Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis

• ClinicalTrials.gov Identifier: NCT03251092
• Recruitment Status: Completed
• First Posted: August 16, 2017
• Last Update Posted: April 22, 2020
• Sponsor: Proteostasis Therapeutics, Inc.
• Information provided by (Responsible Party): Proteostasis Therapeutics, Inc.

Study Description

Brief Summary:

Part 1 of this trial will enroll healthy volunteers into a single ascending dose (SAD), multiple ascending dose (MAD), and Food Effect (FE) treatment groups.

The SAD treatment group is comprised of at least 3 ascending dose level cohorts where healthy adult subjects will be randomized to receive a single dose of either PTI-808 or placebo and will be followed for 7 days post dose. A safety review committee (SRC) will convene after the completion of each cohort to evaluate safety and pharmacokinetic (PK) data.

Following the conclusion of the respective SAD level dose groups and after sufficient review of study data and approval by the SRC, a second set of healthy adult subjects will participate in an assigned MAD treatment group. The MAD treatment group is comprised of 3 ascending dose level cohorts where subjects will be randomized to receive either PTI-808 or placebo daily for 7 days and will be followed for 7 days after receiving the last dose.

Also following the conclusion of the respective SAD level dose groups, healthy adult subjects will participate in the FE treatment group.

Part 2 of this will enroll healthy volunteers to assess the safety, tolerability, and PK of PTI 808 co administered with PTI 801 and PTI 428 to HVs with daily dosing for 7 consecutive days.

Part 3 will enroll adult subjects with cystic fibrosis (CF) into a MAD treatment group consisting of 2 cohorts. Subjects will receive PTI-808 co-administered with PTI-801 and PTI-428. PTI-808 will be administered daily for 7 consecutive days followed by PTI-808 + PTI-801 + PTI-428 administered daily for 14 consecutive days.

Part 4 will enroll adult subjects with cystic fibrosis (CF) into 28-day cohorts. Subjects will receive PTI-808 co-administered with PTI-801 with or without PTI-428 versus matching placebo.

Condition or disease	Intervention/treatment	Phase
Healthy Volunteer - Complete; Cystic Fibrosis - Complete	Drug: PTI-808; Drug: Placebo; Drug: PTI-428; Drug: PTI-801	Phase 1; Phase 2

Detailed Description:

Part 1 of this trial will enroll healthy volunteers into a single ascending dose (SAD), multiple ascending dose (MAD), and Food Effect (FE) treatment groups.

The SAD treatment group is comprised of at least 3 ascending dose level cohorts where healthy adult subjects will be randomized to receive a single dose of either PTI-808 or placebo and will be followed for 7 days post dose.

The MAD treatment group is comprised of 3 ascending dose level cohorts where subjects will be randomized to receive either PTI-808 or placebo daily for 7 days and will be followed for 7 days after receiving the last dose.

Following the conclusion of the respective SAD level dose groups the food effect portion of the study will be initiated and subjects will be randomized to receive an initial single dose of PTI-808 either after an overnight fast of at least 10 hours (fasted group) or after an overnight fast of at least 10 hours followed the consumption of a high fat high calorie meal (fed group). After a 10 day washout period, subjects will cross over to the opposite group and receive a second dose of PTI-808. Subjects will be followed for up to 7 days following dosing.

Part 2 of this will enroll healthy volunteers to assess the safety, tolerability, and PK of PTI 808 co administered with PTI 801 and PTI 428 to HVs with daily dosing for 7 consecutive days.

Part 3 - Part 3 will enroll adult subjects with CF to assess the safety, tolerability, and PK of multiple ascending doses of PTI-808 co-administered with PTI-801 and PTI-428. Subjects will receive 7 days of PTI-808 or placebo followed by 14 days of PTI-808 or placebo co-administered with PTI-801+PTI-428 or matching placebos.

Part 4 - Part 4 will assess the safety, tolerability, PK, and the effects of PTI-808 co-administered with PTI-801 with or without PTI-428 over a 28-day treatment period in CF subjects who are either homozygous for the F508del CFTR genotype or are heterozygous for the F508del CFTR genotype. Subjects will be randomized to receive treatment with PTI-808 co-administered with PTI-801 with or without PTI-428 versus matching placebo.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 179 participants
• Allocation: Randomized
• Intervention Model: Sequential Assignment
• Masking: Triple (Participant, Care Provider, Investigator)
• Primary Purpose: Treatment
• Official Title: A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults With Cystic Fibrosis
• Actual Study Start Date: July 17, 2017
• Actual Primary Completion Date: December 23, 2019
• Actual Study Completion Date: December 23, 2019

Arms and Interventions

Arm	Intervention/treatment
Active Comparator: SAD PTI-808 Active; Three cohorts of SAD are planned for evaluation where subjects will be randomized to PTI-808 or placebo.	Drug: PTI-808; Active
Placebo Comparator: SAD PTI-808 Placebo; Three cohorts of SAD are planned for evaluation where subjects will be randomized to PTI-808 or placebo.	Drug: Placebo; Placebo
Active Comparator: MAD PTI-808 Active; Three cohorts of MAD are planned for evaluation where subjects will be randomized to PTI-808 or placebo.	Drug: PTI-808; Active
Placebo Comparator: MAD PTI-808 Placebo; Three cohorts of MAD are planned for evaluation where subjects will be randomized to PTI-808 or placebo.	Drug: Placebo; Placebo
Active Comparator: FE PTI-808 Active; Subjects will be randomized to Fed or Fasted on Days 1 and 12. Follow up visits will occur 7 days post Day 12 dose.	Drug: PTI-808; Active
Placebo Comparator: FE PTI-808 Placebo; Subjects will be randomized to Fed or Fasted on Days 1 and 12. Follow up visits will occur 7 days post Day 12 dose.	Drug: Placebo; Placebo
Experimental: Part 2 PTI-808 + PTI-801 + PTI-428 Active; One cohort is planned where subjects will be randomized to either the triple active arm (dosed with PTI 808+PTI 801+PTI 428) OR placebo arm.	Drug: PTI-808; Active; Drug: PTI-428; Active; Drug: PTI-801; Active
Placebo Comparator: Part 2 matching Placebos; In all three cohorts in part 2, subjects will be randomized to active drug or placebo. The placebo arm for all cohorts consists of placebo capsules matching PTI-808+PTI-801+PTI-428.	Drug: Placebo; Placebo
Experimental: Part 2 dual active arm PTI-801+PTI-428+ PTI-808 placebo; One cohort is planned where subjects are randomized to either 808 placebo + dual active arm (dosed with placebo matching PTI 808 plus PTI 801 + PTI 428) OR placebo arm.	Drug: Placebo; Placebo; Drug: PTI-428; Active; Drug: PTI-801; Active
Active Comparator: Part 2 dual active arm PTI-801+PTI-808+PTI-428 placebo; One cohort is planned where subjects are randomized to either 428 placebo + dual active arm (dosed with placebo matching PTI 428 plus PTI 808 and PTI 801) OR placebo arm.	Drug: PTI-808; Active; Drug: Placebo; Placebo; Drug: PTI-801; Active
Active Comparator: Part 3 CF MAD PTI-808 + PTI-801 + PTI-428; In all cohorts in Part 3, subjects will be will be randomized to receive 7 days of PTI-808 or placebo followed by 14 days of co-administration of PTI-808+PTI-801+PTI-428 or matching placebos. A follow-up will occur on Day 28.	Drug: PTI-808; Active; Drug: PTI-428; Active; Drug: PTI-801; Active
Placebo Comparator: Part 3 CF MAD PTI-808 placebo+PTI-801 placebo+PTI-428 placebo; In all cohorts in Part 3, subjects will be randomized to receive 7 days of PTI-808 or placebo followed by 14 days of co-administration of PTI-808+PTI-801+PTI-428 or matching placebos. A follow-up will occur on Day 28.	Drug: Placebo; Placebo
Active Comparator: Part 4 CF PTI-808 + PTI-801 + PTI-428; In cohorts 3 & 4 subjects will be randomized to receive PTI-808 + PTI-801 with or without PTI-428 or matching placebos. A follow-up will occur on Day 42.	Drug: PTI-808; Active; Drug: PTI-428; Active; Drug: PTI-801; Active
Active Comparator: Part 4 CF PTI-808 + PTI-801 + PTI-428 placebo; In cohorts 3 & 4, subjects will be randomized to receive PTI-808 + PTI-801 with or without PTI-428 or matching placebos. A follow-up will occur on Day 42.	Drug: PTI-808; Active; Drug: PTI-801; Active
Placebo Comparator: Part 4 CF PTI-808 placebo + PTI-801 placebo + PTI-428 placebo; In cohorts 3 & 4, subjects will be randomized to receive PTI-808 + PTI-801 with or without PTI-428 or matching placebos. A follow-up will occur on Day 42.	Drug: Placebo; Placebo

Outcome Measures

Primary Outcome Measures :

1. Part 1 SAD and MAD: Adverse Events [ Time Frame: Baseline to up to 14 days ]
   Safety and tolerability measure by number of subjects who experience adverse events
2. Part 1 SAD and MAD: Physical Exams [ Time Frame: Baseline to up to 14 days ]
   Safety and tolerability measure by number of subjects who experience potential clinically significant changes in physical examinations
3. Part 1 SAD and MAD: The number of subjects who experience potential clinically significant changes in vital signs [ Time Frame: Baseline to up to 14 days ]
   Safety and tolerability measure by number of subjects who experience potential clinically significant changes in vital signs
4. Part 1 SAD and MAD: ECGs [ Time Frame: Baseline to up to 14 days ]
   Safety and tolerability measure by number of subjects who experience potential clinically significant changes in ECGs
5. Part 1 SAD and MAD: The number of subjects who experience potential clinically significant changes in safety labs [ Time Frame: Baseline to up to 14 days ]
   Safety and tolerability measure by number of subjects who experience potential clinically significant changes in safety labs
6. Part 1 SAD and FE: terminal half life [ Time Frame: Through 72 hours post dose ]
   Apparent terminal half-life (t1/2) of single oral dose
7. Part 1 SAD and FE : Tmax [ Time Frame: Through 72 hours post dose ]
   Time to reach maximum plasma concentration (Tmax) of single oral dose
8. Part 1 SAD and FE: Cmax [ Time Frame: Through 72 hours post dose ]
   Maximum plasma concentration (Cmax) of single oral dose
9. Part 1 SAD : AUC [ Time Frame: Through 24 hours post dose ]
   Area under the concentration-time curve from time 0 to 24 hours post dose (AUC 0-24) of single oral dose
10. Part 1 SAD and FE: AUC0 [ Time Frame: Through 72 hours post dose ]
    AUC from time 0 to time of last measurable concentration (AUC0-last) of single oral dose
11. Part 1 SAD and FE: AUC0-inf [ Time Frame: Through 72 hours post dose ]
    AUC from time 0 to infinity (AUC0-inf) of single dose
12. Part 1 MAD: t1/2 [ Time Frame: Through 72 hours post dose ]
    t1/2 of multiple oral dose
13. Part 1 MAD: Tmax [ Time Frame: Through 72 hours post dose ]
    Tmax of multiple oral doses
14. Part 1 MAD: Cmax [ Time Frame: Through 72 hours post last dose ]
    Cmax of multiple oral doses
15. Part 1 MAD: AUC0-24 [ Time Frame: Through 24 hours post last dose ]
    AUC0-24 of multiple oral dose
16. Part 1 MAD: AUC0-last [ Time Frame: Through 72 hours post last dose ]
    AUC0-last of multiple oral doses
17. Part 1 MAD: Urine [ Time Frame: Through 24 hours post last dose ]
    Cumulative amount of PTI-808 excreted unchanged in urine (Ae) as appropriate of multiple oral doses
18. Part 1 MAD: CLR [ Time Frame: Through 24 hours post dose ]
    Renal clearance (CLR) of multiple oral doses
19. Part 2: Physical Exams [ Time Frame: Baseline up to 14 days ]
    Safety and tolerability measure by number of subjects who experience potential clinically significant changes in physical examinations
20. Part 2: ECGs [ Time Frame: Baseline up to 14 days ]
    Safety and tolerability measure by number of subjects who experience potential clinically significant changes in ECGs
21. Part 2: Safety Labs [ Time Frame: Baseline up to 14 days ]
    Safety and tolerability measure by number of subjects who experience potential clinically significant changes in safety labs
22. Part 2: Vitals Signs [ Time Frame: Baseline up to 14 days ]
    Measure by number of subjects who experience potential clinically significant changes in vital signs
23. Part 3 CF: Physical Exams [ Time Frame: Baseline up to 28 days ]
    Safety and tolerability measured by number of subjects who experience potential clinically significant changes in physical examinations
24. Part 3 CF: ECGs [ Time Frame: Baseline up to 28 days ]
    Safety and tolerability measured by number of subjects who experience potential clinically significant changes in ECGs
25. Part 3 CF: Safety Labs [ Time Frame: Baseline up to 28 days ]
    Safety and tolerability measured by number of subjects who experience potential clinically significant changes in safety labs
26. Part 3 CF: Vital Signs [ Time Frame: Baseline up to 28 days ]
    Measured by number of subjects who experience potential clinically significant changes in vital signs
27. Part 4 CF: Physical Exams [ Time Frame: Baseline up to 42 days ]
    Safety and tolerability measured by number of subjects who experience potential clinically significant changes in physical examinations
28. Part 4 CF: ECGs [ Time Frame: Baseline up to 42 days ]
    Safety and tolerability measured by number of subjects who experience potential clinically significant changes in ECGs
29. Part 4 CF: Safety Labs [ Time Frame: Baseline up to 42 days ]
    Safety and tolerability measured by number of subjects who experience potential clinically significant changes in safety labs
30. Part 4 CF: Vital Signs [ Time Frame: Baseline up to 42 days ]
    Safety and tolerability measured by number of subjects who experience potential clinically significant changes in physical examinations

Secondary Outcome Measures :

1. Part 2: Apparent terminal half life (t1/2) of multiple oral doses PTI 808 is co administered with PTI 801 and PTI 428 [ Time Frame: Day 1 through Day 10 ]
   Evaluate the PK profile of PTI 808, PTI 801, and PTI 428 when PTI 808 is co administered with PTI 801 and PTI 428 in healthy adults
2. Part 2: Maximum plasma concentration (Cmax) of multiple oral doses PTI 808 is co administered with PTI 801 and PTI 428 [ Time Frame: Day 1 through Day 10 ]
   Evaluate the PK profile of PTI 808, PTI 801, and PTI 428 when PTI 808 is co administered with PTI 801 and PTI 428 in healthy adults
3. Part 2: Time to reach maximum plasma concentration (Tmax) of multiple oral doses PTI 808 is co administered with PTI 801 and PTI 428 [ Time Frame: Day 1 through Day 10 ]
   Evaluate the PK profile of PTI 808, PTI 801, and PTI 428 when PTI 808 is co administered with PTI 801 and PTI 428 in healthy adults
4. Part 2: AUC0-last of multiple oral doses when PTI 808 is co administered with PTI 801 and PTI 428 [ Time Frame: Day 1 through Day 10 ]
   Evaluate the PK profile of PTI 808, PTI 801, and PTI 428 when PTI 808 is co administered with PTI 801 and PTI 428 in healthy adults
5. Part 2: AUC from time 0 to infinity (AUC0-inf) of multiple oral doses when PTI 808 is co administered with PTI 801 and PTI 428 [ Time Frame: Day 1 through Day 10 ]
   Evaluate the PK profile of PTI 808, PTI 801, and PTI 428 when PTI 808 is co administered with PTI 801 and PTI 428 in healthy adults
6. Part 3 CF: Time to reach maximum plasma concentration (Tmax) of multiple oral doses PTI 808 is co administered with PTI 801 and PTI 428 [ Time Frame: Day 1 through Day 22 ]
   Evaluate the PK profile of PTI 808, PTI 801, and PTI 428 when PTI 808 is co administered with PTI 801 and PTI 428 in adults with CF
7. Part 3 CF: Maximum plasma concentration (Cmax) of multiple oral doses PTI 808 is co administered with PTI 801 and PTI 428 [ Time Frame: Day 1 through Day 22 ]
   Evaluate the PK profile of PTI 808, PTI 801, and PTI 428 when PTI 808 is co administered with PTI 801 and PTI 428 in adults with CF
8. Part 3 CF: AUC0-last of multiple oral doses when PTI 808 is co administered with PTI 801 and PTI 428 [ Time Frame: Day 1 through Day 22 ]
   Evaluate the PK profile of PTI 808, PTI 801, and PTI 428 when PTI 808 is co administered with PTI 801 and PTI 428 in adults with CF
9. Part 3 CF: FEV1 [ Time Frame: Baseline through Day 28 ]
   Change in forced expiratory volume in one second (FEV1) over time
10. Part 4 CF: Time to reach maximum plasma concentration (Tmax) of multiple oral doses of PTI 808 + PTI 801 co-administered with or without PTI 428 [ Time Frame: Day 1 through Day 28 ]
    Evaluate the PK profile of PTI 808, PTI 801, and PTI 428 when PTI 808 + PTI 801 is co administered with or with out PTI 428 in adults with CF
11. Part 4 CF: Maximum plasma concentration (Cmax) of multiple oral doses of PTI 808 + PTI 801 co-administered with or without PTI 428 [ Time Frame: Day 1 through 28 ]
    Evaluate the PK profile of PTI 808, PTI 801, and PTI 428 when PTI 808 + PTI 801 is co administered with or with out PTI 428 in adults with CF
12. Part 4 CF: AUC0-last of multiple oral doses when PTI 808 + PTI 801 is coadministered with or without PTI 428 in adults with CF [ Time Frame: Day 1 through 28 ]
    Evaluate the PK profile of PTI 808, PTI 801, and PTI 428 when PTI 808 + PTI 801 is co administered with or without PTI 428 in adults with CF
13. Part 4 CF: FEV1 [ Time Frame: Baseline through Day 42 ]
    Change in forced expiratory volume in one second (FEV1) over time
14. Part 4 CF Sweat Chloride [ Time Frame: Baseline through Day 42 ]
    Change in sweat chloride concentrations over time

Other Outcome Measures:

1. Part 2 Nasal biomarker [ Time Frame: Baseline up to 14 days ]
   change in nasal epithelial mRNA and protein over time
2. Part 3 CF Sweat Chloride [ Time Frame: Baseline up to 28 days ]
   Change in sweat chloride concentrations over time
3. Part 3 CF Nasal biomarker [ Time Frame: Baseline up to 28 days ]
   Change in nasal epithelial mRNA and protein expression over time
4. Part 4 CF Weight and BMI [ Time Frame: Baseline up to 42 days ]
   Change in weight and BMI over time
5. Part 4 CF Blood Glucose [ Time Frame: Baseline up to 42 days ]
   Change in blood glucose over time
6. Part 4 CF disease-specific health related quality of life [ Time Frame: Baseline up to 42 days ]
   Change in disease-specific health related quality of life over time
7. Part 4 CF Nasal biomarker [ Time Frame: Baseline up to 42 days ]
   Change in nasal epithelial mRNA and protein expression over time

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 99 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes

Criteria

Part 1 and Part 2 Inclusion Criteria:

1. Adults aged 18 to 55 years old, inclusive, at the time of informed consent
2. Body mass index ≥18 and <30 kg/m2
3. Subject must be a non-smoker and non-tobacco user for a minimum of 30 days prior to screening and for the duration of the study.
4. Subject understands the full nature and purpose of the study, including possible risks and side effects, and is willing and able to comply with all compulsory study procedures and provides informed consent/permission prior to any study procedures being performed.
5. Females of childbearing potential and males capable of fathering a child must meet the contraception requirements

Part 1 & Part 2 Exclusion Criteria:

1. 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
2. Prolonged QT interval with Fridericia's correction >450 msec at screening
3. Abnormal liver function as defined by aspartate transaminase (AST), alanine transaminase (ALT), or bilirubin >1.5× the upper limit of the normal range
4. Abnormal renal function at screening defined as creatinine clearance <90 mL/min using the Cockroft-Gault equation
5. Clinically significant screening results that would exclude subject from the study (e.g., medical histories, PE, ECGs, vital signs, and laboratory profiles) as deemed by the investigator
6. Participation in another clinical study or treatment with an investigational agent within 30 days or five half-lives, whichever is longer, prior to Study Day 1
7. History of cancer within the past 5 years (excluding non-melanoma skin cancer)
8. History or current evidence of alcohol or drug abuse or dependence within 12 months of screening as determined by the investigator
9. 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
10. Positive blood screen for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or hepatitis C virus antibody (HCVAb)
11. Clinically significant infection within 3 months of screening as determined by the investigator
12. Known or suspected hypersensitivity or idiosyncratic reaction to study medication or any components thereof
13. Has donated blood within 3 months of screening or plans to donate blood within 3 months of study completion
14. Pregnant or nursing women
15. Any conditions that, in the opinion of the investigator, would make the subject unsuitable for enrollment or could interfere with the subject's participation in or completion of the study
16. Use of prohibited medications within 14 days prior to dosing of study drug

Part 3 CF Inclusion Criteria:

1. Confirmed diagnosis of CF with the F508del/F508del genotype
2. Forced expiratory volume in 1 second (FEV1) 40-90% predicted, inclusive
3. Non-smoker and non-tobacco user for a minimum of 30 days prior to screening

Part 3 CF Exclusion Criteria:

1. 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
2. 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)
3. History of organ transplantation
4. 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
5. 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
6. History or current evidence of alcohol or drug abuse or dependence within 12 months of screening as determined by the investigator
7. Pregnant or nursing women
8. Currently taking or has taken a CFTR modulator within 30 days prior to initial dose of study drugs

Part 4 CF Inclusion Criteria:

1. Confirmed diagnosis of CF with either the F508del CFTR homozygous genotype on record or for heterozygote subjects, only one copy of the F508del CFTR mutation on record
2. Forced expiratory volume in 1 second (FEV1) 40-90% predicted, inclusive
3. Non-smoker and non-tobacco user for a minimum of 30 days prior to screening

Part 4 CF Exclusion Criteria:

1. 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
2. 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)
3. History of organ transplantation
4. 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 28 days of Day 1
5. 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 of Day 1
6. History or current evidence of alcohol or drug abuse or dependence within 12 months of screening as determined by the investigator
7. Pregnant or nursing women
8. Currently taking or has taken a CFTR modulator within 14 days prior to the screening visit

Contacts and Locations

Locations

United States, Arizona

Banner University of Arizona Medical Center

Tucson, Arizona, United States, 85724

United States, California

Stanford University Medical Center

Stanford, California, United States, 94305

United States, Colorado

National Jewish Health

Denver, Colorado, United States, 80206

United States, Florida

Central Florida Pulmonary Group

Altamonte Springs, Florida, United States, 32803

United States, Georgia

Emory Children's Center

Atlanta, Georgia, United States, 30322

United States, Illinois

Northwestern Memorial Hospital

Chicago, Illinois, United States, 60611

United States, Maryland

Johns Hopkins University

Baltimore, Maryland, United States, 21287

United States, Massachusetts

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

Boston Children's Hospital

Boston, Massachusetts, United States, 02115

United States, Michigan

Michigan Medicine, University of Michigan

Ann Arbor, Michigan, United States, 48109

Harper University Hospital

Detroit, Michigan, United States, 48201

United States, Minnesota

University of Minnesota

Minneapolis, Minnesota, United States, 55455

United States, Missouri

Children's Mercy

Kansas City, Missouri, United States, 64108

United States, Montana

Billings Clinic

Billings, Montana, United States, 59101

United States, Nebraska

University of Nebraska Medical Center

Omaha, Nebraska, United States, 68198

United States, New Hampshire

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, United States, 03756

United States, New York

New York Medical College

Valhalla, New York, United States, 10595

United States, North Carolina

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States, 27517

United States, Pennsylvania

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

United States, South Carolina

Medical University of South Carolina

Charleston, South Carolina, United States, 29425

United States, Texas

ICON Early Phase Services

San Antonio, Texas, United States, 78209

United States, Utah

University of Utah

Salt Lake City, Utah, United States, 84132

Australia, New South Wales

John Hunter Hospital

Lambton, New South Wales, Australia, 2305

Belgium

Universitair ziekenhuis Brussel

Brussels, Belgium, 1090

UZ Leuven

Leuven, Belgium, 3000

Canada, British Columbia

St. Paul's Hospital

Vancouver, British Columbia, Canada, V6Z1Y6

Canada, Quebec

Centre hospitalier de l'Université de Montréal (CHUM)

Montréal, Quebec, Canada, H2X3E4

McGill University Health Centre

Montréal, Quebec, Canada, H4A3J1

Canada

Institut universitaire de cardiologie et de pneumologie de Québec-Université Laval

Québec, Canada, G1V4G5

Denmark

University of Copenhagen Rigshospitalet

Copenhagen, Denmark, 2100

France

Hôpital Pasteur

Nice, Alpes-Maritimes, France, 06001

Hôpital Haut Lévêque

Pessac, Gironde, France, 33600

Hôpital Guillaume-et-René-Laennec

Nantes, Loire-Atlantique, France, 44093

Hôpital Maison Blanche Maladies respiratoires et allergologie

Reims, Marne, France, 51092

Hospices Civils de Lyon

Lyon, France, 69495

Hôpital Cochin

Paris, France, 75014

Germany

Charité Universitätsmedizin Berlin

Berlin, Germany, 10117

University Hospital Cologne

Cologne, Germany, 50937

Universitätsklinikum Essen

Essen, Germany, 45239

Klinikum der J.W. Goethe Universität

Frankfurt, Germany, 60590

Klinikum des Universität München

München, Germany, 81377

New Zealand

Auckland Clinical Studies Ltd.

Grafton, Auckland, New Zealand, 1010

United Kingdom

Royal Devon and Exeter Hospital

Exeter, Devon, United Kingdom, EX2 5DW

Western General Hospital

Edinburgh, Scotland, United Kingdom, EH42XU

Birmingham Heartlands Hospital

Birmingham, West Midlands, United Kingdom, B9 5SS

Belfast City Hospital

Belfast, United Kingdom, BT9 7AB

King's College Hospital

London, United Kingdom, SE59RS

University Hospital Southampton

Southampton, United Kingdom, SO16 6YD

Sponsors and Collaborators

Proteostasis Therapeutics, Inc.

Investigators

• Principal Investigator: Cassandra Key, MD; ICON Early Phase Services (Parts 1 & 2)
• Principal Investigator: Patrick Flume, MD; Medical University of South Carolina (Parts 3 & 4)

More Information

• Responsible Party: Proteostasis Therapeutics, Inc.
• ClinicalTrials.gov Identifier: NCT03251092
• Other Study ID Numbers: PTI-808-01
• First Posted: August 16, 2017
• Last Update Posted: April 22, 2020
• Last Verified: April 2020

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Cystic Fibrosis; Fibrosis; Pathologic Processes; Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Infant, Newborn, Diseases