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NP001, Alzheimer's Disease, and Blood Markers of Inflammation

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03179501
Recruitment Status : Terminated (Poor recruitment)
First Posted : June 7, 2017
Last Update Posted : October 22, 2018
Neuraltus Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Beau Nakamoto, University of Hawaii

Brief Summary:
This is a Phase 1 placebo-controlled biomarker study of NP001 in individuals with Alzheimer's Disease.

Condition or disease Intervention/treatment Phase
Alzheimer Disease Drug: NP001 Drug: Placebo Phase 1

Detailed Description:

Abnormal inflammatory monocytes/macrophages, systemically and locally in the central nervous system (CNS), are implicated in the neuro-inflammatory process seen in Alzheimer's disease. NP001 is a novel immune regulator of inflammatory monocytes/macrophages.

Given the key role inflammatory monocytes/macrophages may play in the pathogenesis of AD, this study will assess the changes in inflammatory monocyte-associated biomarkers, including CD16 and HLA-DR, pre- and post- NP001.

This is a Phase 1 single-site, randomized, double-blind, placebo-controlled pilot biomarker study of a single dose of NP001 in individuals with mild-to-moderate Alzheimer's disease. Fourteen individuals will be enrolled and randomized 1:1 to NP001 and placebo. Drug or placebo will be given intravenously. Biomarkers will be measured at baseline and 1 and 7 days following infusion.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Single-site, randomized, double-blind, placebo-controlled
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Double-blind
Primary Purpose: Basic Science
Official Title: Effect of Single Dose NP001 on Blood Markers of Inflammation in Individuals With Mild-to-Moderate Alzheimer's Disease
Actual Study Start Date : September 1, 2017
Actual Primary Completion Date : July 31, 2018
Actual Study Completion Date : July 31, 2018

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: NP001
Drug: NP001

Placebo Comparator: Placebo
Normal saline
Drug: Placebo
Normal saline

Primary Outcome Measures :
  1. Inflammatory monocyte-associated biomarkers [ Time Frame: 7 days ]
    The primary endpoint is changes from baseline at 1 and 7 days following dosing in percent monocyte expression levels of CD16 and HLA-DR.

Secondary Outcome Measures :
  1. Adverse Events [ Time Frame: 7 days ]
    The secondary endpoint is reported and observed adverse events following dosing and at 1 and 7 days post-infusion.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   55 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Male or female, 55 years of age or older,
  2. Diagnosis of probable Alzheimer's disease using the National Institute of Neurological and Communicative Disorders and Stroke and the Alzheimer's disease and Related Disorders Association criteria by Principal Investigator,
  3. Score 14 to 24 (inclusive) on the Mini-Mental Status Examination,
  4. Global Clinical Dementia Rating (CDR) Scale ≥ 0.5 or greater with CDR memory ≥ 0.5 or greater,
  5. Score ≤ 4 or lower on the Hachinski Ischemic Scale,
  6. Score ≤ 5 on the Geriatric Depression Scale (GDS),
  7. Current (stable dose for 4 weeks or longer) or past treatment with acetylcholinesterase inhibitors, memantine, or cognitive enhancers are allowed,
  8. Females must not be of childbearing potential (i.e., must be post-menopausal with cessation of menses for ≥ 12 months or have been surgically sterilized which includes hysterectomy, bilateral oophorectomy, bilateral salpingectomy, or tubal ligation),
  9. Males must agree not to engage in sexual relations with a woman of childbearing potential without effective means of birth control during the study and for 30 days after study drug administration. Must also agree to refrain from sperm donation from receipt of study drug and for 90 days thereafter.
  10. Be capable of providing written informed consent using a form that has been approved by the IRB.
  11. Have veins suitable for intravenous administration of study drug or alternatively, have a venous access device.

Exclusion Criteria:

  1. Diagnosis of another neurologic disorder which can mimic Alzheimer's disease including dementia with Lewy Bodies, frontotemporal dementia and normal pressure hydrocephalus,
  2. Diagnosis of other neurologic disorders which can also impair cognition including stroke, MS, seizures, CNS tumors,
  3. Uncontrolled major psychiatric disorder,
  4. History of unstable medical illness in the 3 months prior to screening including emergent hospitalizations,
  5. Diagnosis of any of the following disorders: systemic sclerosis/scleroderma, inflammatory bowel disease, systemic lupus erythematosus (SLE), rheumatoid arthritis, mixed connective tissue disease, polymyalgia rheumatica, giant cell arteritis, polymyositis, dermatomyositis, and psoriasis,
  6. Active pulmonary disease under treatment including uncontrolled asthma, chronic obstructive pulmonary disease, pulmonary fibrosis, pulmonary infection in the last 3 months, or history of aspiration,
  7. History of unexplained jaundice by subject report,
  8. History of Hepatitis A, B, or C or HIV by subject report,
  9. History of stem cell therapy,
  10. History of immune modulator therapy (e.g., corticosteroids, IV immunoglobulin, immunosuppressive chemotherapeutic agents, plasma exchange, GM-CSF, MCSF, interferons, infliximab, natalizumab, fingolimod [GILENYA], masitinib, ibudilast, tofacitinib citrate [XELJANZ], or any other approved drugs intended to affect the immune system) within 12 weeks of Screening Visit. Locally-acting corticosteroids (inhaled, intranasal, and topical) are permitted,
  11. Participation in an experimental drug trial (of agents other than immune modulators) within 12 weeks prior to Screening Visit. Observational trials with no intervention are acceptable provided permission from the other study sponsor is obtained in writing,
  12. Systolic blood pressure < 100 mm Hg or > 160 mm Hg, diastolic blood pressure > 98 mm Hg. Patients on stable treatment for at least 3 months for hypertension are allowed as long as they meet entry criteria,
  13. Hematocrit < 33%, platelet count < lower limit of normal, or neutrophil count < 1,500/mm3,
  14. Estimated creatinine clearance (eCCr) < 50 mL/minute by Cockcroft-Gault Formula,
  15. Elevated aspartate aminotransferase (AST) or alanine aminotransferance (ALT) greater than 3 times the upper limit of normal,
  16. Pregnant or lactating females,
  17. Have any condition which, in the opinion of the investigator, would put the subject at risk by participating in this study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03179501

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United States, Hawaii
University of Hawaii Clinics at Kakaako
Honolulu, Hawaii, United States, 96813
Sponsors and Collaborators
Beau Nakamoto
Neuraltus Pharmaceuticals, Inc.
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Principal Investigator: Beau Nakamoto, MD PhD University of Hawaii
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Responsible Party: Beau Nakamoto, Associate Professor, Department of Medicine, John A. Burns School of Medicine, University of Hawaii at Manoa, University of Hawaii Identifier: NCT03179501    
Other Study ID Numbers: H039
First Posted: June 7, 2017    Key Record Dates
Last Update Posted: October 22, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Individual participant data will not be made available to other researchers.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Alzheimer Disease
Pathologic Processes
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurodegenerative Diseases
Neurocognitive Disorders
Mental Disorders