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

Completed · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

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.

Key facts

Study ID

NCT02718495

Run by

Proteostasis Therapeutics, Inc.

People needed

56

Starts

2016-07-19

Expected to finish

2017-11-28

Last updated by the study team

2019-03-21

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed diagnosis of CF.
• 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.

You may not qualify if…

• 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.

Where it is running

• Massachusetts General Hospital — Boston, Massachusetts, United States
• Childrens Hospital Boston — Boston, Massachusetts, United States
• Universiy of Michigan Health System — Ann Arbor, Michigan, United States
• Mayo Clinic — Rochester, Minnesota, United States
• Children's Lung Specialists — Las Vegas, Nevada, United States
• Duke University Health System — Durham, North Carolina, United States
• University of Oklahoma Health Sciences Center — Oklahoma City, Oklahoma, United States
• Penn State Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States
• Drexel University College of Medicine — Philadelphia, Pennsylvania, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• St. Paul's Hospital Pacific Lung Research Center — Vancouver, British Columbia, Canada
• St. Michael's Hospital — Toronto, Ontario, Canada
• Institut de Recherches Cliniques de Montreal — Montreal, Quebec, Canada
• Institut Universitaire de Cardiologie et de Pneumologie de Quebec — Québec, Quebec, Canada
• University of Copenhagen Rigshospitalet — Copenhagen, Denmark
• Groupe Hospitalier Pellegrin - Hôpital des Enfants — Bordeaux, France
• Hôpital Cochin — Paris, France
• Charite - Campus Virchow-Klinikum — Berlin, Germany
• Universitaetsklinikum Frankfurt-Zentrum der Inneren Medizin — Frankfurt, Germany
• Stanford University Medical Center — Stanford, California, United States
• Central Florida Pulmonary Group — Altamonte Springs, Florida, United States
• University of Florida College of Medicine — Gainesville, Florida, United States
• St. Luke's Cystic Fibrosis Center of Idaho — Boise, Idaho, United States
• Northwestern University Memorial Hospital — Chicago, Illinois, United States

Full record on ClinicalTrials.gov

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