Ignite Creation Date:
2025-12-24 @ 9:08 PM
Ignite Modification Date:
2025-12-30 @ 10:34 PM
Study NCT ID:
NCT05200104
Status:
WITHDRAWN
Last Update Posted:
2024-12-16
First Post:
2022-01-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000326', 'term': 'Adrenoleukodystrophy'}], 'ancestors': [{'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020279', 'term': 'Hereditary Central Nervous System Demyelinating Diseases'}, {'id': 'D056784', 'term': 'Leukoencephalopathies'}, {'id': 'D003711', 'term': 'Demyelinating Diseases'}, {'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D018901', 'term': 'Peroxisomal Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D000309', 'term': 'Adrenal Insufficiency'}, {'id': 'D000307', 'term': 'Adrenal Gland Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 0}}, 'statusModule': {'whyStopped': 'Funding unavailable', 'overallStatus': 'WITHDRAWN', 'startDateStruct': {'date': '2023-09', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-12', 'completionDateStruct': {'date': '2024-09', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-12-10', 'studyFirstSubmitDate': '2022-01-06', 'studyFirstSubmitQcDate': '2022-01-06', 'lastUpdatePostDateStruct': {'date': '2024-12-16', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2022-01-20', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-09', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Pharmacokinetic', 'timeFrame': 'Week 4', 'description': 'Deuterated and protonated forms of (R)-pioglitazone and (S)-pioglitazone: Cmax'}, {'measure': 'PharmacoKinetic', 'timeFrame': 'Week 4', 'description': 'Deuterated and protonated forms of (R)-pioglitazone and (S)-pioglitazone: AUC0-24 (AUCtau)'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['ALD (Adrenoleukodystrophy)']}, 'descriptionModule': {'briefSummary': 'A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL065 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN).', 'detailedDescription': "A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL065 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN).\n\nThere will be a total of 3 study periods.\n\n* Screening period of a maximum of 4 weeks prior to the open-label Treatment Period (Baseline Visit - V2). This period can be exceptionally extended for 2 weeks\n* Open-label Treatment Period of 12 weeks\n* Follow-up Period of 2 weeks after the last intake of the treatment (V5-End of Treatment Visit (EoT)).\n\nDuring the treatment period, very long chain fatty acids (VLCFA) will be assessed every 4 weeks, to evaluate the kinetics of the PXL065 effect. Neurofilament light (NfL) will be assessed after 8 and 12 weeks of treatment, and other exploratory biomarkers after 12 weeks of treatment. A follow up period will allow continued monitoring the subjects' safety and evaluation of the kinetics of the 2 main biomarkers (VLCFA and NfL) at 2 weeks after the last intake of the treatment."}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male subjects with either a confirmed diagnosis of AMN by genetic testing (mutation in the ATP binding cassette subfamily D (ABCD1 gene)) or a family history of X-linked adrenoleukodystrophy (ALD) together with an elevation in VLCFA obtained from overnight fasting plasma sample at Screening Visit (V1).\n* Age: ≥ 18 to ≤ 65 years at informed consent signature.\n* Normal brain magnetic resonance imaging (MRI) or brain MRI showing non-specific abnormalities that can be observed in AMN subjects without signs of cerebral form of ALD (C-ALD). MRI must be performed within 6 months prior to V2. If there is no available brain MRI within this period, a brain MRI must be performed before V2\n\nExclusion Criteria:\n\n* Any progressive neurological disease other than AMN.\n* Arrested or progressing C-ALD as defined by cerebral lesions (except for non-specific abnormalities that can be observed in AMN subjects).\n* Prior receipt of an allogeneic hematopoietic stem cell transplant or gene therapy'}, 'identificationModule': {'nctId': 'NCT05200104', 'briefTitle': 'Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Poxel SA'}, 'officialTitle': 'An Open-label Phase 2a Study to Assess the Pharmacokinetics and Pharmacodynamic of PXL065 Parameters After 12 Weeks of Treatment in Male Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)', 'orgStudyIdInfo': {'id': 'PXL065-005'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'PXL065', 'description': 'PXL065 22.5 mg QD', 'interventionNames': ['Drug: PXL065']}], 'interventions': [{'name': 'PXL065', 'type': 'DRUG', 'description': 'PXL065 22.5 mg QD', 'armGroupLabels': ['PXL065']}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Poxel SA', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}