
Other mental health disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - BIIB080
Treatment: Drugs - BIIB080-matching placebo

Placebo comparator: Placebo Q12W - Participants 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.

Experimental: BIIB080 Low Dose Q24W - Participants 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.

Experimental: BIIB080 High Dose Q24W - Participants 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.

Experimental: BIIB080 High Dose Q12W - Participants 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.


Treatment: Drugs: BIIB080
Administered as specified in the treatment arm.

Treatment: Drugs: BIIB080-matching placebo
Administered as specified in the treatment arm.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Dose response in Change From Baseline to Week 76 on the CDR-SB
Timepoint [1] 0 0
Baseline to Week 76
Secondary outcome [1] 0 0
Change From Baseline to Week 76 on the CDR-SB
Timepoint [1] 0 0
Baseline to Week 76
Secondary outcome [2] 0 0
Change From Baseline to Week 76 on the Alzheimer's Disease Cooperative Study Activities of Daily Living for Mild Cognitive Impairment (ADCS-ADL-MCI)
Timepoint [2] 0 0
Baseline to Week 76
Secondary outcome [3] 0 0
Change From Baseline to Week 76 on the Alzheimer's Disease Assessment Scale Cognitive Subscale (ADAS-Cog 13)
Timepoint [3] 0 0
Baseline to Week 76
Secondary outcome [4] 0 0
Change From Baseline to Week 76 on the Mini Mental State Examination (MMSE)
Timepoint [4] 0 0
Baseline to Week 76
Secondary outcome [5] 0 0
Change From Baseline to Week 76 on the Modified Integrated Alzheimer's Disease Rating Scale (iADRS)
Timepoint [5] 0 0
Baseline to Week 76
Secondary outcome [6] 0 0
Change From Baseline to Week 76 on the Alzheimer's Disease Composite Score (ADCOMS)
Timepoint [6] 0 0
Baseline to Week 76
Secondary outcome [7] 0 0
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timepoint [7] 0 0
From first dose of study drug up to end of study of placebo-controlled period (up to Week 96)

Eligibility
Key inclusion criteria
Key Inclusion Criteria for Placebo-controlled Period:

* Must meet all the clinical staging criteria for MCI due to AD (Stage 3) or mild AD dementia (Stage 4) according to the National Institute on Aging at National Institutes of Health and the Alzheimer's Association (NIA-AA) and must have the following at Screening Visit 1:

1. Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Delayed Memory Index score of =85, indicative of objective evidence of memory impairment.
2. CDR global score of 0.5 for MCI due to AD or 0.5 or 1 for mild AD dementia
3. MMSE score of 21 to 30 (inclusive).
4. CDR Memory Box score of =0.5.
* Evidence of amyloid pathology as measured by positive emission tomography (PET) or cerebrospinal fluid (CSF) sampling.
* Must have 1 care partner who, in the Investigator's judgment, has frequent and sufficient contact with the participant (at least 10 hours/week) to be able to provide accurate information about the participant's cognitive and functional abilities.

Key Inclusion Criteria for LTE Period

* Ability of the participant and/or his/her legally authorized representative (e.g., parent, spouse, or legal guardian), where local regulations and institutional practices permit, as appropriate and applicable, to understand the purpose and risks of the study, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations. Incapacitated individuals will not be enrolled in the EU (European Union) and other countries where local laws, regulations, and practices do not permit their inclusion.
* Participants must have completed the placebo-controlled period of the study, including the Week 76 visit.
* Participants must have taken at least 5 doses of BIIB080 or placebo during the placebo-controlled period.
* Medically able to undergo the study procedures (including LP [lumbar puncture]) and to adhere to the visit schedule at the time of study entry into the LTE period, as determined by the Investigator.
* Apart from a clinical diagnosis of AD, the participant must be in good health as determined by the Investigator, based on medical history.
* Must have 1 care partner who, in the Investigator's judgment, has frequent and sufficient contact with the participant (at least 10 hours/week) to be able to provide accurate information about the participant's cognitive and functional abilities.

Key
Minimum age
50 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria for Placebo-controlled Period:

* Known allergy to BIIB080 or a history of hypersensitivity to any of the inactive ingredients in the drug product.
* Previous participation in this study or previous studies with BIIB080.
* Use of non-disease-modifying AD medications (including but not limited to donepezil, rivastigmine, galantamine, tacrine, and memantine) at doses that have not been stable for at least 8 weeks prior to Screening Visit 1 and during the screening period up to Day 1.
* Use of any commercially available disease-modifying AD medications such as anti-amyloid monoclonal antibodies.
* Prior participation in any active or passive immunotherapy study targeting Aß, unless documentation of receipt of placebo is available.
* Prior participation in any passive immunotherapy study targeting tau, unless the last administration occurred 6 months or 5 half-lives, whichever is sooner, prior to Screening or documentation of receipt of placebo is available.
* Prior participation in any study involving an investigational treatment targeting tau that is not a passive immunotherapy, unless documentation of receipt of placebo is available.
* Prior participation in a study of any other agent(s) not included in exclusion criteria 5, 6, and 7 with a purported disease-modifying effect in AD within 12 months, unless documentation of receipt of placebo is available.
* Prior participation in a study of any gene therapy with a purported disease-modifying effect in AD, unless documentation of receipt of placebo is available.
* Current use or previous use of medications with a purported disease-modifying effect in AD, outside of investigational studies.
* Any vaccination given within 10 days prior to Day -1. Coronavirus disease 2019 (COVID-19) vaccinations using RNA or deoxyribonucleic acid (DNA) technology are allowed during the study, as well as other types of immunization/vaccination/booster, except during the 10 days before and after clinic visits.
* Contraindications to having a brain magnetic resonance imaging (MRI) [e.g., MRI-incompatible pacemaker; MRI-incompatible aneurysm clips, artificial heart valves, or other metal foreign body; claustrophobia that cannot be medically managed]. If the MRI compatibility of implanted devices is unknown, the participant must be excluded from the study.
* Current enrollment or a plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy for investigational use is administered within 52 weeks prior to the Baseline Visit.

Key Exclusion Criteria for LTE Period

* Any medical or psychiatric contraindication or clinically significant abnormality that, in the opinion of the Investigator, will substantially increase the risk associated with the participant's enrollment in and completion of the study.

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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/08/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
8/01/2029
Actual
Sample size
Target
Accrual to date
Final
416
Recruitment in Australia
Recruitment state(s)
NSW,QLD
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
St Vincent's Hospital Sydney - Darlinghurst
Recruitment hospital [3] 0 0
Southern Neurology - Kogarah
Recruitment hospital [4] 0 0
Liverpool Hospital - Liverpool
Recruitment hospital [5] 0 0
Mater Hospital Brisbane - South Brisbane
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [3] 0 0
2217 - Kogarah
Recruitment postcode(s) [4] 0 0
2170 - Liverpool
Recruitment postcode(s) [5] 0 0
4101 - South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Hawaii
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
North Carolina
Country [9] 0 0
United States of America
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Ohio
Country [10] 0 0
United States of America
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Rhode Island
Country [11] 0 0
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Tennessee
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United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
United States of America
State/province [13] 0 0
Washington
Country [14] 0 0
Belgium
State/province [14] 0 0
Brussel
Country [15] 0 0
Belgium
State/province [15] 0 0
Bruxelles
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Belgium
State/province [16] 0 0
Kortrijk
Country [17] 0 0
Belgium
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Leuven
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Canada
State/province [18] 0 0
British Columbia
Country [19] 0 0
Canada
State/province [19] 0 0
Ontario
Country [20] 0 0
Canada
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Quebec
Country [21] 0 0
Czechia
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Brno
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Czechia
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Hradec Kralove
Country [23] 0 0
Czechia
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Ostrava
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Czechia
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Praha 5
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Czechia
State/province [25] 0 0
Praha 6
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Czechia
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Rychnov nad Kneznou
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Denmark
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Copenhagen
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Denmark
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Ålborg
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Finland
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Kuopio
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Finland
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Turku
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France
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Bas Rhin
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France
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Herault
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Loire Atlantique
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Paris
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France
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Seine Maritime
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Germany
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Baden Wuerttemberg
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Germany
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Bayern
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Germany
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Hessen
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Germany
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Niedersachsen
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Germany
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Nordrhein Westfalen
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Germany
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Thueringen
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Germany
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Berlin
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Germany
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Bochum
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Italy
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Lecce
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Italy
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Palermo
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Italy
State/province [48] 0 0
Vicenza
Country [49] 0 0
Italy
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Brescia
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Italy
State/province [50] 0 0
Milano
Country [51] 0 0
Italy
State/province [51] 0 0
Perugia
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Italy
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Roma
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Japan
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Ehime-Ken
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Hyogo-Ken
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Japan
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Kanagawa-Ken
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Japan
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Osaka-Fu
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Japan
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Tokyo-To
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Netherlands
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Amsterdam
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Poland
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Bialystok
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Bydgoszcz
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Katowice
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Poland
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Krakow
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Poland
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Lodz
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Lublin
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Poland
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Sopot
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Poland
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Warszawa
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Spain
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Navarra
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Spain
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Vizcaya
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Spain
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Barcelona
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Spain
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Cordoba
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Spain
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Lleida
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Spain
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Sevilla
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Spain
State/province [73] 0 0
Valencia
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Sweden
State/province [74] 0 0
Mölndal
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Sweden
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Stockholm
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Switzerland
State/province [76] 0 0
Ticino
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Switzerland
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Biel/Bienne
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Switzerland
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Geneve
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Switzerland
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St. Gallen
Country [80] 0 0
United Kingdom
State/province [80] 0 0
Greater London
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United Kingdom
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Greater Manchester
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United Kingdom
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Hampshire
Country [83] 0 0
United Kingdom
State/province [83] 0 0
Oxfordshire
Country [84] 0 0
United Kingdom
State/province [84] 0 0
South Yorkshire
Country [85] 0 0
United Kingdom
State/province [85] 0 0
Strathclyde
Country [86] 0 0
United Kingdom
State/province [86] 0 0
West Midlands
Country [87] 0 0
United Kingdom
State/province [87] 0 0
Bristol

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Biogen
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT05399888
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Biogen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
US Biogen Clinical Trial Center
Address 0 0
Country 0 0
Phone 0 0
866-633-4636
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05399888