Ignite Creation Date:
2025-12-24 @ 1:41 PM
Ignite Modification Date:
2025-12-27 @ 9:52 PM
Study NCT ID:
NCT02718495
Status:
COMPLETED
Last Update Posted:
2019-03-21
First Post:
2016-03-10
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Czechia', 'United Kingdom']}, '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': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 56}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-07-19', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-03', 'completionDateStruct': {'date': '2017-11-28', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-03-19', 'studyFirstSubmitDate': '2016-03-10', 'studyFirstSubmitQcDate': '2016-03-18', 'lastUpdatePostDateStruct': {'date': '2019-03-21', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2016-03-24', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2017-11-28', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'SAD: change in nasal epithelial CFTR mRNA and protein expression', 'timeFrame': 'Baseline through Day 7'}, {'measure': 'MAD: change in nasal epithelial CFTR mRNA and protein expression', 'timeFrame': 'Baseline through Day 14'}, {'measure': 'MAD: change in sweat chloride over time', 'timeFrame': 'Baseline through Day 14'}, {'measure': 'Part B and Part C Cohorts 2 and 3: change in nasal epithelial CFTR mRNA and protein expression', 'timeFrame': 'Baseline through Day 35'}, {'measure': 'Part B and Part C Cohorts 2 and 3: change in CFQ-R over time', 'timeFrame': 'Baseline through Day 28'}, {'measure': 'Part C Cohort 1: change in nasal epithelial CFTR mRNA and protein expression', 'timeFrame': 'Baseline through Day 49'}, {'measure': 'Part C Cohort 1: change in CFQ-R over time', 'timeFrame': 'Baseline through Day 42'}, {'measure': 'Part C Cohort 3: change in fecal elastase over time', 'timeFrame': 'Baseline through Day 35'}, {'measure': 'Part C Cohort 3: change in fecal calprotectin over time', 'timeFrame': 'Baseline through Day 35'}], 'primaryOutcomes': [{'measure': 'SAD: safety and tolerability as assessed by adverse events, safety labs: hematology, chemistry, and urinalysis, electrocardiograms (ECGs), physical examinations, and vital signs', 'timeFrame': 'Baseline to Day 7'}, {'measure': 'MAD: safety and tolerability as assessed by adverse events, pulomonary function tests, safety labs: hematology, chemistry, and urinalysis, electrocardiograms (ECGs), physical examinations, and vital signs', 'timeFrame': 'Baseline to Day 14'}, {'measure': 'Part B and Part C Cohorts 2 and 3: safety and tolerability as assessed by adverse events, safety labs: hematology, chemistry, and urinalysis, electrocardiograms (ECGs), physical examinations, and vital signs', 'timeFrame': 'Baseline to Day 35'}, {'measure': 'Part C Cohort 1: safety and tolerability as assessed by adverse events, safety labs: hematology, chemistry, and urinalysis, electrocardiograms (ECGs), physical examinations, and vital signs', 'timeFrame': 'Baseline to Day 49'}], 'secondaryOutcomes': [{'measure': 'SAD: apparent terminal half-life (t1/2) of single oral dose', 'timeFrame': 'Baseline through 72 hours post dose'}, {'measure': 'SAD: time to reach maximum plasma concentration (Tmax) of single oral dose', 'timeFrame': 'Baseline through 72 hours post dose'}, {'measure': 'SAD: maximum plasma concentration (Cmax) of single oral dose', 'timeFrame': 'Baseline through 72 hours post dose'}, {'measure': 'SAD: area under the concentration-time curve from time 0 to time of last measurable concentration (AUC0-t) of single oral dose', 'timeFrame': 'Baseline through 72 hours post dose'}, {'measure': 'MAD: t1/2 of multiple oral doses', 'timeFrame': 'Baseline through 24 hours post Day 7 dose'}, {'measure': 'MAD: Tmax of multiple oral doses', 'timeFrame': 'Baseline through 24 hours post Day 7 dose'}, {'measure': 'MAD: Cmax of multiple oral doses', 'timeFrame': 'Baseline through 24 hours post Day 7 dose'}, {'measure': 'MAD: AUC0-t of multiple oral doses', 'timeFrame': 'Baseline through 24 hours post Day 7 dose'}, {'measure': 'MAD: area under the concentration-time curve from time 0 to infinity (AUC0-∞) of multiple oral doses', 'timeFrame': 'Baseline through 24 hours post Day 7 dose'}, {'measure': 'Part B and Part C Cohorts 2 and 3: t1/2 of multiple oral doses', 'timeFrame': 'Baseline through 24 hours post Day 28 dose'}, {'measure': 'Part B and Part C Cohorts 2 and 3: Tmax of multiple oral doses', 'timeFrame': 'Baseline through 24 hours post Day 28 dose'}, {'measure': 'Part B and Part C Cohorts 2 and 3: Cmax of multiple oral doses', 'timeFrame': 'Baseline through 24 hours post Day 28 dose'}, {'measure': 'Part B and Part C Cohorts 2 and 3: AUC0-t of multiple oral doses', 'timeFrame': 'Baseline through 24 hours post Day 28 dose'}, {'measure': 'Part B and Part C Cohorts 2 and 3: AUC0-∞ of multiple oral doses', 'timeFrame': 'Baseline through 24 hours post Day 28 dose'}, {'measure': 'Part B and Part C Cohorts 2 and 3: change in forced expiratory volume in one second (FEV1) over time', 'timeFrame': 'Baseline through Day 35'}, {'measure': 'Part B and Part C Cohorts 2 and 3: change in sweat chloride over time', 'timeFrame': 'Baseline through Day 35'}, {'measure': 'Part B and Part C Cohorts 2 and 3: change in weight over time', 'timeFrame': 'Baseline through Day 35'}, {'measure': 'Part C Cohort 1: t1/2 of multiple oral doses', 'timeFrame': 'Baseline through Day 42'}, {'measure': 'Part C Cohort 1: Tmax of multiple oral doses', 'timeFrame': 'Baseline through Day 42'}, {'measure': 'Part C Cohort 1: Cmax of multiple oral doses', 'timeFrame': 'Baseline through Day 42'}, {'measure': 'Part C Cohort 1: AUC0-t of multiple oral doses', 'timeFrame': 'Baseline through Day 42'}, {'measure': 'Part C Cohort 1: AUC0-∞ of multiple oral doses', 'timeFrame': 'Baseline through Day 42'}, {'measure': 'Part C Cohort 1: change in FEV1 over time', 'timeFrame': 'Baseline through Day 49'}, {'measure': 'Part C Cohort 1: change in sweat chloride over time', 'timeFrame': 'Baseline through Day 49'}, {'measure': 'Part C Cohort 1: change in weight over time', 'timeFrame': 'Baseline through Day 49'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'descriptionModule': {'briefSummary': 'This trial will consist of three arms: Part A, Part B, and Part C. Part A has two groups. The first group will enroll adult subjects with cystic fibrosis (CF) into a single ascending dose (SAD) treatment group. The second group will enroll adult subjects with CF, including those on background treatment with ORKAMBI® and those not on a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, into a multiple ascending dose (MAD) treatment group. Part B will enroll adult subjects with CF currently on stable ORKAMBI® background therapy for a minimum of 3 months into a Phase II treatment group consisting of two cohorts. Part C will enroll adult subjects with CF, including those on background treatment with KALYDECO® and those not on a CFTR modulator, into a Phase II treatment group consisting of three cohorts. Approximately 136 subjects will be enrolled.', 'detailedDescription': 'PART A The SAD treatment group is comprised of 3 cohorts where subjects will be randomized to either PTI-428 or placebo. Following the conclusion of at least 3 SAD treatment groups, a set of adult subjects diagnosed with CF will participate in an assigned MAD treatment group. The MAD treatment group is comprised of 3 cohorts. MAD Cohort 1 will enroll adult subjects with CF currently on stable ORKAMBI® background therapy for a minimum of 3 months at the time of randomization. MAD Cohorts 2 and 3 will enroll adult subjects with CF who are not currently on any background therapies. Subjects in all MAD cohorts will be randomized to either PTI-428 or placebo. Each dose will be administered once daily (QD) for a total of 7 Days.\n\nPART B Following the conclusion of MAD Cohort 1, a set of adult subjects diagnosed with CF currently on stable ORKAMBI® background therapy for a minimum of 3 months will participate in Part B. The Part B Phase II treatment group is comprised of 2 cohorts where subjects will be randomized to either PTI-428 or placebo. Each dose will be administered QD for a total of 28 days.\n\nPART C Following the conclusion of Part B Phase II, a set of adult subjects diagnosed with CF will participate in Part C. The Part C Phase II treatment group is comprised of 3 cohorts. Part C Cohort 1 will enroll adult subjects with CF who are eligible to take, but not currently taking, ORKAMBI® in accordance with the approved label. Part C Cohort 2 will enroll adult subjects with CF currently on stable KALYDECO® background therapy for a minimum of 3 months at the time of randomization. Part C Cohort 3 will enroll adult subjects with CF who are not currently on any background therapies and are pancreatic sufficient. Each PTI-428 or placebo dose will be administered QD for a total of 28 days.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Confirmed diagnosis of CF.\n* Forced expiratory volume in 1 second (FEV1) 40-90% predicted.\n* Non-smoker and non-tobacco user for a minimum of 30 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 30 days or 5 half-lives, whichever is longer, prior to Study Day 1.\n* History of cancer within the past five years (excluding cervical CIS with curative therapy for at least one year prior to screening and non-melanoma skin cancer).\n* History of organ transplantation.\n* Any sinopulmonary infection or CF exacerbation requiring a change or addition of medication (including antibiotics) within 1 month of Study Day 1 or any other clinically significant infection as determined by the investigator within 1 month of Day 1.\n* History of alcohol or drug abuse or dependence within 12 months of screening as determined by the Investigator.\n* Male and female of child-bearing potential, unless they are using highly effective methods of contraception during participation in the clinical study and for 4 weeks after termination from study.\n* Pregnant or nursing women.'}, 'identificationModule': {'nctId': 'NCT02718495', 'briefTitle': 'Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis', 'organization': {'class': 'INDUSTRY', 'fullName': 'Proteostasis Therapeutics, Inc.'}, 'officialTitle': 'A Phase I/II, Multi-center, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects With Cystic Fibrosis', 'orgStudyIdInfo': {'id': 'PTI-428-01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'Part A', 'description': 'Part A consists of two treatment groups, SAD and MAD. Both treatment groups will consist of 3 cohorts. In SAD, subjects will receive a single dose of PTI-428 or placebo. In MAD, subjects will receive once daily dosing of PTI-428 or placebo for 7 days.', 'interventionNames': ['Drug: PTI-428', 'Drug: Placebo']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Part B', 'description': 'Part B will consist of 2 cohorts. Subjects will receive once daily dosing of PTI-428 or placebo for 28 days.', 'interventionNames': ['Drug: PTI-428', 'Drug: Placebo']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Part C', 'description': 'Part C will consist of 3 cohorts. Subjects will receive once daily dosing of PTI-428 or placebo for 28 days.', 'interventionNames': ['Drug: PTI-428', 'Drug: Placebo']}], 'interventions': [{'name': 'PTI-428', 'type': 'DRUG', 'armGroupLabels': ['Part A', 'Part B', 'Part C']}, {'name': 'Placebo', 'type': 'DRUG', 'armGroupLabels': ['Part A', 'Part B', 'Part C']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94305', 'city': 'Stanford', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University Medical Center', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}, {'zip': '32803', 'city': 'Altamonte Springs', 'state': 'Florida', 'country': 'United States', 'facility': 'Central Florida Pulmonary Group', 'geoPoint': {'lat': 28.66111, 'lon': -81.36562}}, {'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida College of Medicine', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '83712', 'city': 'Boise', 'state': 'Idaho', 'country': 'United States', 'facility': "St. Luke's Cystic Fibrosis Center of Idaho", 'geoPoint': {'lat': 43.6135, 'lon': -116.20345}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Northwestern University Memorial Hospital', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'facility': 'University of Iowa', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'country': 'United States', 'facility': 'University of Kansas Medical Center Research Institute, Inc.', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '66211', 'city': 'Overland Park', 'state': 'Kansas', 'country': 'United States', 'facility': 'Quintiles Overland Park Phase 1 Unit', 'geoPoint': {'lat': 38.98223, 'lon': -94.67079}}, {'zip': '40202', 'city': 'Louisville', 'state': 'Kentucky', 'country': 'United States', 'facility': 'University of Louisville', 'geoPoint': {'lat': 38.25424, 'lon': -85.75941}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Childrens Hospital Boston', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '48109', 'city': 'Ann Arbor', 'state': 'Michigan', 'country': 'United States', 'facility': 'Universiy of Michigan Health System', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}, {'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Mayo Clinic', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}, {'zip': '89107', 'city': 'Las Vegas', 'state': 'Nevada', 'country': 'United States', 'facility': "Children's Lung Specialists", 'geoPoint': {'lat': 36.17497, 'lon': -115.13722}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University Health System', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '73104', 'city': 'Oklahoma City', 'state': 'Oklahoma', 'country': 'United States', 'facility': 'University of Oklahoma Health Sciences Center', 'geoPoint': {'lat': 35.46756, 'lon': -97.51643}}, {'zip': '17033', 'city': 'Hershey', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Penn State Milton S. Hershey Medical Center', 'geoPoint': {'lat': 40.28592, 'lon': -76.65025}}, {'zip': '19107', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Drexel University College of Medicine', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '29425', 'city': 'Charleston', 'state': 'South Carolina', 'country': 'United States', 'facility': 'Medical University of South Carolina', 'geoPoint': {'lat': 32.77632, 'lon': -79.93275}}, {'zip': '37232', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Vanderbilt University Medical Center', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': 'V6Z 1Y6', 'city': 'Vancouver', 'state': 'British Columbia', 'country': 'Canada', 'facility': "St. Paul's Hospital Pacific Lung Research Center", 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'zip': 'M5B 1W8', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': "St. Michael's Hospital", 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': 'H2X 0A9', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': 'Institut de Recherches Cliniques de Montreal', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'zip': 'G1V 4G5', 'city': 'Québec', 'state': 'Quebec', 'country': 'Canada', 'facility': 'Institut Universitaire de Cardiologie et de Pneumologie de Quebec', 'geoPoint': {'lat': 46.81228, 'lon': -71.21454}}, {'zip': '2100', 'city': 'Copenhagen', 'country': 'Denmark', 'facility': 'University of Copenhagen Rigshospitalet', 'geoPoint': {'lat': 55.67594, 'lon': 12.56553}}, {'zip': '33076', 'city': 'Bordeaux', 'country': 'France', 'facility': 'Groupe Hospitalier Pellegrin - Hôpital des Enfants', 'geoPoint': {'lat': 44.84124, 'lon': -0.58046}}, {'zip': '75014', 'city': 'Paris', 'country': 'France', 'facility': 'Hôpital Cochin', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '10117', 'city': 'Berlin', 'country': 'Germany', 'facility': 'Charite - Campus Virchow-Klinikum', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'zip': '60590', 'city': 'Frankfurt', 'country': 'Germany', 'facility': 'Universitaetsklinikum Frankfurt-Zentrum der Inneren Medizin', 'geoPoint': {'lat': 49.68333, 'lon': 10.53333}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Proteostasis Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}