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Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT03591094
Recruitment Status : Completed
First Posted : July 18, 2018
Last Update Posted : February 27, 2020
Sponsor:
Information provided by (Responsible Party):
Proteostasis Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE July 6, 2018
First Posted Date  ICMJE July 18, 2018
Last Update Posted Date February 27, 2020
Actual Study Start Date  ICMJE August 21, 2018
Actual Primary Completion Date February 18, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 6, 2018)
Number of subjects with treatment-emergent adverse events (TEAEs) [ Time Frame: Baseline through Day 42 ]
Safety and tolerability will be assessed by adverse events (AEs), safety labs, electrocardiograms (ECGs), physical examinations and vital signs.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 6, 2018)
  • Maximum plasma concentration (Cmax) [ Time Frame: 28 days ]
  • Time of Cmax (Tmax) [ Time Frame: 28 days ]
  • Area under the concentration time curve from time 0 to time of last measurable concentration (AUC0-t) [ Time Frame: 28 days ]
  • Change in FEV1 over time [ Time Frame: Baseline through Day 42 ]
  • Change in sweat chloride over time [ Time Frame: Baseline through Day 42 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: July 6, 2018)
  • Change in nasal epithelial CFTR mRNA and protein expression over time [ Time Frame: Baseline through Day 42 ]
  • Change in CFQ-R over time [ Time Frame: Baseline through Day 42 ]
  • Cmax of PTI-428 metabolites, if applicable [ Time Frame: 28 days ]
  • Tmax of PTI-428 metabolites, if applicable [ Time Frame: 28 days ]
  • AUC0-t of PTI-428 metabolites, if applicable [ Time Frame: 28 days ]
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis
Official Title  ICMJE 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
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Drug: PTI-428
    Active
  • Drug: Placebo
    Placebo
Study Arms  ICMJE
  • Active Comparator: PTI-428 dose level 1
    Intervention: Drug: PTI-428
  • Active Comparator: PTI-428 dose level 2
    Intervention: Drug: PTI-428
  • Placebo Comparator: Placebo PTI-428
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 6, 2018)
40
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE February 18, 2019
Actual Primary Completion Date February 18, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Confirmed diagnosis of CF with the F508del/F508del genotype on record
  • On tezacaftor/ivacaftor dosing for both label indication and per label dosing for a minimum of 1 month on Day 1
  • Forced expiratory volume in 1 second (FEV1) 40-90% predicted, inclusive
  • Clinically stable with no significant changes in health status within 14 days of Day 1
  • Non-smoker and non-tobacco user for a minimum of 28 days prior to screening and for the duration of the study

Exclusion Criteria:

  • 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
  • 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)
  • History of organ transplantation
  • 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
  • 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
  • History or current evidence of alcohol or drug abuse or dependence within 12 months of screening as determined by the investigator
  • Pregnant or nursing women
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03591094
Other Study ID Numbers  ICMJE PTI-428-06
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Proteostasis Therapeutics, Inc.
Study Sponsor  ICMJE Proteostasis Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Proteostasis Therapeutics, Inc.
Verification Date February 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP