Cystic FibrosisPhase 1/2NCT03500263
Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic Fibrosis
A Phase 1/2 study testing PTI-808, PTI-801 and PTI-428 in people with Cystic Fibrosis, run by Proteostasis Therapeutics, Inc. at 4 sites in United Kingdom.
Trial summary
The study is a randomized, double-blind, placebo-controlled, study that will be conducted at multiple centers in subjects with Cystic Fibrosis (CF) who are either homozygous for the F508del mutation or heterozygous with at least copy of the F508del mutation. 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-808, PTI-801 and PTI-428
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 4 sitesUnited Kingdom
- Study dates
- 30 Jan 2018to 13 Mar 2019
- Phase
- Phase 1/2
- 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
- Cohorts 1,2 and 4: A Confirmed diagnosis of CF with the F508del/F508del CFTR genotype on record, along with clinical findings consistent with CF such as chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities
- Cohort 3 only: Confirmed diagnosis of CF with at least one copy of the F508del CFTR mutation on record, along with clinical findings consistent with CF, such as chronic sinopulmonary disease or gastrointestinal / nutritional abnormalities
- Forced expiratory volume in 1 second (FEV1) 40-90% predicted, inclusive
- Non-smoker and non-tobacco user for a minimum of 30 days prior to screening
- Cohort 3 only: A sweat chloride value of ≥60 mmol/L based on quantitative pilocarpine iontophoresis (as documented in the subject's medical record or as confirmed at the screening visit)
Who cannot join
- Currently taking or has taken a CFTR modulator within 30 days prior to initial dose of study drugs
- 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
- 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
4 sites in 1 country.
United Kingdom4 sites
Celerion
Belfast, BT9 6AD
Western General Hospital
Edinburgh, EH4 2XU
Queen Elizabeth University Hospital
Glasgow, G514TF
Medicines Evaluation Unit
Manchester
About this study
Official title: A Phase 1 / 2, Randomized, Double-Blind, Placebo-Controlled Study Designed to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic Fibrosis
The study is a randomized, double-blind, placebo-controlled, study that will be conducted at multiple centers in subjects with Cystic Fibrosis (CF) who are either homozygous for the F508del mutation or heterozygous with at least copy of the F508del mutation.
Read the detailed description
Study PTI-808-02 will enroll up to approximately 32 subjects. Subjects in the first cohort will receive PTI-808 and PTI-801. Following completion of Cohort 1, initiation of enrollment into subsequent cohorts will be based upon review and approval by the Safety Review Committee (SRC).
- Conditions
- Cystic Fibrosis
- Being tested
- PTI-808, Placebo, PTI-801, PTI-428
- 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-808Drug
Active
PlaceboDrug
Placebo
PTI-801Drug
Active
PTI-428Drug
Active
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Cohorts 1 and 2: PTI-808 Active Co-admin with PTI-801 Active | Active comparator | Subjects will be randomized to receive either PTI-808 co-administered with PTI-801 or placebos once-a-day for a total of 14 days. A follow up visit will occur on Day 21. Receives: Drug: PTI-808; Drug: PTI-801 |
| Cohorts 1 and 2: PTI-808 Placebo Co-admin with PTI-801 Placebo | Placebo comparator | Subjects will be randomized to receive either PTI-808 co-administered with PTI-801 or placebos once-a-day for a total of 14 days. A follow up visit will occur on Day 21. Receives: Drug: Placebo |
| Cohort 3 PTI-808 Active + PTI-801 Active + PTI-428 Active | Active comparator | Subjects will be randomized to receive either PTI-808 co-administered with PTI-801 and PTI-428 or placebos once-a-day for a total of 14 days. A follow up visit will occur on Day 21. Receives: Drug: PTI-808; Drug: PTI-801; Drug: PTI-428 |
| Cohort 3 PTI-808 placebo + PTI-801 Placebo + PTI-428 Placebo | Placebo comparator | Subjects will be randomized to receive either PTI-808 co-administered with PTI-801 and PTI-428 or placebos once-a-day for a total of 14 days. A follow up visit will occur on Day 21. Receives: Drug: Placebo |
| Cohort 4 PTI-808 Active + PTI-801 Active + PTI-428 Active | Active comparator | Subjects will be randomized to receive either PTI-808 co-administered with PTI-801 and PTI-428 or placebos once-a-day for 7 days immediately followed by PTI-808 co-administered with PTI-801 or placebos once-a-day for 7 days. A follow-up visit will occur on Day 21. Receives: Drug: PTI-808; Drug: PTI-801; Drug: PTI-428 |
| Cohort 4 PTI-808 Placebo + PTI-801 Placebo + PTI-428 Placebo | Placebo comparator | Subjects will be randomized to receive either PTI-808 co-administered with PTI-801 and PTI-428 or placebos once-a-day for 7 days immediately followed by PTI-808 co-administered with PTI-801 or placebos once-a-day for 7 days. A follow-up visit will occur on Day 21. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability measured by the number of subjects who experience adverse events and potentially significant clinical laboratory assessments, electrocardiography, physical examinations, vital signs.
Measured: Baseline through Day 21
Secondary outcomes (4)
Apparent terminal half-life (t1/2) of multiple oral doses of PTI-808 + PTI-801 and PTI-428 (cohorts 3 & 4 only)
Measured: Day 1 through 15
Time to reach maximum plasma concentration (Tmax) of multiple oral doses of PTI-808 + PTI-801 and PTI-428 (cohorts 3 & 4 only)
Measured: Day 1 through 15
Maximum plasma concentration (Cmax) of multiple oral doses of PTI-808 + PTI-801 and PTI-428 (cohorts 3 & 4 only)
Measured: Day 1 through 15
Change in FEV1 over time
Measured: Baseline through Day 21
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2018
- Study start30 Jan 2018
- Primary completion13 Mar 2019
- Study completion13 Mar 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 12 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
- NCT03500263
- Sponsor's protocol ID
- PTI-808-02
- Status last verified
- Mar 2020
- First submitted
- 9 Apr 2018
- Last updated
- 6 Apr 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 6 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 / 2, Randomized, Double-Blind, Placebo-Controlled Study Designed to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT03500263
- Acronym / sponsor ID
- PTI-808-02
- Conditions
- Cystic Fibrosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Proteostasis Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Jan 2018 (actual)
- Primary completion
- 13 Mar 2019 (actual)
- Study completion
- 13 Mar 2019 (actual)
- First posted
- 18 Apr 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 is a randomized, double-blind, placebo-controlled, study that will be conducted at multiple centers in subjects with Cystic Fibrosis (CF) who are either homozygous for the F508del mutation or heterozygous with at least copy of the F508del mutation.
Detailed description
Study PTI-808-02 will enroll up to approximately 32 subjects. Subjects in the first cohort will receive PTI-808 and PTI-801. Following completion of Cohort 1, initiation of enrollment into subsequent cohorts will be based upon review and approval by the Safety Review Committee (SRC).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Cohorts 1 and 2: PTI-808 Active Co-admin with PTI-801 Active | Active comparator | Drug: PTI-808, Drug: PTI-801Subjects will be randomized to receive either PTI-808 co-administered with PTI-801 or placebos once-a-day for a total of 14 days. A follow up visit will occur on Day 21. |
| Cohorts 1 and 2: PTI-808 Placebo Co-admin with PTI-801 Placebo | Placebo comparator | Drug: PlaceboSubjects will be randomized to receive either PTI-808 co-administered with PTI-801 or placebos once-a-day for a total of 14 days. A follow up visit will occur on Day 21. |
| Cohort 3 PTI-808 Active + PTI-801 Active + PTI-428 Active | Active comparator | Drug: PTI-808, Drug: PTI-801, Drug: PTI-428Subjects will be randomized to receive either PTI-808 co-administered with PTI-801 and PTI-428 or placebos once-a-day for a total of 14 days. A follow up visit will occur on Day 21. |
| Cohort 3 PTI-808 placebo + PTI-801 Placebo + PTI-428 Placebo | Placebo comparator | Drug: PlaceboSubjects will be randomized to receive either PTI-808 co-administered with PTI-801 and PTI-428 or placebos once-a-day for a total of 14 days. A follow up visit will occur on Day 21. |
| Cohort 4 PTI-808 Active + PTI-801 Active + PTI-428 Active | Active comparator | Drug: PTI-808, Drug: PTI-801, Drug: PTI-428Subjects will be randomized to receive either PTI-808 co-administered with PTI-801 and PTI-428 or placebos once-a-day for 7 days immediately followed by PTI-808 co-administered with PTI-801 or placebos once-a-day for 7 days. A follow-up visit will occur on Day 21. |
| Cohort 4 PTI-808 Placebo + PTI-801 Placebo + PTI-428 Placebo | Placebo comparator | Drug: PlaceboSubjects will be randomized to receive either PTI-808 co-administered with PTI-801 and PTI-428 or placebos once-a-day for 7 days immediately followed by PTI-808 co-administered with PTI-801 or placebos once-a-day for 7 days. A follow-up visit will occur on Day 21. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Cohorts 1,2 and 4: A Confirmed diagnosis of CF with the F508del/F508del CFTR genotype on record, along with clinical findings consistent with CF such as chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities
- Cohort 3 only: Confirmed diagnosis of CF with at least one copy of the F508del CFTR mutation on record, along with clinical findings consistent with CF, such as chronic sinopulmonary disease or gastrointestinal / nutritional abnormalities
- Forced expiratory volume in 1 second (FEV1) 40-90% predicted, inclusive
- Non-smoker and non-tobacco user for a minimum of 30 days prior to screening
- Cohort 3 only: A sweat chloride value of ≥60 mmol/L based on quantitative pilocarpine iontophoresis (as documented in the subject's medical record or as confirmed at the screening visit)
Key exclusion criteria (8 listed)
- Currently taking or has taken a CFTR modulator within 30 days prior to initial dose of study drugs
- 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
- 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
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-808Active | Drug | Cohort 3 PTI-808 Active + PTI-801 Active + PTI-428 Active, Cohort 4 PTI-808 Active + PTI-801 Active + PTI-428 Active, Cohorts 1 and 2: PTI-808 Active Co-admin with PTI-801 Active |
| PlaceboPlacebo | Drug | Cohort 3 PTI-808 placebo + PTI-801 Placebo + PTI-428 Placebo, Cohort 4 PTI-808 Placebo + PTI-801 Placebo + PTI-428 Placebo, Cohorts 1 and 2: PTI-808 Placebo Co-admin with PTI-801 Placebo |
| PTI-801Active | Drug | Cohort 3 PTI-808 Active + PTI-801 Active + PTI-428 Active, Cohort 4 PTI-808 Active + PTI-801 Active + PTI-428 Active, Cohorts 1 and 2: PTI-808 Active Co-admin with PTI-801 Active |
| PTI-428Active | Drug | Cohort 3 PTI-808 Active + PTI-801 Active + PTI-428 Active, Cohort 4 PTI-808 Active + PTI-801 Active + PTI-428 Active |
| PlaceboPlacebo | Drug | Cohort 3 PTI-808 placebo + PTI-801 Placebo + PTI-428 Placebo, Cohort 4 PTI-808 Placebo + PTI-801 Placebo + PTI-428 Placebo |
Primary outcome
Safety and tolerability measured by the number of subjects who experience adverse events and potentially significant clinical laboratory assessments, electrocardiography, physical examinations, vital signs.
Time frame: Baseline through Day 21
4 secondary outcomes
Apparent terminal half-life (t1/2) of multiple oral doses of PTI-808 + PTI-801 and PTI-428 (cohorts 3 & 4 only)
Time frame: Day 1 through 15
Time to reach maximum plasma concentration (Tmax) of multiple oral doses of PTI-808 + PTI-801 and PTI-428 (cohorts 3 & 4 only)
Time frame: Day 1 through 15
Maximum plasma concentration (Cmax) of multiple oral doses of PTI-808 + PTI-801 and PTI-428 (cohorts 3 & 4 only)
Time frame: Day 1 through 15
Change in FEV1 over time
Time frame: Baseline through Day 21
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 6 Apr 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.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PTI-808, PTI-801 and PTI-428 in people with Cystic Fibrosis, run by Proteostasis Therapeutics, Inc. at 4 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 5 inclusion and 8 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 6 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 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 combined 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.
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?
Safety and tolerability measured by the number of subjects who experience adverse events and potentially significant clinical laboratory assessments, electrocardiography, physical examinations, 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 6 Apr 2020.