Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis

Description

This trial will consist of two parts: Part 1 and Part 2. Part 1 will enroll adult healthy volunteers (HV) into four treatment groups. The first group will enroll HV into a single ascending dose (SAD) treatment group consisting of three cohorts. The second group will enroll HV into a multiple ascending dose (MAD) treatment group consisting of three cohorts. The third group will enroll HV into a food effect (FE) treatment group consisting of one cohort. The fourth group will enroll HV into a drug-drug interactions (DDI) treatment group consisting of one cohort. Part 2 will enroll adult subjects with cystic fibrosis (CF) currently on stable ivacaftor/lumacaftor background therapy for a minimum of three months into a MAD treatment group consisting of three cohorts. Approximately 120 subjects will be enrolled.

Study Start Date

April, 10 2017

Estimated Completion Date

July 2018

Interventions

  • Drug: PTI-801
  • Drug: Talc

Study ID

Proteostasis Therapeutics, Inc. -- PTI-801-01

Status

Unknown

Trial ID

NCT03140527

Study Type

Interventional

Trial Phase

Phase 1

Enrollment Quota

120

Sponsor

Proteostasis Therapeutics, Inc.

Part 1

Inclusion Criteria

  • Adults age 18 to 55 years old, inclusive, at the time of informed consent.
  • Body mass index (BMI) ?18 to <30 kg/m2.
  • Subject must be a nonsmoker and a nontobacco user for a minimum of 30 days prior to screening and for the duration of the study. Part 1

Exclusion Criteria

  • History or current evidence of any clinically significant cardiac, endocrinologic,hematologic, hepatobiliary, immunologic, metabolic, urologic, pulmonary, neurologic,dermatologic, psychiatric, renal, or other major disease, as determined by the investigator.
  • Presence of prolonged QT/ Corrected QT Interval (QTc) interval with Fridericia's correction formula (QTcF) >450 msec at screening.
  • Abnormal liver function as defined by aspartate aminotransferase (AST), alanine aminotransferase (ALT) or bilirubin > upper limit of the normal range.
  • Abnormal renal function at screening defined as: Creatinine clearance <80 mL/min using the Cockcroft-Gault equation.
  • 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 5 years (excluding nonmelanoma skin cancer).
  • History or current evidence of alcohol or drug abuse or dependence within 12 months of screening as determined by the investigator.
  • Positive urine screen for prohibited drugs (cocaine, cannabinoids, nicotine [urine cotinine is the detection mechanism for nicotine], opiates, barbiturates, amphetamines, and benzodiazepines) or positive alcohol test at screening.
  • Positive blood screen for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or hepatitis C virus antibody (HCVAb).
  • Has donated blood within 3 months of screening or plans to donate blood within 3 months of study completion. Part 1 HV DDI Cohort Additional

Exclusion Criteria

  • Concomitant use of known strong or moderate inhibitors or inducers of CYP1A2, CYP2B6, and CYP3A4 within 14 days or 5 half-lives (whichever is longer) prior to Day 1 and through the last PK sampling point on Day 20
  • Use of grapefruit
  • or Seville orange-containing products within 48 hours prior to Day 1 and through the last PK sampling point on Day 20
  • Use of alcohol
  • or caffeine-containing products within 48 hours prior to Day 1 and through the last PK sampling point on Day 20 Part 2

    Inclusion Criteria

    • Confirmed diagnosis of CF with the F508del/F508del genotype on record, along with clinical findings consistent with CF such as chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities
    • Stable on ivacaftor/lumacaftor 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, inclusive
    • Non-smoker and non-tobacco user for a minimum of 30 days prior to screening Part 2

    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

Gender

All

Ages

18 Years and older

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Study Locations and Contact Information (116)

Study Location Distance Name Phone Email
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Robert Fowler None robert.fowler@childrens.harvard.edu
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Robert Fowler None robert.fowler@childrens.harvard.edu
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Robert Fowler None robert.fowler@childrens.harvard.edu
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles Robert Fowler None robert.fowler@childrens.harvard.edu
Maine Medical Center - Portland, Maine 99.0 miles Harmony Renna 207-662-5917 rennah@mmc.org
Maine Medical Center - Portland, Maine 99.0 miles Harmony Renna 207-662-5917 rennah@mmc.org
Maine Medical Center - Portland, Maine 99.0 miles Harmony Renna 207-662-5917 rennah@mmc.org
Maine Medical Center - Portland, Maine 99.0 miles Harmony Renna 207-662-5917 rennah@mmc.org
New York Medical College - Valhalla, New York 164.0 miles La Toya Holness 914-593-8904 latoya_holness@bchphysicians.org
New York Medical College - Valhalla, New York 164.0 miles La Toya Holness 914-593-8904 latoya_holness@bchphysicians.org
New York Medical College - Valhalla, New York 164.0 miles La Toya Holness 914-593-8904 latoya_holness@bchphysicians.org
New York Medical College - Valhalla, New York 164.0 miles La Toya Holness 914-593-8904 latoya_holness@bchphysicians.org
Columbia University Medical Center - New York, New York 187.1 miles Kristina Rivera 212-305-4675 klr2142@columbia.edu
Columbia University Medical Center - New York, New York 187.1 miles Kristina Rivera 212-305-4675 klr2142@columbia.edu
Columbia University Medical Center - New York, New York 187.1 miles Kristina Rivera 212-305-4675 klr2142@columbia.edu
Columbia University Medical Center - New York, New York 187.1 miles Kristina Rivera 212-305-4675 klr2142@columbia.edu
Mount Sinai Beth Israel - New York, New York 187.6 miles Anne Kukral PAC None Anne.Kukral@mountsinai.org
Mount Sinai Beth Israel - New York, New York 187.6 miles Anne Kukral PAC None Anne.Kukral@mountsinai.org
Mount Sinai Beth Israel - New York, New York 187.6 miles Anne Kukral PAC None Anne.Kukral@mountsinai.org
Mount Sinai Beth Israel - New York, New York 187.6 miles Anne Kukral PAC None Anne.Kukral@mountsinai.org
Virginia Commonwealth University - Richmond, Virginia 473.4 miles Ryan Hayden 804-628-3921 ryan.hayden@vcuhealth.org
Virginia Commonwealth University - Richmond, Virginia 473.4 miles Ryan Hayden 804-628-3921 ryan.hayden@vcuhealth.org
Virginia Commonwealth University - Richmond, Virginia 473.4 miles Ryan Hayden 804-628-3921 ryan.hayden@vcuhealth.org
Virginia Commonwealth University - Richmond, Virginia 473.4 miles Ryan Hayden 804-628-3921 ryan.hayden@vcuhealth.org
Childrens Hospital of Pittsburgh of UPMC - Pittsburgh, Pennsylvania 487.9 miles Elizabeth Hartigan MPH RN CRM 412-692-7060 elizabeth.hartigan@chp.edu
Childrens Hospital of Pittsburgh of UPMC - Pittsburgh, Pennsylvania 487.9 miles Elizabeth Hartigan MPH RN CRM 412-692-7060 elizabeth.hartigan@chp.edu
Childrens Hospital of Pittsburgh of UPMC - Pittsburgh, Pennsylvania 487.9 miles Elizabeth Hartigan MPH RN CRM 412-692-7060 elizabeth.hartigan@chp.edu
Childrens Hospital of Pittsburgh of UPMC - Pittsburgh, Pennsylvania 487.9 miles Elizabeth Hartigan MPH RN CRM 412-692-7060 elizabeth.hartigan@chp.edu
Akron Childrens Hospital - Akron, Ohio 545.3 miles Deborah Ouellette 330-543-3089 douellette@chmca.org
Akron Childrens Hospital - Akron, Ohio 545.3 miles Deborah Ouellette 330-543-3089 douellette@chmca.org
Akron Childrens Hospital - Akron, Ohio 545.3 miles Deborah Ouellette 330-543-3089 douellette@chmca.org
Akron Childrens Hospital - Akron, Ohio 545.3 miles Deborah Ouellette 330-543-3089 douellette@chmca.org
Duke University Health System - Durham, North Carolina 609.0 miles Noely MartinezOverby None noely.martinez-overby@duke.edu
Duke University Health System - Durham, North Carolina 609.0 miles Noely MartinezOverby None noely.martinez-overby@duke.edu
Duke University Health System - Durham, North Carolina 609.0 miles Noely MartinezOverby None noely.martinez-overby@duke.edu
Duke University Health System - Durham, North Carolina 609.0 miles Noely MartinezOverby None noely.martinez-overby@duke.edu
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles Terri Johnson 614-722-4756 Terri.Johnson@nationwidechildrens.org
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles Terri Johnson 614-722-4756 Terri.Johnson@nationwidechildrens.org
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles Terri Johnson 614-722-4756 Terri.Johnson@nationwidechildrens.org
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles Terri Johnson 614-722-4756 Terri.Johnson@nationwidechildrens.org
Toledo Childrens Hospital - Toledo, Ohio 644.5 miles Kelly Houser RN 419-291-4630 kelly.houser@promedica.org
Toledo Childrens Hospital - Toledo, Ohio 644.5 miles Kelly Houser RN 419-291-4630 kelly.houser@promedica.org
Toledo Childrens Hospital - Toledo, Ohio 644.5 miles Kelly Houser RN 419-291-4630 kelly.houser@promedica.org
Toledo Childrens Hospital - Toledo, Ohio 644.5 miles Kelly Houser RN 419-291-4630 kelly.houser@promedica.org
University of Michigan Health System - Ann Arbor, Michigan 645.2 miles Dawn Kruse CCRC 734-615-3266 dmkruse@med.umich.edu
University of Michigan Health System - Ann Arbor, Michigan 645.2 miles Dawn Kruse CCRC 734-615-3266 dmkruse@med.umich.edu
University of Michigan Health System - Ann Arbor, Michigan 645.2 miles Dawn Kruse CCRC 734-615-3266 dmkruse@med.umich.edu
University of Michigan Health System - Ann Arbor, Michigan 645.2 miles Dawn Kruse CCRC 734-615-3266 dmkruse@med.umich.edu
Medical University of South Carolina - Charleston, South Carolina 821.2 miles Ashley Warden 843-792-4349 jonesash@musc.edu
Medical University of South Carolina - Charleston, South Carolina 821.2 miles Ashley Warden 843-792-4349 jonesash@musc.edu
Medical University of South Carolina - Charleston, South Carolina 821.2 miles Ashley Warden 843-792-4349 jonesash@musc.edu
Medical University of South Carolina - Charleston, South Carolina 821.2 miles Ashley Warden 843-792-4349 jonesash@musc.edu
University of Louisville - Louisville, Kentucky 823.2 miles Trish Aponte 502-629-5539 trish.aponte@louisville.edu
University of Louisville - Louisville, Kentucky 823.2 miles Trish Aponte 502-629-5539 trish.aponte@louisville.edu
University of Louisville - Louisville, Kentucky 823.2 miles Trish Aponte 502-629-5539 trish.aponte@louisville.edu
University of Louisville - Louisville, Kentucky 823.2 miles Trish Aponte 502-629-5539 trish.aponte@louisville.edu
Northwestern University Memorial Hospital - Chicago, Illinois 847.9 miles Rachel Nelson MS MPH 312-695-0415 rachel.nelson@northwestern.edu
Northwestern University Memorial Hospital - Chicago, Illinois 847.9 miles Rachel Nelson MS MPH 312-695-0415 rachel.nelson@northwestern.edu
Northwestern University Memorial Hospital - Chicago, Illinois 847.9 miles Rachel Nelson MS MPH 312-695-0415 rachel.nelson@northwestern.edu
Northwestern University Memorial Hospital - Chicago, Illinois 847.9 miles Rachel Nelson MS MPH 312-695-0415 rachel.nelson@northwestern.edu
OSF Saint Francis Medical Center - Peoria, Illinois 963.6 miles Ashley Scott RN BSN 309-624-2409 ashley.scott@osfhealthcare.org
OSF Saint Francis Medical Center - Peoria, Illinois 963.6 miles Ashley Scott RN BSN 309-624-2409 ashley.scott@osfhealthcare.org
OSF Saint Francis Medical Center - Peoria, Illinois 963.6 miles Ashley Scott RN BSN 309-624-2409 ashley.scott@osfhealthcare.org
OSF Saint Francis Medical Center - Peoria, Illinois 963.6 miles Ashley Scott RN BSN 309-624-2409 ashley.scott@osfhealthcare.org
University of Iowa - Iowa City, Iowa 1,050.4 miles Mary Teresi 319-384-7546 Mary-teresi@uiowa.edu
University of Iowa - Iowa City, Iowa 1,050.4 miles Mary Teresi 319-384-7546 Mary-teresi@uiowa.edu
University of Iowa - Iowa City, Iowa 1,050.4 miles Mary Teresi 319-384-7546 Mary-teresi@uiowa.edu
University of Iowa - Iowa City, Iowa 1,050.4 miles Mary Teresi 319-384-7546 Mary-teresi@uiowa.edu
University of Alabama at Birmingham - Birmingham, Alabama 1,051.7 miles Ginger Reeves 205-638-5970 greeves@peds.uab.edu
Central Florida Pulmonary Group - Altamonte Springs, Florida 1,115.8 miles Billy Fortson 407-481-1100 bfortson@cfpulmonary.com
Central Florida Pulmonary Group - Altamonte Springs, Florida 1,115.8 miles Billy Fortson 407-481-1100 bfortson@cfpulmonary.com
Central Florida Pulmonary Group - Altamonte Springs, Florida 1,115.8 miles Billy Fortson 407-481-1100 bfortson@cfpulmonary.com
Central Florida Pulmonary Group - Altamonte Springs, Florida 1,115.8 miles Billy Fortson 407-481-1100 bfortson@cfpulmonary.com
Childrens Mercy Kansas City - Kansas City, Missouri 1,249.3 miles Candy Schmoll RN BSN 816-701-1339 cschmoll@cmh.edu
Childrens Mercy Kansas City - Kansas City, Missouri 1,249.3 miles Candy Schmoll RN BSN 816-701-1339 cschmoll@cmh.edu
Childrens Mercy Kansas City - Kansas City, Missouri 1,249.3 miles Candy Schmoll RN BSN 816-701-1339 cschmoll@cmh.edu
Childrens Mercy Kansas City - Kansas City, Missouri 1,249.3 miles Candy Schmoll RN BSN 816-701-1339 cschmoll@cmh.edu
University of Nebraska Medical Center - Omaha, Nebraska 1,283.6 miles James Melson RN BSN 402-559-6275 jmelson@unmc.edu
University of Nebraska Medical Center - Omaha, Nebraska 1,283.6 miles James Melson RN BSN 402-559-6275 jmelson@unmc.edu
University of Nebraska Medical Center - Omaha, Nebraska 1,283.6 miles James Melson RN BSN 402-559-6275 jmelson@unmc.edu
University of Nebraska Medical Center - Omaha, Nebraska 1,283.6 miles James Melson RN BSN 402-559-6275 jmelson@unmc.edu
University of Texas Health Science Center at Tyler - Tyler, Texas 1,490.0 miles Elizabeth Garza 903-877-5435 elizabeth.garza@uthct.edu
University of Texas Health Science Center at Tyler - Tyler, Texas 1,490.0 miles Elizabeth Garza 903-877-5435 elizabeth.garza@uthct.edu
University of Texas Health Science Center at Tyler - Tyler, Texas 1,490.0 miles Elizabeth Garza 903-877-5435 elizabeth.garza@uthct.edu
University of Texas Health Science Center at Tyler - Tyler, Texas 1,490.0 miles Elizabeth Garza 903-877-5435 elizabeth.garza@uthct.edu
University of Oklahoma Health Sciences Center - Oklahoma City, Oklahoma 1,493.6 miles Jodie Fox None Jodie-Fox@ouhsc.edu
University of Oklahoma Health Sciences Center - Oklahoma City, Oklahoma 1,493.6 miles Jodie Fox None Jodie-Fox@ouhsc.edu
University of Oklahoma Health Sciences Center - Oklahoma City, Oklahoma 1,493.6 miles Jodie Fox None Jodie-Fox@ouhsc.edu
Santiago Reyes MD PC - Oklahoma City, Oklahoma 1,495.7 miles Teresa Orf 405-945-4495 teresaorf@yahoo.com
Santiago Reyes MD PC - Oklahoma City, Oklahoma 1,495.7 miles Teresa Orf 405-945-4495 teresaorf@yahoo.com
Santiago Reyes MD PC - Oklahoma City, Oklahoma 1,495.7 miles Teresa Orf 405-945-4495 teresaorf@yahoo.com
Santiago Reyes MD PC - Oklahoma City, Oklahoma 1,495.7 miles Teresa Orf 405-945-4495 teresaorf@yahoo.com
ICON Early Phase Services - San Antonio, Texas 1,763.7 miles None None None
ICON Early Phase Services - San Antonio, Texas 1,763.7 miles None None None
ICON Early Phase Services - San Antonio, Texas 1,763.7 miles None None None
ICON Early Phase Services - San Antonio, Texas 1,763.7 miles None None None
National Jewish Health - Denver, Colorado 1,766.1 miles Connie St Clair None stclairc@njhealth.org
National Jewish Health - Denver, Colorado 1,766.1 miles Connie St Clair None stclairc@njhealth.org
National Jewish Health - Denver, Colorado 1,766.1 miles Connie St Clair None stclairc@njhealth.org
National Jewish Health - Denver, Colorado 1,766.1 miles Connie St Clair None stclairc@njhealth.org
University of Utah - Salt Lake City, Utah 2,094.1 miles Kristyn Packer 801-585-0401 kristyn.packer@hsc.utah.edu
University of Utah - Salt Lake City, Utah 2,094.1 miles Kristyn Packer 801-585-0401 kristyn.packer@hsc.utah.edu
University of Utah - Salt Lake City, Utah 2,094.1 miles Kristyn Packer 801-585-0401 kristyn.packer@hsc.utah.edu
University of Utah - Salt Lake City, Utah 2,094.1 miles Kristyn Packer 801-585-0401 kristyn.packer@hsc.utah.edu
St Lukes CF Center of Idaho - Boise, Idaho 2,256.8 miles Lejla Tucakovic 208-381-4717 tucakovl@slhs.org
St Lukes CF Center of Idaho - Boise, Idaho 2,256.8 miles Lejla Tucakovic 208-381-4717 tucakovl@slhs.org
St Lukes CF Center of Idaho - Boise, Idaho 2,256.8 miles Lejla Tucakovic 208-381-4717 tucakovl@slhs.org
St Lukes CF Center of Idaho - Boise, Idaho 2,256.8 miles Lejla Tucakovic 208-381-4717 tucakovl@slhs.org
Childrens Lung Specialists - Las Vegas, Nevada 2,376.9 miles Ryan Yoshikawa 702-598-4411 rmyoshika@gmail.com
Childrens Lung Specialists - Las Vegas, Nevada 2,376.9 miles Ryan Yoshikawa 702-598-4411 rmyoshika@gmail.com
Childrens Lung Specialists - Las Vegas, Nevada 2,376.9 miles Ryan Yoshikawa 702-598-4411 rmyoshika@gmail.com
Childrens Lung Specialists - Las Vegas, Nevada 2,376.9 miles Ryan Yoshikawa 702-598-4411 rmyoshika@gmail.com
Stanford University Medical Center - Stanford, California 2,694.5 miles Colleen Dunn None cedunn@stanford.edu
Stanford University Medical Center - Stanford, California 2,694.5 miles Colleen Dunn None cedunn@stanford.edu
Stanford University Medical Center - Stanford, California 2,694.5 miles Colleen Dunn None cedunn@stanford.edu
Providence Alaska Medical Center - Anchorage, Alaska 3,372.7 miles Vicki Roberts 907-212-3299 vicki.roberts@providence.org

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