Ignite Creation Date:
2025-12-24 @ 11:37 PM
Ignite Modification Date:
2025-12-25 @ 9:27 PM
Study NCT ID:
NCT04288856
Status:
TERMINATED
Last Update Posted:
2023-04-18
First Post:
2020-02-26
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB078 Administered to Previously Treated Adults C9ORF72-Associated Amyotrophic Lateral Sclerosis (ALS)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000690', 'term': 'Amyotrophic Lateral Sclerosis'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D057177', 'term': 'TDP-43 Proteinopathies'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE', 'maskingDescription': 'During the blinded loading period, the following individuals will be masked:\n\n* Investigator\n* Study staff\n* Participants\n\nAfter the loading period has been completed, subsequent doses will be open-label.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 75}}, 'statusModule': {'whyStopped': 'There was no evidence of benefit across efficacy endpoints in the randomized trial, 245AS101. Accordingly, Biogen has made the difficult decision to discontinue the BIIB078 program including this open label extension trial, 245AS102.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2020-04-28', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-04', 'completionDateStruct': {'date': '2022-05-03', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-04-13', 'studyFirstSubmitDate': '2020-02-26', 'studyFirstSubmitQcDate': '2020-02-26', 'lastUpdatePostDateStruct': {'date': '2023-04-18', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-02-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-05-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants with Adverse Events (AEs)', 'timeFrame': 'Baseline up to Day 785', 'description': 'AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention.'}, {'measure': 'Number of Participants with Serious Adverse Events (SAEs)', 'timeFrame': 'Screening up to Day 785', 'description': 'An SAE is any untoward medical occurrence that, at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect or any other situation according to medical or scientific judgment.'}], 'secondaryOutcomes': [{'measure': 'Serum concentration of BIIB078', 'timeFrame': 'Baseline and at multiple time points up to Day 729'}, {'measure': 'Cerebrospinal Fluid (CSF) concentration of BIIB078', 'timeFrame': 'Baseline and at multiple time points up to Day 729'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Amyotrophic Lateral Sclerosis']}, 'descriptionModule': {'briefSummary': 'The primary objective is to evaluate the long-term safety and tolerability of BIIB078 in participants with chromosome 9 open reading frame 72-amyotrophic lateral sclerosis (C9ORF72-ALS).\n\nThe secondary objective is to evaluate the pharmacokinectic (PK) of BIIB078 in participants with C9ORF72-ALS.', 'detailedDescription': 'This study is an extension study of NCT03626012.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Participants must have completed study NCT03626012 through the first follow-up clinic visit that follows the final dosing visit without missing more than 1 dose of study treatment.\n* Participants taking concomitant riluzole at study entry must be on a stable dose for ≥30 days prior to the first dose of study treatment (Day 1). Participants taking concomitant riluzole must be willing to continue with the same dose regimen throughout the study, unless the Investigator determines that riluzole should be discontinued for medical reasons, in which case it may not be restarted during the study.\n* Participants taking concomitant edaravone at study entry must be on a stable dose for ≥60 days prior to the first dose of study treatment (Day 1). Participants taking concomitant edaravone must be willing to continue with the same dose regimen throughout the study, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study. Edaravone may not be administered on dosing days of this study.\n\nKey Exclusion Criteria:\n\n* History of drug abuse or alcoholism ≤6 months before study enrollment that would limit participation in the study, as determined by the Investigator.\n* Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter.\n* History of or positive test result at Screening for human immunodeficiency virus. The requirement for testing at Screening may be omitted if it is not permitted by local regulations.\n* Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs) or biological agent within 1 month of Screening or 5 half-lives of study agent, whichever is longer.\n\nNote: Other protocol-specific inclusion/exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT04288856', 'briefTitle': 'Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB078 Administered to Previously Treated Adults C9ORF72-Associated Amyotrophic Lateral Sclerosis (ALS)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Biogen'}, 'officialTitle': 'An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB078 Administered to Previously Treated Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis', 'orgStudyIdInfo': {'id': '245AS102'}, 'secondaryIdInfos': [{'id': '2019-004798-14', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort A: BIIB078 First Dosage', 'description': 'BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.', 'interventionNames': ['Drug: BIIB078']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort B: BIIB078 Second Dosage', 'description': 'BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.', 'interventionNames': ['Drug: BIIB078']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort C: BIIB078 Third Dosage', 'description': 'BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.', 'interventionNames': ['Drug: BIIB078']}, {'type': 'EXPERIMENTAL', 'label': 'Possible Cohort D: BIIB078 Fourth Dosage', 'description': 'BIIB078 will be administered as 3 doses during the loading period, approximately 2 weeks apart, and maintenance doses, approximately 4 weeks apart, via IT infusion.', 'interventionNames': ['Drug: BIIB078']}], 'interventions': [{'name': 'BIIB078', 'type': 'DRUG', 'description': 'Administered as specified in the treatment arm.', 'armGroupLabels': ['Cohort A: BIIB078 First Dosage', 'Cohort B: BIIB078 Second Dosage', 'Cohort C: BIIB078 Third Dosage', 'Possible Cohort D: BIIB078 Fourth Dosage']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92037', 'city': 'La Jolla', 'state': 'California', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 32.84727, 'lon': -117.2742}}, {'zip': '90048', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '32224', 'city': 'Jacksonville', 'state': 'Florida', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '68506', 'city': 'Lincoln', 'state': 'Nebraska', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 40.8, 'lon': -96.66696}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '37920', 'city': 'Knoxville', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Research Site', 'geoPoint': {'lat': 35.96064, 'lon': -83.92074}}, {'zip': 'T2N 1N4', 'city': 'Calgary', 'state': 'Alberta', 'country': 'Canada', 'facility': 'Research Site', 'geoPoint': {'lat': 51.05011, 'lon': -114.08529}}, {'zip': 'T6G 2B7', 'city': 'Edmonton', 'state': 'Alberta', 'country': 'Canada', 'facility': 'Research Site', 'geoPoint': {'lat': 53.55014, 'lon': -113.46871}}, {'zip': 'M4N 3M5', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Research Site', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': 'H3A 2B4', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': 'Research Site', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'zip': '3508 GA', 'city': 'Utrecht', 'country': 'Netherlands', 'facility': 'Research Site', 'geoPoint': {'lat': 52.09083, 'lon': 5.12222}}, {'zip': '9007', 'city': 'Sankt Gallen', 'country': 'Switzerland', 'facility': 'Research Site', 'geoPoint': {'lat': 47.42391, 'lon': 9.37477}}, {'zip': 'NW1 2PG', 'city': 'London', 'state': 'Greater London', 'country': 'United Kingdom', 'facility': 'Research Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'SE5 9RS', 'city': 'London', 'state': 'Greater London', 'country': 'United Kingdom', 'facility': 'Research Site', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'S10 2HQ', 'city': 'Sheffield', 'state': 'South Yorkshire', 'country': 'United Kingdom', 'facility': 'Research Site', 'geoPoint': {'lat': 53.38297, 'lon': -1.4659}}], 'overallOfficials': [{'name': 'Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Biogen'}]}, 'ipdSharingStatementModule': {'url': 'https://vivli.org/', 'ipdSharing': 'YES', 'description': "In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/"}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Biogen', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}