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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

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ClinicalTrials.gov Identifier: NCT03140527
Recruitment Status : Recruiting
First Posted : May 4, 2017
Last Update Posted : May 1, 2018
Sponsor:
Information provided by (Responsible Party):
Proteostasis Therapeutics, Inc.

Brief Summary:
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 Cohorts 1 through 3 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. Part 2 Cohort 4 will enroll adult subjects with CF not currently receiving or have received cystic fibrosis conductance regulator (CFTR) modulator therapy within 30 days prior to Day 1. Approximately 125 subjects will be enrolled.

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

Detailed Description:

PART 1 The SAD treatment group is comprised of three cohorts where HV will be randomized to either PTI-801 or placebo. Following the conclusion of at least three SAD cohorts, a set of HV will participate in an assigned MAD treatment group. The MAD treatment group is comprised of three cohorts where subjects will be randomized to receive either PTI-801 or placebo once daily (QD) for a total of 7 days. Following the conclusion of at least three SAD cohorts, a set of HV will participate in a FE treatment group. The FE treatment group is comprised of one cohort where subjects will be randomized to receive an initial single dose of PTI-801 either after an overnight fast of at least 10 hours (fasted group) or after an overnight fast of at least 10 hours followed by the consumption of a high fat high and 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-801. Following the conclusion of at least two MAD cohorts, a set of HV will participate in a DDI treatment group. The DDI treatment group is comprised of one cohort where subjects will receive a 3-drug cocktail consisting of caffeine, bupropion, and midazolam on Day 1. On Day 4, subjects will be randomized to receive either PTI-801 or placebo QD for a total of 12 days. On Day 17, subjects will receive the 3-drug cocktail in combination with PTI-801 or placebo.

PART 2 Part 2 is comprised of a MAD treatment group with three cohorts and a co-administration group with one cohort. Following conclusion of the complementary HV MAD cohort in Part 1, a set of adult subjects diagnosed with CF currently on stable ivacaftor/lumacaftor background therapy for a minimum of three months will participate in the Part 2 complementary CF MAD cohort. Subjects will be randomized to receive either PTI-801 or placebo QD for a total of 14 days. Following conclusion of CF MAD Cohort 1 in Part 2, a set of adult subjects diagnosed with CF not currently receiving or have received background CFTR modulator therapy for a minimum of 30 days prior to Day 1 will participate in the Part 2 CF PTI-801 and PTI-808 co-administration cohort. Subjects will be randomized to receive either PTI-801 co-administered with PTI-808 or placebos QD for a total of 14 days.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 125 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Multi-Center, Randomized, Placebo-Controlled, Phase 1, Two-Part Study Designed to Assess 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
Actual Study Start Date : April 10, 2017
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : August 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Active Comparator: SAD HV PTI-801 Active - Complete
The safety, tolerability, and pharmacokinetic profile of PTI-801 will be evaluated following a single dose of PTI-801. Three cohorts are planned for evaluation where subjects will be randomized to PTI-801 or placebo.The subjects will be followed for 7 days post dose.
Drug: PTI-801
Active

Placebo Comparator: SAD HV PTI-801 Placebo - Complete
The safety, tolerability, and pharmacokinetic profile of PTI-801 will be evaluated following a single dose of PTI-801. Three cohorts are planned for evaluation where subjects will be randomized to PTI-801 or placebo.The subjects will be followed for 7 days post dose.
Drug: Placebo
Placebo

Active Comparator: MAD HV PTI-801 Active - Complete
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 cohorts. Subjects will be randomized to either PTI-801 or placebo. Each dose will be administered once daily (QD) for a total of 7 days. Follow up visits will occur on Days 10, 12 and 14.
Drug: PTI-801
Active

Placebo Comparator: MAD HV PTI-801 Placebo - Complete
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 cohorts. Subjects will be randomized to either PTI-801 or placebo. Each dose will be administered once daily (QD) for a total of 7 days. Follow up visits will occur on Days 10, 12 and 14.
Drug: Placebo
Placebo

Active Comparator: FE HV PTI-801 Active - Complete
Following the conclusion of SAD groups and after sufficient review of study data and approval by the SRC, a third set of healthy adult subjects will participate in the Food Effect cohort. 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-801
Active

Active Comparator: DDI HV PTI-801 Active - Complete
Following the conclusion of HV MAD Cohort 2 and after sufficient review of study data and approval by the SRC, a fourth set of healthy adult subjects will participate in the Drug-Drug Interactions cohort. Subjects will receive a 3-drug cocktail consisting of caffeine, bupropion, and midazolam on Day 1. On Day 4, subjects will be randomized to receive either PTI-801 or placebo QD for a total of 12 days. On Day 17, subjects will receive the 3-drug cocktail in combination with PTI-801 or placebo. Subjects will remain in clinic until Day 20. A follow up visit will occur on Day 24.
Drug: PTI-801
Active

Placebo Comparator: DDI HV PTI-801 Placebo - Complete
Following the conclusion of HV MAD Cohort 2 and after sufficient review of study data and approval by the SRC, a fourth set of healthy adult subjects will participate in the Drug-Drug Interactions cohort. Subjects will receive a 3-drug cocktail consisting of caffeine, bupropion, and midazolam on Day 1. On Day 4, subjects will be randomized to receive either PTI-801 or placebo QD for a total of 12 days. On Day 17, subjects will receive the 3-drug cocktail in combination with PTI-801 or placebo. Subjects will remain in clinic until Day 20. A follow up visit will occur on Day 24.
Drug: Placebo
Placebo

Active Comparator: MAD CF PTI-801 Active
Following conclusion of the complementary HV MAD cohort in Part 1 and after sufficient review of study data and approval by the SRC, a set of adult subjects diagnosed with CF currently on stable ivacaftor/lumacaftor background therapy for a minimum of three months will participate in the Part 2 complementary CF MAD cohort. The CF MAD treatment group is comprised of 3 cohorts. Subjects will be randomized to receive either PTI-801 or placebo QD for a total of 14 days. A follow up visit will occur on Day 21.
Drug: PTI-801
Active

Placebo Comparator: MAD CF PTI-801 Placebo
Following conclusion of the complementary HV MAD cohort in Part 1 and after sufficient review of study data and approval by the SRC, a set of adult subjects diagnosed with CF currently on stable ivacaftor/lumacaftor background therapy for a minimum of three months will participate in the Part 2 complementary CF MAD cohort. The CF MAD treatment group is comprised of 3 cohorts. Subjects will be randomized to receive either PTI-801 or placebo QD for a total of 14 days. A follow up visit will occur on Day 21.
Drug: Placebo
Placebo

Active Comparator: Cohort 4 CF PTI-801 Active co-admin with PTI-808 Active
Following conclusion of CF MAD Cohort 1 in Part 2 and after sufficient review of study data and approval by the SRC, a set of adult subjects diagnosed with CF not currently receiving or have received a CFTR modulator therapy within 30 days prior to Day 1 will participate in the Part 2 CF Cohort 4. Subjects will be randomized to receive either PTI-801 co-administered with PTI-808 or placebos QD for a total of 14 days. A follow up visit will occur on Day 21.
Drug: PTI-801
Active

Drug: PTI-808
Active

Placebo Comparator: Cohort 4 CF PTI-801 Placebo co-admin with PTI-808 Placebo
Following conclusion of CF MAD Cohort 1 in Part 2 and after sufficient review of study data and approval by the SRC, a set of adult subjects diagnosed with CF not currently receiving or have received a CFTR modulator therapy within 30 days prior to Day 1 will participate in the Part 2 CF Cohort 4. Subjects will be randomized to receive either PTI-801 co-administered with PTI-808 or placebos QD for a total of 14 days. A follow up visit will occur on Day 21.
Drug: Placebo
Placebo

Drug: Placebo
Placebo




Primary Outcome Measures :
  1. Part 1 SAD and MAD HV: Safety and tolerability measured by number of subjects who experience adverse events and potential clinically significant changes in safety labs, electrocardiograms (ECGs), physical examinations, and vital signs [ Time Frame: baseline to up to 14 days ]
  2. Part 1 SAD: Apparent terminal half-life (t1/2) of single oral dose [ Time Frame: through 72-hours post dose ]
  3. Part 1 SAD: Time to reach maximum plasma concentration (Tmax) of single oral dose [ Time Frame: through 72-hours post dose ]
  4. Part 1 SAD: Maximum plasma concentration (Cmax) of single oral dose [ Time Frame: through 72-hours post dose ]
  5. Part 1 SAD: Area under the concentration-time curve from time 0 to 24 hours post administration (AUC0-24) of a single oral dose [ Time Frame: through 72-hours post dose ]
  6. Part 1 SAD: Area under the concentration-time curve from time 0 to time of last measurable concentration (AUC0-last) of a single oral dose [ Time Frame: through 72-hours post dose ]
  7. Part 1 SAD: AUC from time 0 to infinity (AUC0-inf) [ Time Frame: through 72-hours post dose ]
    using noncompartmental methods as appropriate of single dose

  8. Part 1 MAD HV: Apparent terminal half-life (t1/2) of multiple oral doses [ Time Frame: through 72-hours post last dose ]
  9. Part 1 MAD HV: Time to reach maximum plasma concentration (Tmax) of multiple oral doses [ Time Frame: through 72-hours post last dose ]
  10. Part 1 MAD HV: Maximum plasma concentration (Cmax) of multiple oral doses [ Time Frame: through 72-hours post last dose ]
  11. Part 1 MAD HV: Area under the concentration-time curve from time 0 to 24 hours post administration (AUC0-24) of multiple oral doses [ Time Frame: through 72-hours post dose ]
  12. Part 1 MAD HV: Area under the concentration-time curve from time 0 to time of last measurable concentration (AUClast) of multiple oral doses [ Time Frame: through 72-hour post last dose ]
  13. Part 1 MAD HV: AUC from time 0 to infinity (AUC0-inf) of multiple oral doses [ Time Frame: through 72-hour post last dose ]
    using noncompartmental methods as appropriate of multiple oral doses

  14. Part 1 MAD HV: Cumulative amount of PTI-801 excreted unchanged in urine (Ae) as appropriate of multiple oral doses [ Time Frame: through 24-hour post last dose ]
  15. Part 1 MAD HV: Cumulative amount of PTI-801 unchanged in renal clearance (CLR) as appropriate of multiple oral doses [ Time Frame: through 24-hour post last dose ]
  16. Part 1 FE: Time to reach maximum plasma concentration (Tmax) [ Time Frame: through 72-hour post last dose ]
  17. Part 1 FE :Maximum plasma concentration (Cmax) [ Time Frame: through 72-hour post last dose ]
  18. Part 1 FE: Area under the concentration-time curve from time 0 to time of last measurable concentration (AUCt) [ Time Frame: through 72-hour post last dose ]
  19. Part 1 FE: AUC from time 0 to infinity (AUC0-inf) [ Time Frame: through 72-hour post last dose ]
  20. Part 1 DDI: Safety and tolerability measured by number of subjects who experience adverse events and potential clinically significant changes in safety labs, electrocardiograms (ECGs), physical examinations, and vital signs [ Time Frame: baseline through 7 days post last dose ]
  21. Part 1 DDI: Maximum plasma concentration (Cmax) of caffeine, bupropion, and midazolam with and without multiple oral doses of PTI-801 [ Time Frame: through 72-hours post dose ]
  22. Part 1 DDI: Area under the concentration-time curve from time 0 to time of last measurable concentration (AUClast) of caffeine, bupropion, and midazolam with and without multiple oral doses of PTI-801 [ Time Frame: through 72-hours post dose ]
  23. Part 1 DDI: Area under the concentration-time curve from time 0 to infinity (AUCinf) of caffeine, bupropion, and midazolam with and without multiple oral doses of PTI-801 [ Time Frame: through 72-hours post dose ]
  24. Part 2 CF: Safety and tolerability measured by number of subjects who experience adverse events and potential clinically significant changes in safety labs, electrocardiograms (ECGs), physical examinations, and vital signs [ Time Frame: baseline through Day 21 ]

Secondary Outcome Measures :
  1. Part 1 FE: Safety and tolerability measured by number of subjects who experience adverse events and potential clinically significant changes in safety labs, electrocardiograms (ECGs), physical examinations, and vital signs [ Time Frame: baseline through 7 days post last dose ]
  2. Part 1 DDI: Apparent terminal half-life (t1/2) of multiple oral doses of PTI-801 with and without caffeine, bupropion, and midazolam [ Time Frame: through 72-hours post dose ]
  3. Part 1 DDI: Time to reach maximum plasma concentration (Tmax) of the metabolites of caffeine, bupropion, and midazolam after single oral doses of caffeine, bupropion, midazolam, and with and without multiple oral doses of PTI-801 [ Time Frame: through 72-hours post dose ]
  4. Part 1 DDI: Maximum plasma concentration (Cmax) of the metabolites of caffeine, bupropion, and midazolam after single oral doses of caffeine, bupropion, midazolam, and with and without multiple oral doses of PTI-801 [ Time Frame: through 72-hours post dose ]
  5. Part 1 DDI: AUClast of the metabolites of caffeine, bupropion, and midazolam after single oral doses of caffeine, bupropion, midazolam, and with and without multiple oral doses of PTI-801 [ Time Frame: through 72-hours post dose ]
  6. Part 1 DDI: Area under the concentration-time curve from time 0 to 24 hours post administration (AUC0-24) of multiple oral doses of PTI-801 with and without caffeine, bupropion, and midazolam [ Time Frame: through 72-hours post dose ]
  7. Part 1 DDI: Metabolite over parent ratio of Cmax of single oral doses of the metabolites of caffeine, bupropion, and midazolam after single oral doses of caffeine, bupropion, midazolam, and with and without multiple oral doses of PTI-801 [ Time Frame: through 72-hours post dose ]
  8. Part 1 DDI: Metabolite over parent ratio of AUClast of the metabolites of caffeine, bupropion, and midazolam after single oral doses of caffeine, bupropion, midazolam, and with and without multiple oral doses of PTI-801 [ Time Frame: through 72-hours post dose ]
  9. Part 1 DDI: Time to reach maximum plasma concentration (Tmax) of multiple oral doses of PTI-801 with and without caffeine, bupropion, and midazolam [ Time Frame: through 72-hours post dose ]
  10. Part 1 DDI: Maximum plasma concentration (Cmax) of multiple oral doses of PTI-801 with and without caffeine, bupropion, and midazolam [ Time Frame: through 72-hours post dose ]
  11. Part 1 DDI: Area under the concentration-time curve from time 0 to time of last measurable concentration (AUClast) of multiple oral doses of PTI-801 with and without caffeine, bupropion, and midazolam [ Time Frame: through 72-hours post dose ]
  12. Part 2 MAD CF: Apparent terminal half-life (t1/2) of multiple oral doses of PTI-801 with ivacaftor/lumacaftor [ Time Frame: Day 1 through Day 15 ]
  13. Part 2 MAD CF: Time to reach maximum plasma concentration (Tmax) of multiple oral doses of PTI-801 with ivacaftor/lumacaftor [ Time Frame: Day 1 through Day 15 ]
  14. Part 2 MAD CF: Maximum plasma concentration (Cmax) of multiple oral doses of PTI-801 with ivacaftor/lumacaftor [ Time Frame: Day 1 through Day 15 ]
  15. Part 2 MAD CF: Area under the concentration-time curve from time 0 to time of last measurable concentration (AUClast) of multiple oral doses of PTI-801 with ivacaftor/lumacaftor [ Time Frame: Day 1 through Day 15 ]
  16. Part 2 MAD CF: Apparent terminal half-life (t1/2) of ivacaftor/lumacaftor with multiple oral doses of PTI-801 [ Time Frame: Day 1 through Day 15 ]
  17. Part 2 MAD CF: Time to reach maximum plasma concentration (Tmax) of ivacaftor/lumacaftor with multiple oral doses of PTI-801 [ Time Frame: Day 1 through Day 15 ]
  18. Part 2 MAD CF: Area under the concentration-time curve from time 0 to time of last measurable concentration (AUClast) of ivacaftor/lumacaftor with multiple oral doses of PTI-801 [ Time Frame: Day 1 through Day 15 ]
  19. Part 2 MAD CF: Maximum plasma concentration (Cmax) of ivacaftor/lumacaftor with multiple oral doses of PTI-801 [ Time Frame: Day 1 through Day 15 ]
  20. Part 2 Cohort 4 CF: Apparent terminal half-life (t1/2) of multiple oral doses of PTI-801 with PTI-808 [ Time Frame: Day 1 through Day 15 ]
  21. Part 2 Cohort 4 CF: Time to reach maximum plasma concentration (Tmax) of multiple oral doses of PTI-801 with PTI-808 [ Time Frame: Day 1 through Day 15 ]
  22. Part 2 Cohort 4 CF: Maximum plasma concentration (Cmax) of multiple oral doses of PTI-801 with PTI-808 [ Time Frame: Day 1 through Day 15 ]
  23. Part 2 Cohort 4 CF: Area under the concentration-time curve from time 0 to time of last measurable concentration (AUClast) of multiple oral doses of PTI-801 with PTI-808 [ Time Frame: Day 1 through Day 15 ]
  24. Part 2 Cohort 4 CF: Apparent terminal half-life (t1/2) of multiple oral doses of PTI-808 with PTI-801 [ Time Frame: Day 1 through Day 15 ]
  25. Part 2 Cohort 4 CF: Time to reach maximum plasma concentration (Tmax) of multiple oral doses of PTI-808 with PTI-801 [ Time Frame: Day 1 through Day 15 ]
  26. Part 2 Cohort 4 CF: Maximum plasma concentration (Cmax) of multiple oral doses of PTI-808 with PTI-801 [ Time Frame: Day 1 through Day 15 ]
  27. Part 2 Cohort 4 CF: Area under the concentration-time curve from time 0 to time of last measurable concentration (AUClast) of multiple oral doses of PTI-808 with PTI-801 [ Time Frame: Day 1 through Day 15 ]
  28. Part 2 CF: change in forced expiratory volume in one second (FEV1) over time [ Time Frame: baseline through Day 21 ]

Other Outcome Measures:
  1. Part 1 SAD, MAD HV, and FE: The effect of PTI-801 on the QT interval as measured by holter monitoring [ Time Frame: baseline through 7 days post last dose ]
  2. Part 1: change in nasal epithelial mRNA and protein expression over time [ Time Frame: baseline through 7 days post last dose ]
  3. Part 2 CF: change in sweat chloride over time [ Time Frame: baseline through Day 21 ]
  4. Part 2 CF: change in nasal epithelial mRNA and protein expression over time [ Time Frame: baseline through Day 21 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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
  • 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 MAD Cohorts Additional Inclusion Criterion:

  • Stable on ivacaftor/lumacaftor dosing for both label indication and per label dosing for a minimum of 3 months at the time of randomization

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

Part 2 Cohort 4 Additional Exclusion Criterion:

  • Currently taking or has taken a CFTR modulator within 30 days prior to initial dose of study drugs

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03140527


Contacts
Contact: Proteostasis Clinical Trials 1-866-223-3262 pticlinicaltrials@proteostasis.com

  Show 36 Study Locations
Sponsors and Collaborators
Proteostasis Therapeutics, Inc.

Additional Information:
Responsible Party: Proteostasis Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03140527     History of Changes
Other Study ID Numbers: PTI-801-01
First Posted: May 4, 2017    Key Record Dates
Last Update Posted: May 1, 2018
Last Verified: April 2018

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

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases