Trial record 1 of 4 for:
Cystic Fibrosis | PTI-428 | Proteostasis [Lead] | Phase 2
Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT03591094 |
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Recruitment Status :
Completed
First Posted : July 18, 2018
Last Update Posted : February 27, 2020
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Sponsor:
Proteostasis Therapeutics, Inc.
Information provided by (Responsible Party):
Proteostasis Therapeutics, Inc.
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Brief Summary:
The study population is comprised of adult subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation and are currently receiving background treatment with tezacaftor/ivacaftor for a minimum of 1 month prior to Day 1. The planned sample size is approximately 40 subjects. 20 subjects will be assigned to PTI-428 dose level 1 or placebo and 20 subjects will be assigned to PTI-428 dose level 2 or placebo. At each dose level, subjects will be randomized at a 3:1 randomization ratio. Subjects will receive once daily oral doses of PTI-428 or placebo for 28 days, while the subjects continue to receive background treatment with tezacaftor/ivacaftor per product label. The study drug administration period will be followed by a 14-day safety follow-up period.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Cystic Fibrosis | Drug: PTI-428 Drug: Placebo | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 40 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect of PTI-428 in Subjects With Cystic Fibrosis |
| Actual Study Start Date : | August 21, 2018 |
| Actual Primary Completion Date : | February 18, 2019 |
| Actual Study Completion Date : | February 18, 2019 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Cystic fibrosis
MedlinePlus related topics:
Cystic Fibrosis
| Arm | Intervention/treatment |
|---|---|
| Active Comparator: PTI-428 dose level 1 |
Drug: PTI-428
Active |
| Active Comparator: PTI-428 dose level 2 |
Drug: PTI-428
Active |
| Placebo Comparator: Placebo PTI-428 |
Drug: Placebo
Placebo |
Primary Outcome Measures :
- Number of subjects with treatment-emergent adverse events (TEAEs) [ Time Frame: Baseline through Day 42 ]Safety and tolerability will be assessed by adverse events (AEs), safety labs, electrocardiograms (ECGs), physical examinations and vital signs.
Secondary Outcome Measures :
- Maximum plasma concentration (Cmax) [ Time Frame: 28 days ]
- Time of Cmax (Tmax) [ Time Frame: 28 days ]
- Area under the concentration time curve from time 0 to time of last measurable concentration (AUC0-t) [ Time Frame: 28 days ]
- Change in FEV1 over time [ Time Frame: Baseline through Day 42 ]
- Change in sweat chloride over time [ Time Frame: Baseline through Day 42 ]
Other Outcome Measures:
- Change in nasal epithelial CFTR mRNA and protein expression over time [ Time Frame: Baseline through Day 42 ]
- Change in CFQ-R over time [ Time Frame: Baseline through Day 42 ]
- Cmax of PTI-428 metabolites, if applicable [ Time Frame: 28 days ]
- Tmax of PTI-428 metabolites, if applicable [ Time Frame: 28 days ]
- AUC0-t of PTI-428 metabolites, if applicable [ Time Frame: 28 days ]
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Confirmed diagnosis of CF with the F508del/F508del genotype on record
- On tezacaftor/ivacaftor dosing for both label indication and per label dosing for a minimum of 1 month on Day 1
- Forced expiratory volume in 1 second (FEV1) 40-90% predicted, inclusive
- Clinically stable with no significant changes in health status within 14 days of Day 1
- Non-smoker and non-tobacco user for a minimum of 28 days prior to screening and for the duration of the study
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
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): NCT03591094
Locations
Show 26 study locations
Sponsors and Collaborators
Proteostasis Therapeutics, Inc.
| Responsible Party: | Proteostasis Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT03591094 |
| Other Study ID Numbers: |
PTI-428-06 |
| First Posted: | July 18, 2018 Key Record Dates |
| Last Update Posted: | February 27, 2020 |
| Last Verified: | February 2020 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Additional relevant MeSH terms:
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Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases |
Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases |