Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation

NCT ID: NCT02794857

Last Updated: 2018-05-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

138 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-08-29

Study Completion Date

2017-12-12

Brief Summary

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This study evaluates NP001 in patients with amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation. Half of participants will receive NP001 and the other half will receive placebo.

Detailed Description

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This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS and evidence of elevated systemic inflammation. Subjects will be allocated (1:1) to NP001 and placebo. Drug or placebo will be given intravenously.

Conditions

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Amyotrophic Lateral Sclerosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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NP001

NP001 2 mg/kg by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6

Group Type EXPERIMENTAL

NP001

Intervention Type DRUG

Placebo

Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Interventions

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NP001

Intervention Type DRUG

Placebo

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of clinically possible, clinically probable (with or without laboratory support), or clinically definite ALS (using the revised El Escorial Criteria)
* Forced vital capacity greater than or equal to 65% of that predicted for age and height
* Onset of ALS-related weakness less than 3 years prior to first dose of study drug
* Plasma high sensitivity C-reactive protein (hs-CRP) concentration of greater than or equal to 0.113 mg/dL at pre-screening/screening
* Stable dose (greater than 30 days) of riluzole if undergoing treatment with this agent
* For females: Not be of childbearing potential or agree to use adequate birth control during the study

Exclusion Criteria

* Life expectancy of less than 6 months
* Tracheotomy or be using ventilatory assistance, including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)
* Active pulmonary disease
* Gastrostomy
* Stem cell therapy
* Immune modulator therapy or participation in studies of other agents within 12 weeks of pre-screening/screening
* Unstable medical condition other than ALS
Minimum Eligible Age

21 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Neuraltus Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Gil Block, MD, PhD

Role: STUDY_DIRECTOR

Neuraltus Pharmaceuticals, Inc.

Robert G. Miller, MD

Role: PRINCIPAL_INVESTIGATOR

California Pacific Medical Center

Jonathan Katz, MD

Role: PRINCIPAL_INVESTIGATOR

California Pacific Medical Center

Locations

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St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics

Phoenix, Arizona, United States

Site Status

Mayo Clinic Arizona

Scottsdale, Arizona, United States

Site Status

Cedars-Sinai Medical Center

Los Angeles, California, United States

Site Status

University of California, Irvine, Department of Neurology

Orange, California, United States

Site Status

Forbes Norris MDA/ALS Research Center, CPMC

San Francisco, California, United States

Site Status

Mayo Clinic Florida

Jacksonville, Florida, United States

Site Status

University of Miami Miller School of Medicine

Miami, Florida, United States

Site Status

Emory University, Department of Neurology

Atlanta, Georgia, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

University of Kentucky, Albert B. Chandler Medical Center

Lexington, Kentucky, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Clinical & Translational Science Institute, University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

Carolinas Medical Center, Neurosciences Instutite-Neurology

Charlotte, North Carolina, United States

Site Status

Duke Neurological Disorders Clinic at Morreene Road

Durham, North Carolina, United States

Site Status

Cleveland Clinic Foundation-Cleveland Clinic Hospital

Cleveland, Ohio, United States

Site Status

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

Site Status

Providence Brain & Spine Institute, ALS Center

Portland, Oregon, United States

Site Status

Houston Methodist Neurological Institute

Houston, Texas, United States

Site Status

University of Texas Health Sciences Center San Antonio

San Antonio, Texas, United States

Site Status

Montreal Neurological Institute

Montreal, Quebec, Canada

Site Status

Countries

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United States Canada

References

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Forrest BD, Goyal NA, Fleming TR, Bracci PM, Brett NR, Khan Z, Robinson M, Azhir A, McGrath M. The Effectiveness of NP001 on the Long-Term Survival of Patients with Amyotrophic Lateral Sclerosis. Biomedicines. 2024 Oct 16;12(10):2367. doi: 10.3390/biomedicines12102367.

Reference Type DERIVED
PMID: 39457678 (View on PubMed)

Other Identifiers

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NP001-10-003

Identifier Type: -

Identifier Source: org_study_id

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