BIIB080 for Alzheimer's Disease

(CELIA Trial)

Not currently recruiting at 167 trial locations

Overseen By Stephen Flitman

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Biogen

Must not be taking: Anti-amyloid antibodies

Disqualifiers: Allergy to BIIB080, Unstable AD meds, MRI contraindications, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 18

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether the drug BIIB080 can slow Alzheimer's disease (AD) progression in its early stages. Participants will receive either a low or high dose of BIIB080 or a placebo (a substance with no active drug) through an injection into the fluid around the spinal cord. The goal is to determine which dose best slows the worsening of AD symptoms. The trial seeks individuals with mild cognitive impairment or mild dementia due to AD who have been stable on certain AD medications for at least 8 weeks and have a care partner to provide regular updates on their condition. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to early-stage findings.

Do I need to stop my current medications to join the trial?

You can continue taking certain Alzheimer's medications, but you must be on the same dose for at least 8 weeks before the screening period. However, you cannot use any disease-modifying Alzheimer's medications or participate in other investigational studies during the trial.

Is there any evidence suggesting that BIIB080 is likely to be safe for humans?

Research shows that BIIB080 seems safe for people. An early study found no serious side effects in participants, indicating good safety. However, researchers are still learning about its long-term effects, especially since it targets tau, a protein linked to Alzheimer's disease. This ongoing study aims to better understand the long-term safety of BIIB080. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for Alzheimer's?

Researchers are excited about BIIB080 for Alzheimer's disease because it represents a novel approach to treating this condition. Unlike current standard treatments, which mainly focus on managing symptoms, BIIB080 targets the underlying disease process by reducing tau protein build-up, a key factor in Alzheimer's progression. Additionally, BIIB080 is administered via intrathecal injection, allowing for direct delivery to the central nervous system, which may enhance its effectiveness. This innovative mechanism and delivery method promise a potential breakthrough in slowing or altering disease progression, rather than just alleviating symptoms.

What evidence suggests that BIIB080 might be an effective treatment for Alzheimer's?

Research has shown that BIIB080 yields promising results for Alzheimer's disease. In earlier studies, patients with early-stage Alzheimer's who took BIIB080 had lower levels of tau proteins in their spinal fluid. This is significant because tau proteins are linked to the progression of Alzheimer's. In this trial, participants will receive either a high or low dose of BIIB080 or a placebo. Specifically, those on a high dose of BIIB080 in previous studies demonstrated better thinking and daily functioning compared to those who took a placebo. These findings suggest that BIIB080 might help slow the worsening of Alzheimer's symptoms, although more research is needed to confirm these results. ^{1 6 7 8 9}

Who Is on the Research Team?

Medical Director

Principal Investigator

Biogen

Are You a Good Fit for This Trial?

This trial is for people aged 50-80 with mild cognitive impairment or mild Alzheimer's dementia, who have certain scores on memory and cognition tests, evidence of amyloid in the brain, and stable use of Alzheimer's medications. Excluded are those allergic to BIIB080, previously treated with it or other specific Alzheimer's therapies, unstable medication doses, recent vaccinations around clinic visits, MRI contraindications.

Inclusion Criteria

Your memory test score is less than or equal to 85, showing that you have memory problems.

Your CDR Memory Box score is at least 0.5.

I have a family history or diagnosis of Mild Cognitive Impairment or Alzheimer's.

Exclusion Criteria

I have a history of a bleeding disorder.

Are you currently using cannabis (in any form, by prescription or recreation) ?

I have a bleeding disorder or a past MRI showed I had a hemorrhage.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Placebo-Controlled Period

Participants receive either a low dose or high dose of BIIB080, or a placebo, as an injection into the cerebrospinal fluid every 12 or 24 weeks

76 weeks

Clinic visits every 6 weeks

Long-Term Extension (LTE) Period

Eligible participants continue to receive BIIB080 to assess long-term safety and effects on daily life, thinking, and memory

96 weeks

Injections every 12 or 24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• BIIB080
• BIIB080-matching placebo

Trial Overview Researchers are testing if BIIB080 can slow down Alzheimer's disease progression compared to a placebo. Participants will receive either a low or high dose of BIIB080 injected into their spinal fluid every 12 weeks for over two years while being monitored using the CDR-SB score that assesses dementia symptoms.

How Is the Trial Designed?

Group I: BIIB080 Low Dose Q24W Experimental Treatment 2 Interventions

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.

Group II: BIIB080 High Dose Q24W Experimental Treatment 2 Interventions

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.

Group III: BIIB080 High Dose Q12W Experimental Treatment 1 Intervention

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.

Group IV: Placebo Q12W Placebo Group 2 Interventions

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.

Who Is Running the Clinical Trial?

Biogen

Lead Sponsor

Trials

655

Recruited

468,000+

Daniel Quirk

Biogen

Chief Medical Officer

MD

Christopher A. Viehbacher

Biogen

Chief Executive Officer since 2022

Graduated from Queen's University, Kingston, Ontario, Canada

Citations

1. investors.biogen.com investors.biogen.com/news-releases/news-release-details/new-data-presented-adpdtm-2023-show-biogens-biib080-mapt-aso

New Data Presented at AD/PD™ 2023 Show Biogen's ... BIIB080 reduced soluble tau protein in cerebrospinal fluid (CSF) in a dose-dependent and sustained manner in patients with early-stage Alzheimer's disease (AD).

2. neurologylive.com neurologylive.com/view/biogen-biib080-exhibits-favorable-outcomes-phase-1b-trial-early-ad

Biogen's BIIB080 Exhibits Favorable Outcomes in Phase ... Patients with mild Alzheimer disease who received high doses of BIIB080 demonstrated improvement in cognitive and functional outcomes compared with placebo.

3. investors.biogen.com investors.biogen.com/static-files/75a32d30-01ce-4816-87a6-20735ff25b3c

Exploratory Clinical Outcomes From the BIIB080 Phase 1b ... • These data continue to support further investigation of the clinical efficacy and safety of BIIB080 in patients with MCI due to AD/mild AD ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11712482/

The First Gene Therapy for Patients with Alzheimer's Disease BIIB080, as a tau synthesis reduction drug, demonstrated promising results on tau biomarkers in individuals with moderate AD, along with favorable tolerability.

5. clinicaltrials.gov clinicaltrials.gov/study/NCT05399888

NCT05399888 | A Study to Learn About the Safety of ... 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 ...

6. alzdiscovery.org alzdiscovery.org/uploads/cognitive_vitality_media/BIIB080_(drug_under_development).pdf

BIIB080 – MAPTRX ASO Safety: In the initial in-human study, BIIB080 appeared to be well-tolerated, with no drug- related serious events. Long-term effects of tau knockdown are not ...

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37902726/

Exploratory Tau Biomarker Results From a Multiple ... In this randomized clinical trial, BIIB080 reduced tau biomarkers, including CSF t-tau, CSF p-tau181, and tau PET, which is associated with cognitive decline, ...

8. investors.biogen.com investors.biogen.com/news-releases/news-release-details/biogens-investigational-tau-targeting-therapy-biib080-receives

Biogen's Investigational Tau-Targeting Therapy BIIB080 ... Biogen's Investigational Tau-Targeting Therapy BIIB080 Receives FDA Fast Track Designation for the Treatment of Alzheimer's Disease. Biogen's ...

9. nature.com nature.com/articles/s41591-023-02326-3

Tau-targeting antisense oligonucleotide MAPT Rx in mild ... Tau plays a key role in Alzheimer's disease (AD) pathophysiology, and accumulating evidence suggests that lowering tau may reduce this ...