Ignite Creation Date:
2025-12-25 @ 5:08 AM
Ignite Modification Date:
2026-03-03 @ 12:44 AM
Study NCT ID:
NCT05059327
Status:
COMPLETED
Last Update Posted:
2025-06-04
First Post:
2021-08-20
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Australia']}, 'conditionBrowseModule': {'meshes': [{'id': 'D014402', 'term': 'Tuberous Sclerosis'}, {'id': 'D012640', 'term': 'Seizures'}], 'ancestors': [{'id': 'D006222', 'term': 'Hamartoma'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D009378', 'term': 'Neoplasms, Multiple Primary'}, {'id': 'D009386', 'term': 'Neoplastic Syndromes, Hereditary'}, {'id': 'D065703', 'term': 'Malformations of Cortical Development, Group I'}, {'id': 'D054220', 'term': 'Malformations of Cortical Development'}, {'id': 'D009421', 'term': 'Nervous System Malformations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020752', 'term': 'Neurocutaneous Syndromes'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000596770', 'term': '2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER', 'interventionModelDescription': 'This is a multi-center, randomized, double-blind, placebo-controlled, 30-week, cross-over (Part A) followed by a 52-week open-label extension (OLE) (Part B) study.'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 61}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2022-03-03', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2025-04-28', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-06-03', 'studyFirstSubmitDate': '2021-08-20', 'studyFirstSubmitQcDate': '2021-09-20', 'lastUpdatePostDateStruct': {'date': '2025-06-04', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-09-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-02-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change in seriousness of disease as assessed by Most Impactful Symptoms Scale in Periods 2 (weeks 13 to 16) and Period 4 (weeks 27 to 30) compared to baseline.', 'timeFrame': '30 weeks'}, {'measure': 'Frequency of seizures detected by the wearable device evaluated as the change from Baseline compared to study treatment in Period 1 (weeks 13 to 16) and Period 4 (weeks 27 to 30).', 'timeFrame': '30 weeks'}, {'measure': 'Intensity of seizures detected by the wearable device evaluated as the change from Baseline compared to study treatment Periods 1 and 4.', 'timeFrame': '30 weeks'}], 'primaryOutcomes': [{'measure': 'Mean percentage in monthly seizure frequency during the maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30).', 'timeFrame': '30 weeks'}], 'secondaryOutcomes': [{'measure': 'Number of patients considered treatment responders.', 'timeFrame': '30 weeks'}, {'measure': 'Longest seizure free interval (i.e., seizure free days).', 'timeFrame': '30 weeks'}, {'measure': 'Change in the severity of symptoms of TSC as measured by Caregiver Global Impression of Change (CGIC) score during maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30) compared to Baseline.', 'timeFrame': '30 weeks'}, {'measure': 'Change in the Sheehan Disability Scale during maintenance dosing in Period 2 (Weeks 13 to 16) and Period 4 (Weeks 27-30) compared to baseline.', 'timeFrame': '30 weeks'}, {'measure': 'Safety of the study drug in children, adolescents and young adults with seizures associated with TSC.', 'timeFrame': '82 weeks', 'description': 'Measured in terms of incidence, nature, and severity of adverse events, vital signs, physical examination, clinical chemistry, hematology, electrocardiograms, and urinalysis, as well as treatment delays, dose reductions, and dose discontinuations. In addition, suicidal ideation will be assessed using S-STS.'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Basimglurant', 'TSC', 'Seizures', 'NOE-101'], 'conditions': ['Tuberous Sclerosis Complex']}, 'descriptionModule': {'briefSummary': 'The study intends to show that basimglurant (NOE-101) provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC).', 'detailedDescription': "The study drug (NOE-101, basimglurant) is a potent inhibitor of metabotropic glutamate receptor 5 (mGluR5) which controls a wide range of processes in the brain, spinal cord, retina, and peripheral nervous system. In animal studies, the inhibition of this receptor has shown therapeutic potential for the treatment of Tuberous Sclerosis Complex (TSC). This receptor's inhibition decreases the frequency of seizures. In previous clinical trials, the study drug has shown an advantageous safety profile in children and adolescents.\n\nThe objective of this study is to find an optimal dose at which the study drug will lead to a decrease in the duration, frequency and intensity of seizures in children, adolescents and young adults with TSC, while being well tolerated. All patients who positively respond and tolerate the medicine will be offered the possibility to continue in an open label extension."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '30 Years', 'minimumAge': '5 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria (summary):\n\n* Ability and willingness to provide informed assent or written consent or consent from their legal representative.\n* Fluency in the language of the study staff\n* Age 5 to 30 years at study entry\n* A documented history of TSC\n* Refractory seizure history\n* Currently receiving one or more anti-epileptic drugs (AEDs)\n* Stable medications or interventions for epilepsy\n* Willingness to complete Patient Reported Outcome assessments\n* For female patients of childbearing potential:\n\n 1. Willingness to undergo serum or urinary pregnancy testing at screening and during the trial period.\n 2. Willingness to use contraception.\n\nExclusion Criteria (summary):\n\n* Neurologic disease other than TSC\n* Recent anoxic episode\n* Patient weight below 15kg\n* Clinically significant unstable medical condition(s)\n* Pregnancy or lactation'}, 'identificationModule': {'nctId': 'NCT05059327', 'briefTitle': 'Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC', 'organization': {'class': 'INDUSTRY', 'fullName': 'Noema Pharma AG'}, 'officialTitle': 'A Phase 2B, Multicenter, 30-week, Prospective, Cross-over, Double-blind, Randomized, Placebo-controlled Study Followed by a 52-Week Open-label Extension Study to Evaluate the Efficacy and Safety of Basimglurant Adjunctive to Ongoing Anticonvulsive Therapy in Children, Adolescents, and Young Adults With Uncontrolled Seizures Associated With Tuberous Sclerosis Complex', 'orgStudyIdInfo': {'id': 'NOE-TSC-201'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Arm A (Basimglurant/NOE-101 to Placebo)', 'description': 'Basimglurant to Placebo', 'interventionNames': ['Drug: Basimglurant with crossover to Placebo']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Arm B (Placebo to Basimglurant/NOE-101)', 'description': 'Placebo to Basimglurant', 'interventionNames': ['Drug: Placebo with crossover to Basimglurant']}], 'interventions': [{'name': 'Basimglurant with crossover to Placebo', 'type': 'DRUG', 'description': 'Basimglurant with crossover to Placebo', 'armGroupLabels': ['Arm A (Basimglurant/NOE-101 to Placebo)']}, {'name': 'Placebo with crossover to Basimglurant', 'type': 'DRUG', 'description': 'Placebo with crossover to Basimglurant', 'armGroupLabels': ['Arm B (Placebo to Basimglurant/NOE-101)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'David Geffen School of Medicine at UCLA (Site #: 101)', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Kennedy Krieger Institute (Site #: 110)', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02115-5724', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital (Site #: 102)", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '48073-6712', 'city': 'Royal Oak', 'state': 'Michigan', 'country': 'United States', 'facility': 'William Beaumont Hospital - Royal Oak (Site #: 104)', 'geoPoint': {'lat': 42.48948, 'lon': -83.14465}}, {'zip': '55113-1306', 'city': 'Roseville', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Minnesota Epilepsy Group PA (Site #: 105)', 'geoPoint': {'lat': 45.00608, 'lon': -93.15661}}, {'zip': '10532', 'city': 'Hawthorne', 'state': 'New York', 'country': 'United States', 'facility': "Boston Children's Health Physicians (BCHP) (Site #: 111)", 'geoPoint': {'lat': 41.10732, 'lon': -73.79597}}, {'zip': '27705-4699', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': "Duke Children's Hospital and Health Center (Site #: 106)", 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '44106-1716', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'University Hospitals Cleveland Medical Center, Rainbow Babies and Childrens Hospital (Site #: 107)', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '77030-3000', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'The University of Texas Medical School at Houston (Site #: 103)', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '500034', 'city': 'Hyderabad', 'state': 'Andhra Pradesh', 'country': 'India', 'facility': 'Citi Neuro Centre (Site # 806)'}, {'zip': '500034', 'city': 'Hyderabad', 'state': 'Andhra Pradesh', 'country': 'India', 'facility': 'Rainbow Childrens Hospital (Site # 803)'}, {'zip': '400026', 'city': 'Mumbai', 'state': 'Maharashtra', 'country': 'India', 'facility': 'Jaslok Hospital and Research Centre (Site # 801)', 'geoPoint': {'lat': 19.07283, 'lon': 72.88261}}, {'zip': '411006', 'city': 'Pune', 'state': 'Maharashtra', 'country': 'India', 'facility': 'Deenanath Mangeshkar Hospital and Research Centre (Site # 805)', 'geoPoint': {'lat': 18.51957, 'lon': 73.85535}}, {'zip': '110029', 'city': 'New Delhi', 'state': 'National Capital Territory of Delhi', 'country': 'India', 'facility': 'All India Institute of Medical Sciences (Site # 804)', 'geoPoint': {'lat': 28.62137, 'lon': 77.2148}}, {'zip': '632004', 'city': 'Vellore', 'state': 'Tamil Nadu', 'country': 'India', 'facility': 'Christian Medical College (Site # 807)', 'geoPoint': {'lat': 12.9184, 'lon': 79.13255}}, {'zip': '91120', 'city': 'Jerusalem', 'country': 'Israel', 'facility': 'Hadassah Medical Center - 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PPDS (Site # 304)', 'geoPoint': {'lat': 53.48771, 'lon': -2.29042}}, {'zip': 'CF14 4XW', 'city': 'Cardiff', 'state': 'South Glamorgan', 'country': 'United Kingdom', 'facility': "Noahs Ark Children's Hospital (Site # 306)", 'geoPoint': {'lat': 51.48, 'lon': -3.18}}], 'overallOfficials': [{'name': 'Clinical Director, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Noema Pharma AG'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Noema Pharma AG', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}