A Study to Learn About the Safety of BIIB080 and Whether it 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 (CELIA)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Biogen Idec Research Limited

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

28 Sep 2023 → ongoing

Decision date (initial)

2023-08-17

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Biogen Idec Research Limited

External identifiers

EU CT number

2022-501644-15-01

ClinicalTrials.gov

NCT05399888

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Dose response, Therapy

Objectives and Endpoints for Placebo-Controlled Period:

To characterize the dose-response in change from Baseline to Week 76 using Clinical Dementia Rating-Sum of Boxes (CDR-SB).

Secondary objectives 3

1. Test superiority of at least one dose arm of BIIB080 versus placebo in change from Baseline to Week 76 using CDR-SB
2. Evaluate efficacy of BIIB080 versus placebo in change from Baseline to Week 76
3. Evaluate safety and tolerability of BIIB080 in participants with mild cognitive impairment (MCI) due to Alzheimer's disease (AD) or with mild AD dementia

Conditions and MedDRA coding

Mild Cognitive Impairment due to Alzheimer's Disease Alzheimer's Disease Dementia

Regulatory references

Plan to share IPD

Yes

IPD plan description

In accordance with Biogen’s Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/ Supporting Information: Time Frame: Access Criteria: URL: https://vivli.org/

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Key Inclusion Criteria for Placebo-controlled Period: Must meet all the clinical 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 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 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 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.
2. Evidence of amyloid pathology as measured by positive emission tomography (PET) or cerebrospinal fluid (CSF) sampling
3. Note: Other protocol defined Inclusion/Exclusion criteria may apply

Exclusion criteria 15

1. Known allergy to BIIB080 or a history of hypersensitivity to any of the inactive ingredients in the drug product
2. Previous participation in this study or previous studies with BIIB080
3. 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 Study Day 1
4. Use of any commercially available disease-modifying AD medications such as anti-amyloid monoclonal antibodies.
5. Prior participation in any active or passive immunotherapy study targeting Aβ, unless documentation of receipt of placebo is available
6. 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
7. Prior participation in any study involving an investigational treatment targeting tau that is not an immunotherapy, unless documentation of receipt of placebo is available
8. 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
9. Prior participation in a study of any other agent(s) [including gene therapy] not included in exclusion criteria 4, 5, and 6 with a purported disease‑modifying effect in AD, unless documentation of receipt of placebo is available
10. Current use or previous use of medications with a purported disease‑modifying effect in AD, outside of investigational studies
11. 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
12. 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
13. Current enrolment or a plan to enrol 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
14. Note: Other protocol defined Inclusion/Exclusion criteria may apply
15. 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 enrolment in and completion of the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Dose-response in change from Baseline to Week 76 on the CDR-SB

Secondary endpoints 3

1. Change from Baseline to Week 76 on the CDR-SB
2. Change from Baseline to Week 76 on the following: • ADCS-ADL-MCI • ADAS-Cog 13 • MMSE • Modified iADRS • ADCOMS
3. Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 1

Auxiliary 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Biogen Idec Research Limited

Sponsor organisation

Biogen Idec Research Limited

Address

Building 5 Foundation Park, Roxborough Way Roxborough Way

City

Maidenhead

Postcode

SL6 3UD

Country

United Kingdom

Scientific contact point

Organisation

Biogen Idec Research Limited

Contact name

Clinical trial information desk

Public contact point

Organisation

Biogen Idec Research Limited

Contact name

Clinical trial information desk

Third parties 13

Locations

11 EU/EEA countries · 62 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 259 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

26 submissions — initial application plus substantial / non-substantial modifications