Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation
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
1 other identifier
interventional
138
2 countries
22
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Aug 2016
Shorter than P25 for phase_2
22 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 24, 2016
CompletedFirst Posted
Study publicly available on registry
June 9, 2016
CompletedStudy Start
First participant enrolled
August 29, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 12, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
December 12, 2017
CompletedMay 7, 2018
May 1, 2018
1.3 years
May 24, 2016
May 3, 2018
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire
Baseline and 6 months
Secondary Outcomes (3)
Change in pulmonary function as measured by slow vital capacity readings
Baseline and 6 months
Time to tracheotomy
Up to 6 months
Change in levels of blood inflammatory biomarkers
Baseline, 3 and 6 months
Study Arms (2)
NP001
EXPERIMENTALNP001 2 mg/kg by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6
Placebo
PLACEBO COMPARATORNormal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (22)
St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
Phoenix, Arizona, 85013, United States
Mayo Clinic Arizona
Scottsdale, Arizona, 85259, United States
Cedars-Sinai Medical Center
Los Angeles, California, 90048, United States
University of California, Irvine, Department of Neurology
Orange, California, 92862, United States
Forbes Norris MDA/ALS Research Center, CPMC
San Francisco, California, 94115, United States
Mayo Clinic Florida
Jacksonville, Florida, 32224, United States
University of Miami Miller School of Medicine
Miami, Florida, 33136, United States
Emory University, Department of Neurology
Atlanta, Georgia, 30322, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
University of Kentucky, Albert B. Chandler Medical Center
Lexington, Kentucky, 40536-0293, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Clinical & Translational Science Institute, University of Minnesota
Minneapolis, Minnesota, 55414, United States
Washington University School of Medicine
St Louis, Missouri, 63110, United States
Columbia University Medical Center
New York, New York, 10032, United States
Carolinas Medical Center, Neurosciences Instutite-Neurology
Charlotte, North Carolina, 28207, United States
Duke Neurological Disorders Clinic at Morreene Road
Durham, North Carolina, 27705, United States
Cleveland Clinic Foundation-Cleveland Clinic Hospital
Cleveland, Ohio, 44195, United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210, United States
Providence Brain & Spine Institute, ALS Center
Portland, Oregon, 97213, United States
Houston Methodist Neurological Institute
Houston, Texas, 77030, United States
University of Texas Health Sciences Center San Antonio
San Antonio, Texas, 78229, United States
Montreal Neurological Institute
Montreal, Quebec, H3A 2B4, Canada
Related Publications (1)
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.
PMID: 39457678DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- 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
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 24, 2016
First Posted
June 9, 2016
Study Start
August 29, 2016
Primary Completion
December 12, 2017
Study Completion
December 12, 2017
Last Updated
May 7, 2018
Record last verified: 2018-05