Ignite Creation Date:
2025-12-26 @ 4:00 PM
Ignite Modification Date:
2025-12-26 @ 4:00 PM
Study NCT ID:
NCT04528706
Status:
UNKNOWN
Last Update Posted:
2023-07-18
First Post:
2020-07-31
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 13}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2019-09-13', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-01', 'completionDateStruct': {'date': '2023-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2023-07-17', 'studyFirstSubmitDate': '2020-07-31', 'studyFirstSubmitQcDate': '2020-08-24', 'lastUpdatePostDateStruct': {'date': '2023-07-18', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-08-27', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-12-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'evaluate whether MIN-102 can halt disease progression of cALD if administered prior to hematopoietic stem-cell transplantation (HSCT), as determined by serial clinical and MRI investigations in pediatric subjects.', 'timeFrame': '6 months- 2 years'}, {'measure': '"arrested disease" will be assessed at 24 weeks and at 96 weeks', 'timeFrame': 'at 24 weeks and 96 weeks', 'description': 'arrested disease is defined using: Cahnge in NFS from Baseline, Free of MFD and lack of lesion progression on MRI'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cerebral Adrenoleukodystrophy']}, 'descriptionModule': {'briefSummary': 'An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT)'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '12 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Males aged ≥2 and ≤12 years with a diagnosis of X-linked ALD\n* White matter involvement as determined by cerebral MRI lesions without Gd enhancement at baseline (Population 1), or with Gd enhancement at baseline (Population 2).\n* Major Functional Disabilities (MFD) score of 0.\n* Baseline Loes score \\>0 and ≤10\n* Gadolinium Intensity Score \\>2\n\nExclusion Criteria:\n\n* Previous HSCT\n* Too far progressed inflammatory brain lesions'}, 'identificationModule': {'nctId': 'NCT04528706', 'acronym': 'NEXUS', 'briefTitle': 'A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Minoryx Therapeutics, S.L.'}, 'officialTitle': 'An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)', 'orgStudyIdInfo': {'id': 'MT-2-02'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'MIN-102', 'interventionNames': ['Drug: MIN-102']}], 'interventions': [{'name': 'MIN-102', 'type': 'DRUG', 'description': 'Once-daily dosing with a volume specified by the pharmacokineteic specialist to achive the desired plasma exposure. Min-102 Oral suspension, strength 15mg/ml.', 'armGroupLabels': ['MIN-102']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'B16641NZ', 'city': 'Bueno Aires', 'state': 'Buenos Aires', 'country': 'Argentina', 'facility': 'Hernan Amartino'}, {'city': 'Paris', 'country': 'France', 'facility': 'CHU Kremlin Bicêtre', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Göttingen', 'country': 'Germany', 'facility': 'Universitätsmedizin Göttingen Georg-August-Universität', 'geoPoint': {'lat': 51.53443, 'lon': 9.93228}}, {'city': 'Hamburg', 'country': 'Germany', 'facility': 'Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin', 'geoPoint': {'lat': 53.55073, 'lon': 9.99302}}, {'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Sant Joan de Déu', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Minoryx Therapeutics, S.L.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}