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COMPLETED Phase 1

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

NCT03140527 · View on ClinicalTrials.gov ↗

Study 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. Approximately 76 subjects will be enrolled in Part 1. 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. Part 2 Cohorts 4 and Cohort 5 will enroll adult subjects with CF not currently receiving cystic fibrosis conductance regulator (CFTR) modulator therapy within 30 days prior to Day 1. Part 2 Cohort 6 will enroll adult subjects with cystic fibrosis on stable tezacaftor/ivacaftor background therapy. Approximately 104 subjects will be enrolled in Part 2.

Interventions

  • DRUG Placebo
  • DRUG PTI-808
  • DRUG PTI-801

Study Locations (20)

Florida

  • Central Florida Pulmonary Group — Altamonte Springs
  • University of Florida College of Medicine — Gainesville
  • University of Miami Health System — Miami

Illinois

  • Northwestern University Memorial Hospital — Chicago
  • OSF Saint Francis Medical Center — Peoria

Massachusetts

  • Massachusetts General Hospital — Boston
  • Boston Children's Hospital — Boston

New York

  • Columbia University Medical Center — New York
  • Mount Sinai Beth Israel — New York

Alabama

  • University of Alabama at Birmingham — Birmingham

Alaska

  • Providence Alaska Medical Center — Anchorage

California

  • Stanford University Medical Center — Stanford

Colorado

  • National Jewish Health — Denver

Trial Details

FieldValue
Enrollment Target 171 participants
Start Date 2017-04-10
Est. Completion 2020-02-27
Phase Phase 1

Sponsor

Proteostasis Therapeutics

3 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT03140527

The ClinicalTrials.gov registry entry for NCT03140527 describes a study currently listed as completed. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 171 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Proteostasis Therapeutics, which has 3 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

The record links to 2 conditions, with Cystic Fibrosis appearing as the primary indexed condition, and to 3 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT03140527 reports 20 study locations spanning 15 distinct geographic areas — top geographies include Florida, Illinois, Massachusetts. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

Frequently Asked Questions

What is clinical trial NCT03140527 about?

NCT03140527 is a clinical study titled "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". 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 ascendi...

What is the current status of trial NCT03140527?

This trial is currently completed. It is a Phase 1 study. The enrollment target is 171 participants. The study started on 2017-04-10. Estimated completion is 2020-02-27.

What conditions does trial NCT03140527 study?

This clinical trial studies the following conditions: Cystic Fibrosis, Healthy Volunteer. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT03140527?

The interventions under investigation include: Placebo (DRUG), PTI-808 (DRUG), PTI-801 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT03140527?

This trial is sponsored by Proteostasis Therapeutics, which has 3 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

Where is trial NCT03140527 being conducted?

This trial has 20 study locations across Alabama, Alaska, California, Colorado, Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial