Ignite Creation Date:
2025-12-25 @ 1:43 AM
Ignite Modification Date:
2025-12-26 @ 12:00 AM
Study NCT ID:
NCT03591094
Status:
COMPLETED
Last Update Posted:
2020-02-27
First Post:
2018-07-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis
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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 40}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-08-21', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-02', 'dispFirstSubmitDate': '2020-02-25', 'completionDateStruct': {'date': '2019-02-18', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-02-25', 'studyFirstSubmitDate': '2018-07-06', 'dispFirstSubmitQcDate': '2020-02-25', 'studyFirstSubmitQcDate': '2018-07-06', 'dispFirstPostDateStruct': {'date': '2020-02-27', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2020-02-27', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-07-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-02-18', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change in nasal epithelial CFTR mRNA and protein expression over time', 'timeFrame': 'Baseline through Day 42'}, {'measure': 'Change in CFQ-R over time', 'timeFrame': 'Baseline through Day 42'}, {'measure': 'Cmax of PTI-428 metabolites, if applicable', 'timeFrame': '28 days'}, {'measure': 'Tmax of PTI-428 metabolites, if applicable', 'timeFrame': '28 days'}, {'measure': 'AUC0-t of PTI-428 metabolites, if applicable', 'timeFrame': '28 days'}], 'primaryOutcomes': [{'measure': 'Number of subjects with treatment-emergent adverse events (TEAEs)', 'timeFrame': 'Baseline through Day 42', 'description': 'Safety and tolerability will be assessed by adverse events (AEs), safety labs, electrocardiograms (ECGs), physical examinations and vital signs.'}], 'secondaryOutcomes': [{'measure': 'Maximum plasma concentration (Cmax)', 'timeFrame': '28 days'}, {'measure': 'Time of Cmax (Tmax)', 'timeFrame': '28 days'}, {'measure': 'Area under the concentration time curve from time 0 to time of last measurable concentration (AUC0-t)', 'timeFrame': '28 days'}, {'measure': 'Change in FEV1 over time', 'timeFrame': 'Baseline through Day 42'}, {'measure': 'Change in sweat chloride over time', 'timeFrame': 'Baseline through Day 42'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'descriptionModule': {'briefSummary': 'The study population is comprised of adult subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation and are currently receiving background treatment with tezacaftor/ivacaftor for a minimum of 1 month prior to Day 1. The planned sample size is approximately 40 subjects. 20 subjects will be assigned to PTI-428 dose level 1 or placebo and 20 subjects will be assigned to PTI-428 dose level 2 or placebo. At each dose level, subjects will be randomized at a 3:1 randomization ratio. Subjects will receive once daily oral doses of PTI-428 or placebo for 28 days, while the subjects continue to receive background treatment with tezacaftor/ivacaftor per product label. The study drug administration period will be followed by a 14-day safety follow-up period.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Confirmed diagnosis of CF with the F508del/F508del genotype on record\n* On tezacaftor/ivacaftor dosing for both label indication and per label dosing for a minimum of 1 month on Day 1\n* Forced expiratory volume in 1 second (FEV1) 40-90% predicted, inclusive\n* Clinically stable with no significant changes in health status within 14 days of Day 1\n* Non-smoker and non-tobacco user for a minimum of 28 days prior to screening and for the duration of the study\n\nExclusion Criteria:\n\n* Participation in another clinical trial or treatment with an investigational agent within 28 days or 5 half-lives, whichever is longer, prior to Study Day 1\n* History of cancer within the past 5 years (excluding cervical cancer in situ with curative therapy for at least one year prior to screening and non-melanoma skin cancer)\n* History of organ transplantation\n* Hospitalization, sinopulmonary infection, CF exacerbation, or other clinically significant infection or illness (as determined by the investigator) requiring an increase or addition of medication, such as antibiotics or corticosteroids, within 14 days of Day 1\n* Initiation of any new chronic therapy (e.g., ibuprofen, hypertonic saline, azithromycin, Pulmozyme®, Cayston®, TOBI®)) or any change in chronic therapy (excluding pancreatic enzyme replacement therapy) within 28 days prior to Day 1\n* History or current evidence of alcohol or drug abuse or dependence within 12 months of screening as determined by the investigator\n* Pregnant or nursing women'}, 'identificationModule': {'nctId': 'NCT03591094', 'briefTitle': 'Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis', 'organization': {'class': 'INDUSTRY', 'fullName': 'Proteostasis Therapeutics, Inc.'}, 'officialTitle': 'A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect of PTI-428 in Subjects With Cystic Fibrosis', 'orgStudyIdInfo': {'id': 'PTI-428-06'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'PTI-428 dose level 1', 'interventionNames': ['Drug: PTI-428']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'PTI-428 dose level 2', 'interventionNames': ['Drug: PTI-428']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo PTI-428', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'PTI-428', 'type': 'DRUG', 'description': 'Active', 'armGroupLabels': ['PTI-428 dose level 1', 'PTI-428 dose level 2']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo', 'armGroupLabels': ['Placebo PTI-428']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'University of Alabama at Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '85724', 'city': 'Tucson', 'state': 'Arizona', 'country': 'United States', 'facility': 'University of Arizona', 'geoPoint': {'lat': 32.22174, 'lon': -110.92648}}, {'zip': '94305', 'city': 'Stanford', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}, {'zip': '80206', 'city': 'Denver', 'state': 'Colorado', 'country': 'United States', 'facility': 'National Jewish Health', 'geoPoint': {'lat': 39.73915, 'lon': -104.9847}}, {'zip': '32701', 'city': 'Altamonte Springs', 'state': 'Florida', 'country': 'United States', 'facility': 'Central Florida Pulmonary Group', 'geoPoint': {'lat': 28.66111, 'lon': -81.36562}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Northwestern University', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '61637', 'city': 'Peoria', 'state': 'Illinois', 'country': 'United States', 'facility': "Cystic Fibrosis Center, Children's Hospital of Illinois at OSF Saint Francis Medical Center", 'geoPoint': {'lat': 40.69365, 'lon': -89.58899}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'facility': 'University of Iowa, Roy J and Lucille A Carver College of Medicine', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '40202', 'city': 'Louisville', 'state': 'Kentucky', 'country': 'United States', 'facility': 'Universitey of Louisville, Kosair Charities Pediatric Clinical Research Unit', 'geoPoint': {'lat': 38.25424, 'lon': -85.75941}}, {'zip': '04102', 'city': 'Portland', 'state': 'Maine', 'country': 'United States', 'facility': 'Maine Medical Center', 'geoPoint': {'lat': 43.65737, 'lon': -70.2589}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts 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{'zip': '10003', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Mount Sinai Beth Israel', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10595', 'city': 'Valhalla', 'state': 'New York', 'country': 'United States', 'facility': 'New York Medical College', 'geoPoint': {'lat': 41.07482, 'lon': -73.77513}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University Medical Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '44308', 'city': 'Akron', 'state': 'Ohio', 'country': 'United States', 'facility': "Akron Children's Hospital", 'geoPoint': {'lat': 41.08144, 'lon': -81.51901}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Pittsburgh of UPMC", 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '29425', 'city': 'Charleston', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Medical University of South Carolina', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'zip': '75708', 'city': 'Tyler', 'state': 'Texas', 'country': 'United States', 'facility': 'The University of Texas Health Science Center at Tyler - Center for Clinical Research', 'geoPoint': {'lat': 32.35126, 'lon': -95.30106}}, {'zip': '84132', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Proteostasis Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}