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Alzheimer DiseasePhase 1NCT03179501

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

A Phase 1 study testing NP001 in people with Alzheimer Disease, run by Beau Nakamoto at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This is a Phase 1 placebo-controlled biomarker study of NP001 in individuals with Alzheimer's Disease. Read more about this study

Who is it for?
Ages 55 years and older
Conditions the study is for
Alzheimer Disease
What is being tested?
NP001
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
4 participants
Where
1 siteUnited States
Study dates
1 Sep 2017to 31 Jul 2018
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 55 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or female, 55 years of age or older,
  • 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,
  • Score 14 to 24 (inclusive) on the Mini-Mental Status Examination,
  • Global Clinical Dementia Rating (CDR) Scale ≥ 0.5 or greater with CDR memory ≥ 0.5 or greater,
  • Score ≤ 4 or lower on the Hachinski Ischemic Scale,
  • Score ≤ 5 on the Geriatric Depression Scale (GDS),
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  • Current (stable dose for 4 weeks or longer) or past treatment with acetylcholinesterase inhibitors, memantine, or cognitive enhancers are allowed,
  • 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),
  • 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.
  • Be capable of providing written informed consent using a form that has been approved by the IRB.
  • Have veins suitable for intravenous administration of study drug or alternatively, have a venous access device.

Who cannot join

  • Diagnosis of another neurologic disorder which can mimic Alzheimer's disease including dementia with Lewy Bodies, frontotemporal dementia and normal pressure hydrocephalus,
  • Diagnosis of other neurologic disorders which can also impair cognition including stroke, MS, seizures, CNS tumors,
  • Uncontrolled major psychiatric disorder,
  • History of unstable medical illness in the 3 months prior to screening including emergent hospitalizations,
  • 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,
  • 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,
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  • History of unexplained jaundice by subject report,
  • History of Hepatitis A, B, or C or HIV by subject report,
  • History of stem cell therapy,
  • 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,
  • 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,
  • 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,
  • Hematocrit < 33%, platelet count < lower limit of normal, or neutrophil count < 1,500/mm3,
  • Estimated creatinine clearance (eCCr) < 50 mL/minute by Cockcroft-Gault Formula,
  • Elevated aspartate aminotransferase (AST) or alanine aminotransferance (ALT) greater than 3 times the upper limit of normal,
  • Pregnant or lactating females,
  • Have any condition which, in the opinion of the investigator, would put the subject at risk by participating in this study.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • University of Hawaii Clinics at Kakaako

    Honolulu, Hawaii, 96813

Other trials in United States

About this study

Official title: Effect of Single Dose NP001 on Blood Markers of Inflammation in Individuals With Mild-to-Moderate Alzheimer's Disease

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

Read the 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.

Being tested
NP001, Placebo
Medical subject headings
Alzheimer Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NP001Drug

    NP001

  • PlaceboDrug

    Normal saline

Study groups in NCT03179501
Study groupRoleWhat they receive
NP001ExperimentalNP001
Receives: Drug: NP001
PlaceboPlacebo comparatorNormal saline
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Inflammatory monocyte-associated biomarkers

    The primary endpoint is changes from baseline at 1 and 7 days following dosing in percent monocyte expression levels of CD16 and HLA-DR.

    Measured: 7 days

Secondary outcomes (1)

  1. Adverse Events

    The secondary endpoint is reported and observed adverse events following dosing and at 1 and 7 days post-infusion.

    Measured: 7 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Jun 2017
  2. Study start1 Sep 2017
  3. Primary completion31 Jul 2018
  4. Study completion31 Jul 2018

Why it stopped: Poor recruitment

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Basic science
Allocation
Randomised
Intervention model
Parallel groups
Model description
Single-site, randomized, double-blind, placebo-controlled
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
4 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03179501
Sponsor's protocol ID
H039
Status last verified
May 2018
First submitted
5 Jun 2017
Last updated
22 Oct 2018
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: Individual participant data will not be made available to other researchers.

Publications

  • Miller RG, Block G, Katz JS, Barohn RJ, Gopalakrishnan V, Cudkowicz M, Zhang JR, McGrath MS, Ludington E, Appel SH, Azhir A; Phase 2 Trial NP001 Investigators. Randomized phase 2 trial of NP001-a novel immune regulator: Safety and early efficacy in ALS. Neurol Neuroimmunol Neuroinflamm. 2015 Apr 9;2(3):e100. doi: 10.1212/NXI.0000000000000100. eCollection 2015 Jun.

    PubMed 25884010

  • Miller RG, Zhang R, Block G, Katz J, Barohn R, Kasarskis E, Forshew D, Gopalakrishnan V, McGrath MS. NP001 regulation of macrophage activation markers in ALS: a phase I clinical and biomarker study. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Dec;15(7-8):601-9. doi: 10.3109/21678421.2014.951940. Epub 2014 Sep 5.

    PubMed 25192333

  • Zhang R, Miller RG, Madison C, Jin X, Honrada R, Harris W, Katz J, Forshew DA, McGrath MS. Systemic immune system alterations in early stages of Alzheimer's disease. J Neuroimmunol. 2013 Mar 15;256(1-2):38-42. doi: 10.1016/j.jneuroim.2013.01.002. Epub 2013 Feb 4.

    PubMed 23380586

  • Pey P, Pearce RK, Kalaitzakis ME, Griffin WS, Gentleman SM. Phenotypic profile of alternative activation marker CD163 is different in Alzheimer's and Parkinson's disease. Acta Neuropathol Commun. 2014 Feb 14;2:21. doi: 10.1186/2051-5960-2-21.

    PubMed 24528486

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing NP001 in people with Alzheimer Disease, run by Beau Nakamoto at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 22 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Poor recruitment). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Inflammatory monocyte-associated biomarkers (time frame: 7 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03179501, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Oct 2018.

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