Ignite Creation Date:
2025-12-24 @ 4:56 PM
Ignite Modification Date:
2025-12-25 @ 2:38 PM
Study NCT ID:
NCT04520750
Status:
COMPLETED
Last Update Posted:
2024-08-29
First Post:
2020-03-06
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'submissionInfos': [{'resetDate': '2025-01-14', 'mcpReleaseN': 3, 'releaseDate': '2024-12-24'}], 'estimatedResultsFirstSubmitDate': '2024-12-24'}}, 'conditionBrowseModule': {'meshes': [{'id': 'D053549', 'term': 'Pachyonychia Congenita'}], 'ancestors': [{'id': 'D004476', 'term': 'Ectodermal Dysplasia'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D012868', 'term': 'Skin Abnormalities'}, {'id': 'D012873', 'term': 'Skin Diseases, Genetic'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009260', 'term': 'Nail Diseases'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Open label'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 36}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-09-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-08', 'completionDateStruct': {'date': '2022-12-15', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-08-28', 'studyFirstSubmitDate': '2020-03-06', 'studyFirstSubmitQcDate': '2020-08-18', 'lastUpdatePostDateStruct': {'date': '2024-08-29', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-08-20', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-06-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of Treatment-Emergent Adverse Events', 'timeFrame': '18 Months'}, {'measure': 'Changes in vital sign measurements from baseline', 'timeFrame': '18 months'}, {'measure': 'Changes in weight measurements from baseline', 'timeFrame': '18 months'}, {'measure': 'Changes in clinical laboratory measurements from baseline', 'timeFrame': '18 months'}], 'secondaryOutcomes': [{'measure': 'Patient Global Assessment of Activities Scale', 'timeFrame': '18 Months'}, {'measure': 'Pain at its worst as assessed by NRS', 'timeFrame': '18 months'}, {'measure': 'PROMIS Pain Interference Short 6A as assessed by likert scale', 'timeFrame': '18 months'}, {'measure': 'PROMIS Physical Function as assessed by likert scale', 'timeFrame': '18 months'}, {'measure': 'Patient Global Impression of Severity as assessed by likert scale', 'timeFrame': '18 months'}, {'measure': 'Clinician Global Impression of Severity as assessed by likert scale', 'timeFrame': '18 months'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Pachyonychia Congenita']}, 'descriptionModule': {'briefSummary': 'VALO-2 is a multicenter, open-label extension (OLE) study enrolling patients with genotyped keratin mutations KRT6A, KRT6B and KRT16 who were previously treated with investigational PTX-022 during the VALO study. The purpose of the OLE study is to investigate long term exposure to investigational PTX-022 and evaluate safety and efficacy data. A sub-study is included to evaluate safety and efficacy of patients with genotyped keratin mutations of KRT6C and KRT17 not previously treated with investigational PTX-022.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Adult patients 18 years or older,\n* Previously completed the VALO protocol, or VALO-2 K6C/17 sub-study, and received meaningful benefit from investigational PTX-022 as determined by the clinician.\n\nKey Exclusion Criteria:\n\n* Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022'}, 'identificationModule': {'nctId': 'NCT04520750', 'acronym': 'VALO-2', 'briefTitle': 'VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita', 'organization': {'class': 'INDUSTRY', 'fullName': 'Palvella Therapeutics, Inc.'}, 'officialTitle': 'VALO-2: A Multicenter, Phase 3b, Open-Label Treatment Extension Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita', 'orgStudyIdInfo': {'id': 'PALV-03'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Open label treatment arm', 'description': 'PTX-022 QTORIN', 'interventionNames': ['Drug: PTX-022']}], 'interventions': [{'name': 'PTX-022', 'type': 'DRUG', 'description': 'Safety and Efficacy of PTX-022 in the Treatment of Adults with Pachyonychia Congenita', 'armGroupLabels': ['Open label treatment arm']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85053', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': 'Arizona Research Center', 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '55432', 'city': 'Fridley', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Minnesota Clinical Study Center', 'geoPoint': {'lat': 45.08608, 'lon': -93.26328}}, {'zip': '84107', 'city': 'Murray', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah', 'geoPoint': {'lat': 40.66689, 'lon': -111.88799}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Palvella Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}