Ignite Creation Date:
2025-12-25 @ 2:39 AM
Ignite Modification Date:
2025-12-31 @ 2:22 PM
Study NCT ID:
NCT05994534
Status:
RECRUITING
Last Update Posted:
2025-12-17
First Post:
2023-08-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
PK and PD Study of NPI-001 and Cysteamine Bitartrate
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003554', 'term': 'Cystinosis'}], 'ancestors': [{'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D003543', 'term': 'Cysteamine'}, {'id': 'C487056', 'term': 'N-Acetylcysteinamide'}], 'ancestors': [{'id': 'D008624', 'term': 'Mercaptoethylamines'}, {'id': 'D005021', 'term': 'Ethylamines'}, {'id': 'D000588', 'term': 'Amines'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D013438', 'term': 'Sulfhydryl Compounds'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 12}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-10-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2026-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-16', 'studyFirstSubmitDate': '2023-08-08', 'studyFirstSubmitQcDate': '2023-08-08', 'lastUpdatePostDateStruct': {'date': '2025-12-17', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2023-08-16', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Concentration of Cystine Levels Over Time', 'timeFrame': '1 day', 'description': 'Cystine concentration over 6 hours'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cystinosis']}, 'descriptionModule': {'briefSummary': 'Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.', 'detailedDescription': 'This study will examine the safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients, aged ≥ 10 years. The ability of NPI-001 to reduce cystine will be assessed and compared with cysteamine.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '10 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Males or females, any race, ≥ 10 years of age.\n2. Diagnosis of nephropathic cystinosis and able to cease cysteamine therapy for 2 days.\n3. Females will be nonpregnant and nonlactating, and females of childbearing potential and males will agree to use contraception as detailed in the protocol.\n4. Able to comprehend and willing to sign an informed consent /assent form and to abide by the study restrictions (travel as necessary, clinical phase 1 unit or similar for up to 3 days).\n\nExclusion Criteria:\n\n1. Have undergone kidney transplantation.\n2. Are receiving dialysis treatment.\n3. History of significant hypersensitivity to NAC or any ingredient of NPI-001 oral solution.\n4. Participation in a clinical study involving administration of an investigational drug (new chemical entity) in the 30 days prior to Day 1.\n5. Inability to provide blood samples, including difficulty with venous access.\n6. Subjects who, in the opinion of the Investigator and/or Sponsor (or designee), should not participate in this study.'}, 'identificationModule': {'nctId': 'NCT05994534', 'acronym': 'INCA', 'briefTitle': 'PK and PD Study of NPI-001 and Cysteamine Bitartrate', 'organization': {'class': 'INDUSTRY', 'fullName': 'Nacuity Pharmaceuticals, Inc.'}, 'officialTitle': 'A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients', 'orgStudyIdInfo': {'id': 'C-23-01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'cysteamine', 'description': 'Single dose, tablets in current treatment dose', 'interventionNames': ['Drug: Cysteamine Bitartrate']}, {'type': 'EXPERIMENTAL', 'label': 'NPI-001', 'description': 'Single dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose.', 'interventionNames': ['Drug: N-Acetylcysteine Amide']}], 'interventions': [{'name': 'Cysteamine Bitartrate', 'type': 'DRUG', 'otherNames': ['Cystagon'], 'description': 'Single dose, tablets at current therapeutic dose', 'armGroupLabels': ['cysteamine']}, {'name': 'N-Acetylcysteine Amide', 'type': 'DRUG', 'otherNames': ['NPI-001'], 'description': 'Single dose, oral solution', 'armGroupLabels': ['NPI-001']}]}, 'contactsLocationsModule': {'locations': [{'zip': '2145', 'city': 'Westmead', 'state': 'New South Wales', 'status': 'RECRUITING', 'country': 'Australia', 'contacts': [{'name': 'Hugh McCarthy', 'role': 'CONTACT', 'email': 'hugh.mccarthy@health.nsw.gov.au'}], 'facility': "Children's Hospital at Westmead", 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}], 'centralContacts': [{'name': 'Jami Kern, PhD', 'role': 'CONTACT', 'email': 'info@nacuity.com', 'phone': '+1-817-336-3000'}], 'overallOfficials': [{'name': 'Hugh McCarthy, PhD, FRACP', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Sydney Children's Health Network"}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO', 'description': 'Individual participant data will not be made available to other researchers.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Nacuity Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}