Proof-of-Concept Study of a Selective p38 MAPK Alpha Inhibitor, Neflamapimod, in Subjects With Mild Alzheimer's Disease

NCT ID: NCT03402659

Last Updated: 2021-10-27

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

161 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-12-29

Study Completion Date

2019-07-31

Brief Summary

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This is a phase 2b, double-blind, placebo controlled proof-of-concept study of a an oral small molecule selective inhibitor of p38 alpha kinase, neflamapimod, administered for 24 weeks in subjects with mild Alzheimer's disease. The primary objective is to demonstrate significant improvement relative to placebo-treatment in episodic memory function, as assessed by the Hopkins Verbal Learning Test. Secondary endpoints include Clinical Dementia Rating scale (CDR), Wechsler Memory Scale (WMS), Mini-Mental-Status-Examination (MMSE) and Cerebrospinal fluid (CSF) biomarkers of AD disease activity and progression.

Detailed Description

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Details provided elsewhere.

Conditions

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Alzheimer Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Double-blind, placebo-controlled
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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neflamapimod

40 mg hard gelatin capsules, taken twice daily with food.

Group Type EXPERIMENTAL

neflamapimod

Intervention Type DRUG

40 mg neflamapimod capsule

placebo

hard gelatin capsules containing excipients only, weight- and size-matched; taken twice daily with food.

Group Type PLACEBO_COMPARATOR

placebo

Intervention Type OTHER

matching placebo capsule

Interventions

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neflamapimod

40 mg neflamapimod capsule

Intervention Type DRUG

placebo

matching placebo capsule

Intervention Type OTHER

Other Intervention Names

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VX-745

Eligibility Criteria

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Inclusion Criteria

1. Men and women age 55 to 85 years, inclusive.
2. Willing and able to provide informed consent.
3. Must have mild cognitive impairment (MCI) or mild AD with evidence of progression ("Mild-AD"), as defined by the following:

1. CDR-Global Score of 0.5 or 1.0, with CDR memory subscore of at least 0.5.
2. MMSE score ranging from 20 to 28, inclusive.
3. Positive biomarker for AD, as defined by a CSF Aβ1-42R below the threshold and phospho-tau above the threshold for the assay utilized in the study and assessed by the central laboratory.
4. Computed tomography (CT) or magnetic resonance imaging (MRI) findings within 2 years of Screening that are compatible with AD and no other pathologic processes that might potentially account for the subject's cognitive impairment.
5. If the subject is taking a single drug for AD (e.g., donepezil or other cholinesterase inhibitors or memantine; dual therapy is excluded), he/she has been on a stable dose for at least 2 months prior to baseline, and the dose must remain unchanged during the study unless required for management of adverse events (AEs).
6. Adequate visual and auditory abilities to perform all aspects of the cognitive and functional assessments.
7. Must have reliable informant or caregiver.

Exclusion Criteria

1. Evidence that the primary basis for cognitive impairment is neurodegenerative disease other than AD, including, but not limited to, vascular dementia, dementia with Lewy bodies, and Parkinson's disease.
2. Suicidality, defined as active suicidal thoughts within 6 months before Screening or at Baseline, defined as answering yes to items 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS), or history of suicide attempt in previous 2 years, or, in the Investigator's opinion, at serious risk of suicide.
3. History of major and active psychiatric disorder, moderate to severe depressive symptoms, and or other concurrent medical condition that, EIP-VX17-745-304, Version 1.0, 17 November, 2017 Page 7 of 46 EIP Pharma, LLC Confidential in the opinion of the Investigator, might compromise safety and/or compliance with study requirements.
4. Diagnosis of alcohol or drug abuse within the previous 2 years.
5. History of cancer within the last 5 years, except basal cell carcinoma, squamous skin carcinoma, prostate cancer or carcinoma in situ with no significant progression over the past 2 years.
6. Poorly controlled clinically significant medical illness.
7. History of serum B12 abnormality, anemia with hemoglobin ≤10 g/dL, thyroid function abnormality, electrolyte abnormality, or positive syphilis serology that have not been corrected and/or otherwise addressed.
8. History of epilepsy or unexplained seizure within the past 5 years.
9. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>3 × the upper limit of normal (ULN), total bilirubin \>2 × ULN, and/or International Normalized Ratio (INR) \>1.5
10. Known human immunodeficiency virus, hepatitis B, or active hepatitis C virus infection.
11. Subject participated in a study of an investigational drug less than 3 months or 5 half-lives of the investigation drug, whichever is longer, before enrollment in this study.
Minimum Eligible Age

55 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Worldwide Clinical Trials

OTHER

Sponsor Role collaborator

Amsterdam UMC, location VUmc

OTHER

Sponsor Role collaborator

EIP Pharma Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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John Alam, MD

Role: STUDY_DIRECTOR

EIP Pharma

Locations

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Alliance for Research

Long Beach, California, United States

Site Status

Pacific Research Network

San Diego, California, United States

Site Status

CITrials

Santa Ana, California, United States

Site Status

Southern California Research, LLC

Simi Valley, California, United States

Site Status

Viking Clinical Research

Temecula, California, United States

Site Status

Miami Dade Medical Research Institute

Miami, Florida, United States

Site Status

Sensible Healthcare, LLC

Ocoee, Florida, United States

Site Status

Anchor Neuroscience

Pensacola, Florida, United States

Site Status

Progressive Medical Research

Port Orange, Florida, United States

Site Status

Suncoast Neuroscience Associates, Inc.

St. Petersburg, Florida, United States

Site Status

Florida Premier Research Institute

Winter Park, Florida, United States

Site Status

Northwest Clinical Trials

Boise, Idaho, United States

Site Status

MassGeneral Institute for Neurodegenerative Disease

Charlestown, Massachusetts, United States

Site Status

Manhattan Behavioral Medicine

New York, New York, United States

Site Status

Alzheimer's Memory Center and Research Institute

Charlotte, North Carolina, United States

Site Status

Northwest Clinical Research Center

Seattle, Washington, United States

Site Status

Neuro HK, s.r.o. POLIKLINIKA CHOCEŇ, a.s.

Choceň, , Czechia

Site Status

Cerebrovaskulární poradna s.r.o.

Moravská Ostrava, , Czechia

Site Status

Clintrial S.R.O

Prague, , Czechia

Site Status

Private Psychiatric Centre

Prague, , Czechia

Site Status

Vestra Clinics S.R.O

Rychnov nad Kněžnou, , Czechia

Site Status

CCBR Clinical Research, Aalborg

Aalborg, , Denmark

Site Status

CCBR Clinical Research, Ballerup

Ballerup Municipality, , Denmark

Site Status

CCBR Clinical Research, Vejle

Vejle, , Denmark

Site Status

Jeroen Bosch Ziekenhuis

's-Hertogenbosch, , Netherlands

Site Status

Alzheimer Research Center

Amsterdam, , Netherlands

Site Status

Amphia Ziekhuis

Breda, , Netherlands

Site Status

MAC Clinical Research Tankersley

Barnsley, , United Kingdom

Site Status

Re:Cognition Health Birmingham

Birmingham, , United Kingdom

Site Status

MAC Clinical Research Blackpool

Blackpool, , United Kingdom

Site Status

Fulbourn Hospital

Cambridge, , United Kingdom

Site Status

MAC Clinical Research Leeds

Leeds, , United Kingdom

Site Status

MAC Clinical Research Liverpool

Liverpool, , United Kingdom

Site Status

Re:Cognition Health London

London, , United Kingdom

Site Status

St. Pancras Clinical Research

London, , United Kingdom

Site Status

MAC Clinical Research Manchester

Manchester, , United Kingdom

Site Status

Re:Cognition Health Plymouth

Plymouth, , United Kingdom

Site Status

5 Boroughs/North West Boroughs Healthcare NHS Foundation Trust

Warrington, , United Kingdom

Site Status

Countries

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United States Czechia Denmark Netherlands United Kingdom

References

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Prins ND, Harrison JE, Chu HM, Blackburn K, Alam JJ, Scheltens P; REVERSE-SD Study Investigators. A phase 2 double-blind placebo-controlled 24-week treatment clinical study of the p38 alpha kinase inhibitor neflamapimod in mild Alzheimer's disease. Alzheimers Res Ther. 2021 May 27;13(1):106. doi: 10.1186/s13195-021-00843-2.

Reference Type DERIVED
PMID: 34044875 (View on PubMed)

Tormahlen NM, Martorelli M, Kuhn A, Maier F, Guezguez J, Burnet M, Albrecht W, Laufer SA, Koch P. Design and Synthesis of Highly Selective Brain Penetrant p38alpha Mitogen-Activated Protein Kinase Inhibitors. J Med Chem. 2022 Jan 27;65(2):1225-1242. doi: 10.1021/acs.jmedchem.0c01773. Epub 2021 May 11.

Reference Type DERIVED
PMID: 33974419 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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EIP-VX17-745-304

Identifier Type: -

Identifier Source: org_study_id