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Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background Therapy

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: NCT03258424
Recruitment Status : Completed
First Posted : August 23, 2017
Last Update Posted : March 26, 2020
Sponsor:
Information provided by (Responsible Party):
Proteostasis Therapeutics, Inc.

Brief Summary:
The trial will consist of a single treatment group enrolling adult subjects with CF on background therapy with KALYDECO®. Approximately 16 subjects will be enrolled.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: PTI-428 Drug: Placebo Phase 1

Detailed Description:
The single treatment group will enroll adult subjects with CF currently on stable KALYDECO® background therapy for a minimum of 3 months at the time of randomization. Subjects will be randomized to either PTI-428 or placebo. Each dose will be administered once daily (QD) for a total of 14 Days. The subjects will continue treatment with KALYDECO® throughout the study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase I, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects With Cystic Fibrosis.
Actual Study Start Date : July 28, 2017
Actual Primary Completion Date : October 3, 2018
Actual Study Completion Date : October 3, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Active Comparator: PTI-428
Subjects will receive once daily dosing of PTI-428 or placebo for 14 days.
Drug: PTI-428
PTI-428 or placebo will be given QD for 14 days.

Placebo Comparator: Placebo
Subjects will receive once daily dosing of PTI-428 or placebo for 14 days.
Drug: Placebo
PTI-428 or placebo will be given QD for 14 days.




Primary Outcome Measures :
  1. Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs [ Time Frame: Baseline through Day 21 ]

Secondary Outcome Measures :
  1. t1/2 of multiple oral doses [ Time Frame: Baseline through Day 14 ]
  2. Tmax of multiple oral doses [ Time Frame: Baseline through Day 14 ]
  3. Cmax of multiple oral doses [ Time Frame: Baseline through Day 14 ]
  4. AUC0-t of multiple oral doses [ Time Frame: Baseline through Day 14 ]

Other Outcome Measures:
  1. Change in nasal epithelial mRNA and protein expression over time [ Time Frame: Baseline through Day 21 ]
  2. Change in sweat chloride over time [ Time Frame: Baseline through Day 21 ]
  3. Change in FEV1 over time [ Time Frame: Baseline through Day 21 ]
  4. Change in weight over time [ Time Frame: Baseline through Day 21 ]


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.
  • Stable on Kalydeco dosing for both label indication and per label dosing for a minimum of 3 months at the time of randomization
  • Forced expiratory volume in 1 second (FEV1) 40-90% predicted.
  • Non-smoker and non-tobacco user for a minimum of 30 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 30 days or 5 half-lives, whichever is longer, prior to Study Day 1.
  • 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).
  • History of organ transplantation.
  • 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.
  • History of alcohol or drug abuse or dependence within 12 months of screening as determined by the Investigator.
  • 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.
  • 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): NCT03258424


Locations
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United Kingdom
Celerion
Belfast, United Kingdom, BT9 6AD
Medicines Evaluation Unit
Manchester, United Kingdom
Sponsors and Collaborators
Proteostasis Therapeutics, Inc.
Additional Information:
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Responsible Party: Proteostasis Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03258424    
Other Study ID Numbers: PTI-428-05
First Posted: August 23, 2017    Key Record Dates
Last Update Posted: March 26, 2020
Last Verified: March 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes

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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