Ignite Creation Date:
2025-12-26 @ 10:36 AM
Ignite Modification Date:
2026-01-01 @ 12:56 AM
Study NCT ID:
NCT00004428
Status:
COMPLETED
Last Update Posted:
2015-03-25
First Post:
1999-10-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase I Randomized Study of CPX for the Treatment of Adult Patients With Mild Cystic Fibrosis
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D035583', 'term': 'Rare Diseases'}], 'ancestors': [{'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE'}, 'primaryPurpose': 'TREATMENT'}, 'enrollmentInfo': {'count': 35}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '1997-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2000-01', 'completionDateStruct': {'date': '1999-10'}, 'lastUpdateSubmitDate': '2015-03-24', 'studyFirstSubmitDate': '1999-10-18', 'studyFirstSubmitQcDate': '1999-10-18', 'lastUpdatePostDateStruct': {'date': '2015-03-25', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '1999-10-19', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ['cardiovascular and respiratory diseases', 'cystic fibrosis', 'genetic diseases and dysmorphic syndromes', 'rare disease'], 'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '37983082', 'type': 'DERIVED', 'citation': 'Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.'}, {'pmid': '33331662', 'type': 'DERIVED', 'citation': 'Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.'}]}, 'descriptionModule': {'briefSummary': 'OBJECTIVES: I. Evaluate the safety of ascending doses of CPX administered to adult patients with mild cystic fibrosis.\n\nII. Evaluate the pharmacokinetics of ascending doses of CPX in this patient population.', 'detailedDescription': 'PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled, dose escalation study.\n\nThere are 7 experimental cohorts, each treated with a different oral dose of CPX or placebo. Within each cohort, 4 patients receive a single dose of CPX and 1 patient receives placebo. Each patient is monitored 24 hours postdose. Escalation to the next dose level for each subsequent cohort begins only after the safety data obtained from the previous cohort is reviewed and found not to limit dose escalation.\n\nAll patients return for a follow up evaluation 1 week after dosing.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'PROTOCOL ENTRY CRITERIA:\n\n* Mild cystic fibrosis\n* Not pregnant or nursing Negative pregnancy test'}, 'identificationModule': {'nctId': 'NCT00004428', 'briefTitle': 'Phase I Randomized Study of CPX for the Treatment of Adult Patients With Mild Cystic Fibrosis', 'organization': {'class': 'FED', 'fullName': 'FDA Office of Orphan Products Development'}, 'orgStudyIdInfo': {'id': '199/13364'}, 'secondaryIdInfos': [{'id': 'SCICLONE-FDA-OP-97-1'}]}, 'armsInterventionsModule': {'interventions': [{'name': 'CPX', 'type': 'DRUG'}]}, 'contactsLocationsModule': {'overallOfficials': [{'name': 'Eduardo Martins', 'role': 'STUDY_CHAIR', 'affiliation': 'SciClone Pharmaceuticals'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'FDA Office of Orphan Products Development', 'class': 'FED'}, 'collaborators': [{'name': 'SciClone Pharmaceuticals', 'class': 'INDUSTRY'}]}}}