Cystic FibrosisPhase 2NCT03591094
Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis
A Phase 2 study testing PTI-428 in people with Cystic Fibrosis, run by Proteostasis Therapeutics, Inc. at 26 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- PTI-428
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 26 sitesUnited States
- Study dates
- 21 Aug 2018to 18 Feb 2019
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
26 sites in 1 country.
26 sites in 1 country
United States26 sites
Akron Children's Hospital
Akron, Ohio, 44308
Central Florida Pulmonary Group
Altamonte Springs, Florida, 32701
Michigan Medicine, University of Michigan
Ann Arbor, Michigan, 48109
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Massachusetts General Hospital
Boston, Massachusetts, 02114
Boston Children's Hospital
Boston, Massachusetts, 02115
Medical University of South Carolina
Charleston, South Carolina, 29425
Northwestern University
Chicago, Illinois, 60611
Nationwide Children's Hospital
Columbus, Ohio, 43205
National Jewish Health
Denver, Colorado, 80206
Duke University Medical Center
Durham, North Carolina, 27710
University of Iowa, Roy J and Lucille A Carver College of Medicine
Iowa City, Iowa, 52242
Children's Mercy Hospital
Kansas City, Missouri, 64108
Universitey of Louisville, Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, 40202
Dartmouth Hitchcock Medical Center
Manchester, New Hampshire, 03104
University of Minnesota
Minneapolis, Minnesota, 55455
Mount Sinai Beth Israel
New York, New York, 10003
Columbia University Medical Center
New York, New York, 10032
Cystic Fibrosis Center, Children's Hospital of Illinois at OSF Saint Francis Medical Center
Peoria, Illinois, 61637
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Maine Medical Center
Portland, Maine, 04102
University of Utah
Salt Lake City, Utah, 84132
Stanford University
Stanford, California, 94305
University of Arizona
Tucson, Arizona, 85724
The University of Texas Health Science Center at Tyler - Center for Clinical Research
Tyler, Texas, 75708
New York Medical College
Valhalla, New York, 10595
About this study
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
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.
- Conditions
- Cystic Fibrosis
- Being tested
- PTI-428, Placebo
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PTI-428Drug
Active
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| PTI-428 dose level 1 | Active comparator | Not described Receives: Drug: PTI-428 |
| PTI-428 dose level 2 | Active comparator | Not described Receives: Drug: PTI-428 |
| Placebo PTI-428 | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with treatment-emergent adverse events (TEAEs)
Safety and tolerability will be assessed by adverse events (AEs), safety labs, electrocardiograms (ECGs), physical examinations and vital signs.
Measured: Baseline through Day 42
Secondary outcomes (5)
Maximum plasma concentration (Cmax)
Measured: 28 days
Time of Cmax (Tmax)
Measured: 28 days
Area under the concentration time curve from time 0 to time of last measurable concentration (AUC0-t)
Measured: 28 days
Change in FEV1 over time
Measured: Baseline through Day 42
Change in sweat chloride over time
Measured: Baseline through Day 42
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2018
- Study start21 Aug 2018
- Primary completion18 Feb 2019
- Study completion18 Feb 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Proteostasis Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03591094
- Sponsor's protocol ID
- PTI-428-06
- Status last verified
- Feb 2020
- First submitted
- 6 Jul 2018
- Last updated
- 27 Feb 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03591094
- Acronym / sponsor ID
- PTI-428-06
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Proteostasis Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 21 Aug 2018 (actual)
- Primary completion
- 18 Feb 2019 (actual)
- Study completion
- 18 Feb 2019 (actual)
- First posted
- 18 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| PTI-428 dose level 1 | Active comparator | Drug: PTI-428 |
| PTI-428 dose level 2 | Active comparator | Drug: PTI-428 |
| Placebo PTI-428 | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PTI-428Active | Drug | PTI-428 dose level 1, PTI-428 dose level 2 |
| PlaceboPlacebo | Drug | Placebo PTI-428 |
Primary outcome
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.
5 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2020.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
26 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PTI-428 in people with Cystic Fibrosis, run by Proteostasis Therapeutics, Inc. at 26 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of subjects with treatment-emergent adverse events (TEAEs) (time frame: Baseline through Day 42). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Feb 2020.