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PTI-428 for Cystic Fibrosis

Phase 1 & 2
Waitlist Available
Research Sponsored by Proteostasis Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline through 24 hours post day 28 dose
Awards & highlights

Study Summary

This trial will consist of three arms: Part A, Part B, and Part C. Part A has two groups. The first group will enroll adult subjects with cystic fibrosis (CF) into a single ascending dose (SAD) treatment group. The second group will enroll adult subjects with CF, including those on background treatment with ORKAMBI® and those not on a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, into a multiple ascending dose (MAD) treatment group. Part B will enroll adult subjects with CF currently on stable ORKAMBI® background therapy for a minimum of 3 months into a Phase II treatment group consisting of two cohorts. Part C will enroll adult subjects with CF, including those on background treatment with KALYDECO® and those not on a CFTR modulator, into a Phase II treatment group consisting of three cohorts. Approximately 136 subjects will be enrolled.

Eligible Conditions
  • Cystic Fibrosis

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline through 24 hours post day 28 dose
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through 24 hours post day 28 dose for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
MAD: safety and tolerability as assessed by adverse events, pulomonary function tests, safety labs: hematology, chemistry, and urinalysis, electrocardiograms (ECGs), physical examinations, and vital signs
Part B and Part C Cohorts 2 and 3: safety and tolerability as assessed by adverse events, safety labs: hematology, chemistry, and urinalysis, electrocardiograms (ECGs), physical examinations, and vital signs
Part C Cohort 1: safety and tolerability as assessed by adverse events, safety labs: hematology, chemistry, and urinalysis, electrocardiograms (ECGs), physical examinations, and vital signs
+1 more
Secondary outcome measures
MAD: AUC0-t of multiple oral doses
MAD: Cmax of multiple oral doses
Oral cavity
+22 more
Other outcome measures
MAD: change in nasal epithelial CFTR mRNA and protein expression
MAD: change in sweat chloride over time
Part B and Part C Cohorts 2 and 3: change in CFQ-R over time
+6 more

Trial Design

3Treatment groups
Placebo Group
Group I: Part APlacebo Group2 Interventions
Part A consists of two treatment groups, SAD and MAD. Both treatment groups will consist of 3 cohorts. In SAD, subjects will receive a single dose of PTI-428 or placebo. In MAD, subjects will receive once daily dosing of PTI-428 or placebo for 7 days.
Group II: Part BPlacebo Group2 Interventions
Part B will consist of 2 cohorts. Subjects will receive once daily dosing of PTI-428 or placebo for 28 days.
Group III: Part CPlacebo Group2 Interventions
Part C will consist of 3 cohorts. Subjects will receive once daily dosing of PTI-428 or placebo for 28 days.

Find a Location

Who is running the clinical trial?

Proteostasis Therapeutics, Inc.Lead Sponsor
6 Previous Clinical Trials
511 Total Patients Enrolled
5 Trials studying Cystic Fibrosis
417 Patients Enrolled for Cystic Fibrosis

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis
2016. "Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02718495.
~6 spots leftby May 2025
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