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Trial record 2 of 6 for:    PTI-428

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03591094
Recruitment Status : Completed
First Posted : July 18, 2018
Last Update Posted : April 17, 2019
Sponsor:
Information provided by (Responsible Party):
Proteostasis Therapeutics, Inc.

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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: PTI-428 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : August 21, 2018
Actual Primary Completion Date : February 18, 2019
Actual Study Completion Date : February 18, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Active Comparator: PTI-428 dose level 1 Drug: PTI-428
Active

Active Comparator: PTI-428 dose level 2 Drug: PTI-428
Active

Placebo Comparator: Placebo PTI-428 Drug: Placebo
Placebo




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Maximum plasma concentration (Cmax) [ Time Frame: 28 days ]
  2. Time of Cmax (Tmax) [ Time Frame: 28 days ]
  3. Area under the concentration time curve from time 0 to time of last measurable concentration (AUC0-t) [ Time Frame: 28 days ]
  4. Change in FEV1 over time [ Time Frame: Baseline through Day 42 ]
  5. Change in sweat chloride over time [ Time Frame: Baseline through Day 42 ]

Other Outcome Measures:
  1. Change in nasal epithelial CFTR mRNA and protein expression over time [ Time Frame: Baseline through Day 42 ]
  2. Change in CFQ-R over time [ Time Frame: Baseline through Day 42 ]
  3. Cmax of PTI-428 metabolites, if applicable [ Time Frame: 28 days ]
  4. Tmax of PTI-428 metabolites, if applicable [ Time Frame: 28 days ]
  5. AUC0-t of PTI-428 metabolites, if applicable [ Time Frame: 28 days ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03591094


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Sponsors and Collaborators
Proteostasis Therapeutics, Inc.

Additional Information:
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Responsible Party: Proteostasis Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03591094     History of Changes
Other Study ID Numbers: PTI-428-06
First Posted: July 18, 2018    Key Record Dates
Last Update Posted: April 17, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases