Ignite Creation Date:
2025-12-24 @ 1:00 PM
Ignite Modification Date:
2026-06-08 @ 3:25 PM
Study NCT ID:
NCT03587961
Status:
RECRUITING
Last Update Posted:
2025-10-03
First Post:
2018-07-03
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Personalized Theratyping Trial
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}], 'ancestors': [{'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000654124', 'term': 'tezacaftor, ivacaftor drug combination'}, {'id': 'C000599212', 'term': 'lumacaftor, ivacaftor drug combination'}, {'id': 'C545203', 'term': 'ivacaftor'}]}}, 'protocolSection': {'designModule': {'phases': ['EARLY_PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 20}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2019-08-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'completionDateStruct': {'date': '2026-09-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-09-29', 'studyFirstSubmitDate': '2018-07-03', 'studyFirstSubmitQcDate': '2018-07-03', 'lastUpdatePostDateStruct': {'date': '2025-10-03', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2018-07-16', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-01-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'spirometry', 'timeFrame': '32 weeks', 'description': 'change in lung function as measured via spirometry'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs.', 'detailedDescription': 'The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs. Symdeko (Tezacaftor/Ivacaftor), Orkambi (Ivacaftor and Lumacaftor), correctors of CFTR misfolding and Kalydeco (Ivacaftor), a potentiator of abnormal CFTR gating, will be explored as a treatment for patients with other CF mutations than those currently approved. Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Symdeko, or Orkambi, depending on the in vitro response pattern. Patients who have mutation response to a potentiator of CFTR function will be given Ivacaftor monotherapy. Patients with a mutation equivalent to wild type will be given Ivacaftor. If the patient is 6-12 years old, we will only study Orkambi or ivacaftor as symdeko is not yet FDA approved in this patient population.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of CF\n* Age ≥6 y.o.\n* CFTR mutation that may respond to approved correctors/potentiators in the opinion of the study investigators\n* Informed Consent/Assent\n* Stable CF pulmonary regimen\n\nExclusion Criteria:\n\n* Exacerbation requiring antibiotic or steroids for \\>28 days before trial entry\n* Ongoing participation in a CFTR modulator study\n* Active smoking in the past 6 months\n* History of solid organ transplant\n* Any condition which precludes the use of CFTR modulators: e.g. advanced cirrhosis, End-stage Renal Disease (ESRD)\n* Any condition that precludes the patient from participation in the opinion of the investigator\n* Any meds that have significant drug-drug interactions or any other off label use of CFTR modulators'}, 'identificationModule': {'nctId': 'NCT03587961', 'briefTitle': 'Personalized Theratyping Trial', 'organization': {'class': 'OTHER', 'fullName': 'University of Alabama at Birmingham'}, 'officialTitle': 'Personalized Theratyping Trial', 'orgStudyIdInfo': {'id': 'IRB-300001867'}, 'secondaryIdInfos': [{'id': 'IMPACT-Solomon', 'type': 'OTHER', 'domain': 'UAB'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Symdeko', 'description': 'Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Symdeko, depending on the in vitro response pattern', 'interventionNames': ['Drug: Symdeko']}, {'type': 'EXPERIMENTAL', 'label': 'Ivacaftor', 'description': 'Patients who have mutation response to a potentiator of CFTR function will be given Ivacaftor monotherapy.. Patients with a mutation equivalent to wild type will be given Ivacaftor.', 'interventionNames': ['Drug: Symdeko']}, {'type': 'EXPERIMENTAL', 'label': 'Orkambi', 'description': 'Patients who have a mutation that responds to a CFTR corrector from in vitro study will be given Orkambi, depending on the in vitro response pattern', 'interventionNames': ['Drug: Symdeko']}], 'interventions': [{'name': 'Symdeko', 'type': 'DRUG', 'otherNames': ['Orkambi', 'Ivacaftor'], 'description': 'explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not currently approved for these drugs.', 'armGroupLabels': ['Ivacaftor', 'Orkambi', 'Symdeko']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Heather Hathorne, PhD', 'role': 'CONTACT', 'email': 'hhathorne@peds.uab.edu', 'phone': '205-638-9568'}, {'name': 'Ginger Reeves, BS', 'role': 'CONTACT', 'email': 'greeves@peds.uab.edu', 'phone': '205-638-5970'}, {'name': 'George Solomon, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'University of Alabama at Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}], 'centralContacts': [{'name': 'Heather Hathorne, PhD', 'role': 'CONTACT', 'email': 'hhathorne@peds.uab.edu', 'phone': '205-638-9568'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'George Solomon', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Principal Investigator', 'investigatorFullName': 'George Solomon', 'investigatorAffiliation': 'University of Alabama at Birmingham'}}}}