Ignite Creation Date:
2025-12-24 @ 6:33 PM
Ignite Modification Date:
2026-02-23 @ 8:07 PM
Study NCT ID:
NCT02794857
Status:
COMPLETED
Last Update Posted:
2018-05-07
First Post:
2016-05-24
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000690', 'term': 'Amyotrophic Lateral Sclerosis'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D007249', 'term': 'Inflammation'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D057177', 'term': 'TDP-43 Proteinopathies'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 138}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-08-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-05', 'completionDateStruct': {'date': '2017-12-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2018-05-03', 'studyFirstSubmitDate': '2016-05-24', 'studyFirstSubmitQcDate': '2016-06-06', 'lastUpdatePostDateStruct': {'date': '2018-05-07', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2016-06-09', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2017-12-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire', 'timeFrame': 'Baseline and 6 months'}], 'secondaryOutcomes': [{'measure': 'Change in pulmonary function as measured by slow vital capacity readings', 'timeFrame': 'Baseline and 6 months'}, {'measure': 'Time to tracheotomy', 'timeFrame': 'Up to 6 months'}, {'measure': 'Change in levels of blood inflammatory biomarkers', 'timeFrame': 'Baseline, 3 and 6 months'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['ALS', 'Amyotrophic Lateral Sclerosis', 'Motor Neuron Disease', 'Central Nervous System Diseases', 'Nervous System Diseases', 'Neurodegenerative Diseases', 'Neuromuscular Diseases', 'Inflammation', 'C-Reactive Protein'], 'conditions': ['Amyotrophic Lateral Sclerosis']}, 'referencesModule': {'references': [{'pmid': '39457678', 'type': 'DERIVED', 'citation': '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.'}]}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '21 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Diagnosis of clinically possible, clinically probable (with or without laboratory support), or clinically definite ALS (using the revised El Escorial Criteria)\n* Forced vital capacity greater than or equal to 65% of that predicted for age and height\n* Onset of ALS-related weakness less than 3 years prior to first dose of study drug\n* Plasma high sensitivity C-reactive protein (hs-CRP) concentration of greater than or equal to 0.113 mg/dL at pre-screening/screening\n* Stable dose (greater than 30 days) of riluzole if undergoing treatment with this agent\n* For females: Not be of childbearing potential or agree to use adequate birth control during the study\n\nKey Exclusion Criteria:\n\n* Life expectancy of less than 6 months\n* Tracheotomy or be using ventilatory assistance, including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)\n* Active pulmonary disease\n* Gastrostomy\n* Stem cell therapy\n* Immune modulator therapy or participation in studies of other agents within 12 weeks of pre-screening/screening\n* Unstable medical condition other than ALS'}, 'identificationModule': {'nctId': 'NCT02794857', 'briefTitle': 'Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation', 'organization': {'class': 'INDUSTRY', 'fullName': 'Neuraltus Pharmaceuticals, Inc.'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'NP001-10-003'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'NP001', 'description': 'NP001 2 mg/kg by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6', 'interventionNames': ['Drug: NP001']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'NP001', 'type': 'DRUG', 'armGroupLabels': ['NP001']}, {'name': 'Placebo', 'type': 'DRUG', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85013', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': "St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics", 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '85259', 'city': 'Scottsdale', 'state': 'Arizona', 'country': 'United States', 'facility': 'Mayo Clinic Arizona', 'geoPoint': {'lat': 33.50921, 'lon': -111.89903}}, {'zip': '90048', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Cedars-Sinai Medical Center', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '92862', 'city': 'Orange', 'state': 'California', 'country': 'United States', 'facility': 'University of California, Irvine, Department of Neurology', 'geoPoint': {'lat': 33.78779, 'lon': -117.85311}}, {'zip': '94115', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'Forbes Norris MDA/ALS Research Center, CPMC', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '32224', 'city': 'Jacksonville', 'state': 'Florida', 'country': 'United States', 'facility': 'Mayo Clinic Florida', 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami Miller School of Medicine', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University, Department of Neurology', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'University of Kansas Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '40536-0293', 'city': 'Lexington', 'state': 'Kentucky', 'country': 'United States', 'facility': 'University of Kentucky, Albert B. Chandler Medical Center', 'geoPoint': {'lat': 37.98869, 'lon': -84.47772}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55414', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Clinical & Translational Science Institute, University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University School of Medicine', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '28207', 'city': 'Charlotte', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Carolinas Medical Center, Neurosciences Instutite-Neurology', 'geoPoint': {'lat': 35.22709, 'lon': -80.84313}}, {'zip': '27705', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke Neurological Disorders Clinic at Morreene Road', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '44195', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'Cleveland Clinic Foundation-Cleveland Clinic Hospital', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '43210', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': 'The Ohio State University Wexner Medical Center', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '97213', 'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'facility': 'Providence Brain & Spine Institute, ALS Center', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Houston Methodist Neurological Institute', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '78229', 'city': 'San Antonio', 'state': 'Texas', 'country': 'United States', 'facility': 'University of Texas Health Sciences Center San Antonio', 'geoPoint': {'lat': 29.42412, 'lon': -98.49363}}, {'zip': 'H3A 2B4', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': 'Montreal Neurological Institute', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}], 'overallOfficials': [{'name': 'Gil Block, MD, PhD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Neuraltus Pharmaceuticals, Inc.'}, {'name': 'Robert G. Miller, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'California Pacific Medical Center'}, {'name': 'Jonathan Katz, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'California Pacific Medical Center'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Neuraltus Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}