Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting
Trial record 3 of 5 for:    NP001

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2016 by Neuraltus Pharmaceuticals, Inc.
Sponsor:
Information provided by (Responsible Party):
Neuraltus Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT02794857
First received: May 24, 2016
Last updated: September 21, 2016
Last verified: September 2016
  Purpose
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.

Condition Intervention Phase
Amyotrophic Lateral Sclerosis
Drug: NP001
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS) and Evidence of Elevated Systemic Inflammation

Resource links provided by NLM:


Further study details as provided by Neuraltus Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire [ Time Frame: Baseline and 6 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in pulmonary function as measured by slow vital capacity readings [ Time Frame: Baseline and 6 months ] [ Designated as safety issue: No ]
  • Time to tracheotomy [ Time Frame: Up to 6 months ] [ Designated as safety issue: No ]
  • Change in levels of blood inflammatory biomarkers [ Time Frame: Baseline, 3 and 6 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 120
Study Start Date: August 2016
Estimated Primary Completion Date: September 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 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
Drug: NP001
Placebo Comparator: Placebo
Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6
Drug: Placebo

Detailed Description:
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.
  Eligibility

Ages Eligible for Study:   21 Years to 80 Years   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Key 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

Key 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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02794857

Contacts
Contact: Frances Faurot 415-912-1981 ffaurot@neuraltus.com

Locations
United States, Arizona
St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics Recruiting
Phoenix, Arizona, United States, 85013
Contact: Ashley Reece    602-406-4775    Ashley.Reece@dignityhealth.org   
Principal Investigator: Shafeeq Ladha, MD         
United States, California
Forbes Norris MDA/ALS Research Center, CPMC Recruiting
San Francisco, California, United States, 94115
Contact: Dallas A. Forshew, RN, BSN    415-309-5178    ForsheD@cpmcri.org   
Principal Investigator: Jonathan S. Katz, MD         
United States, Florida
University of Miami Miller School of Medicine Recruiting
Miami, Florida, United States, 33136
Contact: Julie Steele, RN    305-243-7336    jsteele@med.miami.edu   
Principal Investigator: Michael Benatar, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Christy Nichols    913-945-9945    Cnichols2@kumc.edu   
Principal Investigator: Richard J. Barohn, MD         
United States, Kentucky
University of Kentucky, Albert B. Chandler Medical Center Recruiting
Lexington, Kentucky, United States, 40536-0293
Contact: Meghann Bruno, RN, BSN    859-218-5064    Meghann.bruno@uky.edu   
Principal Investigator: Edward J. Kasarskis, MD, PhD         
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Leah Miller    617-724-7398    lmiller33@partners.org   
Contact: Taylor Mezoian    617-643-0312    tmezoian@partners.org   
Principal Investigator: James Berry, MD, MPH         
United States, Oregon
Providence Brain & Spine Institute, ALS Center Recruiting
Portland, Oregon, United States, 97213
Contact: Arlena Georgeson    503-962-1171    arlena.georgeson@providence.org   
Principal Investigator: Kimberly Goslin, MD, PhD         
Sponsors and Collaborators
Neuraltus Pharmaceuticals, Inc.
Investigators
Study Director: Gil Block, MD, PhD Neuraltus Pharmaceuticals, Inc.
Principal Investigator: Robert G. Miller, MD California Pacific Medical Center
Principal Investigator: Jonathan Katz, MD California Pacific Medical Center
  More Information

Responsible Party: Neuraltus Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02794857     History of Changes
Other Study ID Numbers: NP001-10-003 
Study First Received: May 24, 2016
Last Updated: September 21, 2016
Health Authority: United States: Food and Drug Administration

Keywords provided by Neuraltus Pharmaceuticals, Inc.:
ALS
Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Central Nervous System Diseases
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Diseases
Inflammation
C-Reactive Protein

Additional relevant MeSH terms:
Sclerosis
Inflammation
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on September 30, 2016