Cystic FibrosisPhase 1NCT03258424
Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background Therapy
A Phase 1 study testing PTI-428 in people with Cystic Fibrosis, run by Proteostasis Therapeutics, Inc. at 2 sites in United Kingdom.
Trial summary
The trial will consist of a single treatment group enrolling adult subjects with CF on background therapy with KALYDECO®. Approximately 16 subjects will be enrolled. 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
- 15 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- 28 Jul 2017to 3 Oct 2018
- Phase
- Phase 1
- 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.
- Stable on Kalydeco dosing for both label indication and per label dosing for a minimum of 3 months at the time of randomization
- Forced expiratory volume in 1 second (FEV1) 40-90% predicted.
- Non-smoker and non-tobacco user for a minimum of 30 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 30 days or 5 half-lives, whichever is longer, prior to Study Day 1.
- History of cancer within the past five years (excluding cervical CIS with curative therapy for at least one year prior to screening and non-melanoma skin cancer).
- History of organ transplantation.
- Any sinopulmonary infection or CF exacerbation requiring a change or addition of medication (including antibiotics) within 1 month of Study Day 1 or any other clinically significant infection as determined by the investigator within 1 month of Day 1.
- History of alcohol or drug abuse or dependence within 12 months of screening as determined by the Investigator.
- Male and female of child-bearing potential, unless they are using highly effective methods of contraception during participation in the clinical study and for 4 weeks after termination from study.
- Pregnant or nursing women.
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
Celerion
Belfast, BT9 6AD
Medicines Evaluation Unit
Manchester
About this study
Official title: A Phase I, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects With Cystic Fibrosis.
The trial will consist of a single treatment group enrolling adult subjects with CF on background therapy with KALYDECO®. Approximately 16 subjects will be enrolled.
Read the detailed description
The single treatment group will enroll adult subjects with CF currently on stable KALYDECO® background therapy for a minimum of 3 months at the time of randomization. Subjects will be randomized to either PTI-428 or placebo. Each dose will be administered once daily (QD) for a total of 14 Days. The subjects will continue treatment with KALYDECO® throughout the study.
- 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
PTI-428 or placebo will be given QD for 14 days.
PlaceboDrug
PTI-428 or placebo will be given QD for 14 days.
| Study group | Role | What they receive |
|---|---|---|
| PTI-428 | Active comparator | Subjects will receive once daily dosing of PTI-428 or placebo for 14 days. Receives: Drug: PTI-428 |
| Placebo | Placebo comparator | Subjects will receive once daily dosing of PTI-428 or placebo for 14 days. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs
Measured: Baseline through Day 21
Secondary outcomes (4)
t1/2 of multiple oral doses
Measured: Baseline through Day 14
Tmax of multiple oral doses
Measured: Baseline through Day 14
Cmax of multiple oral doses
Measured: Baseline through Day 14
AUC0-t of multiple oral doses
Measured: Baseline through Day 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2017
- Study start28 Jul 2017
- Primary completion3 Oct 2018
- Study completion3 Oct 2018
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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 15 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
- NCT03258424
- Sponsor's protocol ID
- PTI-428-05
- Status last verified
- Mar 2020
- First submitted
- 18 Aug 2017
- Last updated
- 26 Mar 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects With Cystic Fibrosis.
- ClinicalTrials.gov ID
- NCT03258424
- Acronym / sponsor ID
- PTI-428-05
- Conditions
- Cystic Fibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Proteostasis Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Jul 2017 (actual)
- Primary completion
- 3 Oct 2018 (actual)
- Study completion
- 3 Oct 2018 (actual)
- First posted
- 23 Aug 2017 (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 trial will consist of a single treatment group enrolling adult subjects with CF on background therapy with KALYDECO®. Approximately 16 subjects will be enrolled.
Detailed description
The single treatment group will enroll adult subjects with CF currently on stable KALYDECO® background therapy for a minimum of 3 months at the time of randomization. Subjects will be randomized to either PTI-428 or placebo. Each dose will be administered once daily (QD) for a total of 14 Days. The subjects will continue treatment with KALYDECO® throughout the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PTI-428 | Active comparator | Drug: PTI-428Subjects will receive once daily dosing of PTI-428 or placebo for 14 days. |
| Placebo | Placebo comparator | Drug: PlaceboSubjects will receive once daily dosing of PTI-428 or placebo for 14 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Confirmed diagnosis of CF.
- Stable on Kalydeco dosing for both label indication and per label dosing for a minimum of 3 months at the time of randomization
- Forced expiratory volume in 1 second (FEV1) 40-90% predicted.
- Non-smoker and non-tobacco user for a minimum of 30 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 30 days or 5 half-lives, whichever is longer, prior to Study Day 1.
- History of cancer within the past five years (excluding cervical CIS with curative therapy for at least one year prior to screening and non-melanoma skin cancer).
- History of organ transplantation.
- Any sinopulmonary infection or CF exacerbation requiring a change or addition of medication (including antibiotics) within 1 month of Study Day 1 or any other clinically significant infection as determined by the investigator within 1 month of Day 1.
- History of alcohol or drug abuse or dependence within 12 months of screening as determined by the Investigator.
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-428PTI-428 or placebo will be given QD for 14 days. | Drug | PTI-428 |
| PlaceboPTI-428 or placebo will be given QD for 14 days. | Drug | Placebo |
Primary outcome
Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs
Time frame: Baseline through Day 21
4 secondary outcomes
t1/2 of multiple oral doses
Time frame: Baseline through Day 14
Tmax of multiple oral doses
Time frame: Baseline through Day 14
Cmax of multiple oral doses
Time frame: Baseline through Day 14
AUC0-t of multiple oral doses
Time frame: Baseline through Day 14
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 26 Mar 2020.
- Individual participant dataSharing planned: Yes
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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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PTI-428 in people with Cystic Fibrosis, run by Proteostasis Therapeutics, Inc. at 2 sites in United Kingdom.
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 4 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 26 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United Kingdom. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs (time frame: Baseline through Day 21). 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 26 Mar 2020.