Cystic FibrosisPhase 1NCT01543191
A Safety and Tolerability Study OF PUR118 In Subjects With Cystic Fibrosis
A Phase 1 study testing PUR118 in people with Cystic Fibrosis, run by Pulmatrix Inc. at 1 site in United Kingdom.
Trial summary
The purpose of this study is to determine whether PUR118 is safe and tolerable in a population of subjects with Cystic Fibrosis. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- PUR118
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 10 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Feb 2012to Dec 2012
- 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 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Males or non-pregnant, non-lactating healthy female.
- Age 18 to 55 years of age (inclusive) on day of signing informed consent.
- Subject has a BMI of 17 or greater.
- Subject has a confirmed diagnosis of cystic fibrosis documented by a compatible clinical or radiographic presentation, and definitive accepted lab and clinical criteria (e.g., sweat chloride > 60 mEq/L or 2 disease causing CFTR mutations) OR is regularly attending a Consultant led clinic where the diagnosis of Cystic Fibrosis is not in doubt.
- Subject has an FEV1 ≥ 45% of predicted at screening.
- Subject has an oxygen saturation >92% on room air as determined by pulse oximetry at screening.
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- Subject is a non-smoker or ex-smoker has not smoked for at least six months prior to screening.
- Subject is otherwise medically stable without clinically significant abnormal screening results that depart from their usual baseline level of health as a subject with CF.
- Must be willing and able to communicate in English and participate in the whole study.
- Must provide written informed consent.
Who cannot join
- Subject has unstable lung disease, as defined by a change in medical regimen during the preceding 2-weeks, or a significant new finding on chest radiograph (pneumothorax, lobar/segmental collapse), or in the opinion of the Investigator, has had a decline in pulmonary status within the last year not considered a part of the usual, chronic progression of CF lung disease and part of their baseline health condition as a subject with CF.
- Subject has had an exacerbation of respiratory symptoms within the past 30 days that required initiation of a new or altered respiratory therapy.
- Subject had either an upper or lower respiratory illness within the 30 days prior to dosing days, or has symptoms from such an illness that have not resolved.
- Subject has a history of lung transplantation.
- Females of child bearing potential who are pregnant, or lactating. Females who are sexually active and either not surgically sterile or not willing to use an acceptable form of contraception
- Clinically significant abnormal biochemistry, haematology or urinalysis not consistent with CF as judged by the Investigator
- Failure to satisfy the Investigator of fitness to participate for any other reason.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1 Dose-escalation Study TO Evaluate the Safety and Tolerability of PUR118 and Placebo in Subjects With CF
The purpose of this study is to determine whether PUR118 is safe and tolerable in a population of subjects with Cystic Fibrosis.
- Conditions
- Cystic Fibrosis
- Being tested
- PUR118
- Keywords
- Lung Diseases, Respiratory Tract Diseases, Fibrosis
- Medical subject headings
- Cystic Fibrosis, Lung Diseases, Respiratory Tract Diseases, Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PUR118Drug
low dose and high dose for 3 doses, BID
| Study group | Role | What they receive |
|---|---|---|
| PUR118 | Experimental | Not described Receives: Drug: PUR118 |
| Placebo | Placebo comparator | Not described Receives: Drug: PUR118 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability
Changes in clinical signs and symptoms from physical examination and chest examination, changes in clinical safety laboratory tests, vital signs, spirometry, lung clearance index, 24-hour sputum weight and volume, oxygen saturation, ECG, telemetry, and adverse events.
Measured: 4-6 weeks
Secondary outcomes (1)
Pharmacodynamics
To assess impact of PUR118 on mobilization and expectorated volume of airway mucus secretions.
To evaluate impact of PUR118 on measurements of lung function, including ventilation inhomogeneity, by both spirometry and measurement of the lung clearance index (LCI).
To evaluate an impact of PUR118 on sputum, serum and plasma biomarkers related to airway inflammation.
Measured: 36 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2012 (estimated)
- Study startFeb 2012
- Primary completionDec 2012
- Study completionDec 2012
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
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pulmatrix Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Celerion
- Responsible party
- Sponsor
- Investigators
- Stephen Smith, MD, Principal investigator, Celerion
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01543191
- Sponsor's protocol ID
- 601-0009
- Status last verified
- Jul 2013
- First submitted
- 27 Feb 2012
- Last updated
- 19 Jul 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Dose-escalation Study TO Evaluate the Safety and Tolerability of PUR118 and Placebo in Subjects With CF
- ClinicalTrials.gov ID
- NCT01543191
- Acronym / sponsor ID
- 601-0009
- Conditions
- Cystic Fibrosis
- Keywords
- Lung Diseases, Respiratory Tract Diseases, Fibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Pulmatrix Inc.Industry (pharma, biotech, devices)
- Collaborators
- Celerion
- Study start
- Feb 2012
- Primary completion
- Dec 2012 (actual)
- Study completion
- Dec 2012 (actual)
- First posted
- 2 Mar 2012 (estimated)
- 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 purpose of this study is to determine whether PUR118 is safe and tolerable in a population of subjects with Cystic Fibrosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PUR118 | Experimental | Drug: PUR118 |
| Placebo | Placebo comparator | Drug: PUR118 |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Males or non-pregnant, non-lactating healthy female.
- Age 18 to 55 years of age (inclusive) on day of signing informed consent.
- Subject has a BMI of 17 or greater.
- Subject has a confirmed diagnosis of cystic fibrosis documented by a compatible clinical or radiographic presentation, and definitive accepted lab and clinical criteria (e.g., sweat chloride > 60 mEq/L or 2 disease causing CFTR mutations) OR is regularly attending a Consultant led clinic where the diagnosis of Cystic Fibrosis is not in doubt.
- Subject has an FEV1 ≥ 45% of predicted at screening.
Key exclusion criteria (7 listed)
- Subject has unstable lung disease, as defined by a change in medical regimen during the preceding 2-weeks, or a significant new finding on chest radiograph (pneumothorax, lobar/segmental collapse), or in the opinion of the Investigator, has had a decline in pulmonary status within the last year not considered a part of the usual, chronic progression of CF lung disease and part of their baseline health condition as a subject with CF.
- Subject has had an exacerbation of respiratory symptoms within the past 30 days that required initiation of a new or altered respiratory therapy.
- Subject had either an upper or lower respiratory illness within the 30 days prior to dosing days, or has symptoms from such an illness that have not resolved.
- Subject has a history of lung transplantation.
- Females of child bearing potential who are pregnant, or lactating. Females who are sexually active and either not surgically sterile or not willing to use an acceptable form of contraception
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 |
|---|---|---|
| PUR118low dose and high dose for 3 doses, BID | Drug | PUR118, Placebo |
Primary outcome
Safety and Tolerability
Time frame: 4-6 weeks
Changes in clinical signs and symptoms from physical examination and chest examination, changes in clinical safety laboratory tests, vital signs, spirometry, lung clearance index, 24-hour sputum weight and volume, oxygen saturation, ECG, telemetry, and adverse events.
1 secondary outcome
Pharmacodynamics
Time frame: 36 hours
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 19 Jul 2013.
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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.
Stephen Smith, MD principal investigator
Celerion
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PUR118 in people with Cystic Fibrosis, run by Pulmatrix Inc. at 1 site 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 10 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 19 Jul 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 (time frame: 4-6 weeks). 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 19 Jul 2013.