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Restore CFTR Function Closed to Enrollment

Phase 1/2 study of PTI-801 drug in healthy adults and then in adults with cystic fibrosis (Proteostasis PTI-801-01)

This study is taking place at multiple care centers across the U.S. It will look at the safety and tolerability of the drug PTI-801.

 This study is placebo-controlled, meaning that some participants will receive PTI-801, and others will receive placebo. Researchers will test the safety and tolerability of PTI-801 by tracking adverse events and other changes in the body. They will also monitor how long PTI-801 stays in the body.This study is for people who have two copies of the F508del CFTR mutation. This study is not for people who have received an organ transplant. This study may require lung function tests, sweat tests, blood draws and/or other measurements.

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    18 Years and Older

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    40 to 90%

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

This study is enrolling three different groups of participants:

Group 1: participants must currently be taking lumacaftor/ivacaftor (Orkambi®)

Group 2:  participants must currently be taking tezacaftor/ivacaftor (Symdeko®)

Group 3:  participants must not be taking any CFTR modulator (Orkambi®, Symdeko®, or Kalydeco®) for at least 30 days prior to this study

Study Design

  • Study Type: ?more info

    Interventional

  • Randomized Study: ?more info

    Yes

  • Placebo Controlled: ?more info

    Yes

  • Length of Participation:

    30 days

  • Number of Study Visits:

    8

Additional Information

Study Sites

  • Closed to Enrollment

    Alabama

    University of Alabama at Birmingham, Birmingham, AL 35294

  • Closed to Enrollment

    Alaska

    Providence Alaska Medical Center, Anchorage, AK 99508

  • Closed to Enrollment

    California

    Stanford University Medical Center, Palo Alto, CA 94304

  • Closed to Enrollment

    Colorado

    National Jewish Health, Denver, CO 80206

  • Closed to Enrollment

    Florida

    Central Florida Pulmonary Group, Orlando, FL 32803

  • Closed to Enrollment

    Florida

    University of Florida, Gainesville, FL 32610

  • Closed to Enrollment

    Florida

    University of Miami, Miami, FL 33136

  • Closed to Enrollment

    Idaho

    Saint Luke's Cystic Fibrosis Center of Idaho, Boise, ID 83702

  • Closed to Enrollment

    Illinois

    OSF Saint Francis Medical Center, Peoria, IL 61637

  • Closed to Enrollment

    Illinois

    Northwestern University, Chicago, IL 60611

  • Closed to Enrollment

    Iowa

    University of Iowa, Iowa City, IA 52242

  • Closed to Enrollment

    Kentucky

    University of Louisville, Louisville, KY 40202

  • Closed to Enrollment

    Kentucky

    University of Louisville, Louisville, KY 40202

  • Closed to Enrollment

    Maine

    Maine Medical Center, Portland, ME 04102

  • Closed to Enrollment

    Massachusetts

    Boston Children's Hospital, Boston, MA 02115

  • Closed to Enrollment

    Massachusetts

    Massachusetts General Hospital, Boston, MA 02114

  • Closed to Enrollment

    Michigan

    University of Michigan, Michigan Medicine, Ann Arbor, MI 48109

  • Closed to Enrollment

    Missouri

    Children's Mercy Kansas City, Kansas City, MO 64108

  • Closed to Enrollment

    Nebraska

    University of Nebraska Medical Center, Omaha, NE 68198

  • Closed to Enrollment

    Nevada

    Adult Cystic Fibrosis Center of Southern Nevada, Las Vegas, NV 89109

  • Closed to Enrollment

    New York

    Columbia University Cystic Fibrosis Program, New York, NY 10032

  • Closed to Enrollment

    New York

    New York Medical College at Westchester Medical Center, Valhalla, NY 10595

  • Closed to Enrollment

    North Carolina

    Duke University Medical Center, Durham, NC 27710

  • Closed to Enrollment

    Ohio

    Toledo Children's Hospital, Toledo, OH 43606

  • Closed to Enrollment

    Ohio

    Children's Hospital Medical Center of Akron, Akron, OH 44308

  • Closed to Enrollment

    Ohio

    Nationwide Children's Hospital, Columbus, OH 43205

  • Closed to Enrollment

    Pennsylvania

    Children's Hospital of Philadelphia, Philadelphia, PA 19104

  • Closed to Enrollment

    Pennsylvania

    University of Pittsburgh Medical Center, Pittsburgh, PA 15224

  • Closed to Enrollment

    South Carolina

    Medical University of South Carolina, Charleston, SC 29425

  • Closed to Enrollment

    Texas

    University of Texas Health Center at Tyler, Tyler, TX 9038775271

  • Closed to Enrollment

    Utah

    Intermountain Cystic Fibrosis Center, University of Utah, Salt Lake City, UT 84132

  • Closed to Enrollment

    Virginia

    Virginia Commonwealth University, Richmond, VA 23219

Eligibility

See other primary eligibility criteria for more information.

  • Age:

    18 Years and Older

  • Mutation(s):

    Two Copies F508del

  • FEV1% Predicted:

    40 to 90%

For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.

Other Primary Eligibility Criteria

This study is enrolling three different groups of participants:

Group 1: participants must currently be taking lumacaftor/ivacaftor (Orkambi®)

Group 2:  participants must currently be taking tezacaftor/ivacaftor (Symdeko®)

Group 3:  participants must not be taking any CFTR modulator (Orkambi®, Symdeko®, or Kalydeco®) for at least 30 days prior to this study

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