A Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)

NCT ID: NCT01281631

Last Updated: 2012-11-22

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

136 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-02-28

Study Completion Date

2012-09-30

Brief Summary

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This is a Phase 2 randomized, double-blind, placebo-controlled, multicenter study of NP001 in subjects with ALS.

Detailed Description

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This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS conducted in multiple centers. Drug or placebo will be given intravenously. Approximately 105 subjects will be enrolled. Subjects will be allocated (1:1:1) to placebo and 2 dose levels of NP001.

Conditions

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Amyotrophic Lateral Sclerosis

Keywords

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ALS

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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Low dose NP001

Low drug dose

Group Type EXPERIMENTAL

NP001

Intervention Type DRUG

Low dose of NP001

High dose NP001

High drug dose

Group Type EXPERIMENTAL

NP001

Intervention Type DRUG

High dose of NP001

Placebo

normal saline

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

normal saline

Interventions

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NP001

Low dose of NP001

Intervention Type DRUG

NP001

High dose of NP001

Intervention Type DRUG

Placebo

normal saline

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Onset of symptoms less than 3 years prior to study entry.
* Forced Vital Capacity (FVC) at least 70% of that predicted for age and height.
* Stable dose 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

* Unstable medical condition(s) other than ALS.
* Life expectancy of less than 6 months.
* Require life-sustaining interventions for the 6 months following randomization.
* Have a tracheotomy or be using ventilatory assistance \[including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)\].
* Active pulmonary disease.
* Immune modulator therapy within 12 weeks of study entry or participation in studies of other agents within the last 4 weeks prior to the randomization.
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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Robert G. Miller, MD

Role: PRINCIPAL_INVESTIGATOR

Forbes Norris ALS Treatment and Research, California Pacific Medical Center

Locations

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Mayo Clinic, Scottsdale

Scottsdale, Arizona, United States

Site Status

UC, Irvine

Irvine, California, United States

Site Status

UCLA

Los Angeles, California, United States

Site Status

California Pacific Med Center Forbes Norris MDA/ALS Research and Treatment Center

San Francisco, California, United States

Site Status

Mayo Clinic, Jacksonville

Jacksonville, Florida, United States

Site Status

The Emory Clinic

Atlanta, Georgia, United States

Site Status

University of Kansas Medical Center, Landon Center on Aging

Kansas City, Kansas, United States

Site Status

University of Kentucky, Department of Neurology

Lexington, Kentucky, United States

Site Status

Massachusetts General Hospital

Charlestown, Massachusetts, United States

Site Status

Columbia University

New York, New York, United States

Site Status

SUNY Upstate Medical University, Syracuse

Syracuse, New York, United States

Site Status

Carolinas Medical Center

Charlotte, North Carolina, United States

Site Status

Duke University, Dept of Neurology

Durham, North Carolina, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

Providence ALS Center

Portland, Oregon, United States

Site Status

Methodist Hospital Research Institute, Methodist Neurologic Institute

Houston, Texas, United States

Site Status

Providence Saint Peter Hospital

Centralia, Washington, United States

Site Status

Countries

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United States

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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NP001-10-002

Identifier Type: -

Identifier Source: org_study_id