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Restore CFTR Protein 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
-
Phase: ?more info
Phase One -
Study Sponsor: ?more info
Proteostasis -
Study Drugs:
Study Sites
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Closed to Enrollment
Alabama
University of Alabama at Birmingham, Birmingham, AL 35294
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Closed to Enrollment
Alaska
Providence Alaska Medical Center, Anchorage, AK 99508
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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
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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
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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
Beth Israel Medical Center, New York, NY 10003
-
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
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Closed to Enrollment
Ohio
Toledo Children's Hospital, Toledo, OH 43606
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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
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Closed to Enrollment
Oklahoma
Dr. Santiago Reyes Private Practice (Oklahoma City, Oklahoma), Oklahoma City, OK 73112
-
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 75708
-
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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