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Cystic FibrosisPhase 2NCT03903913

Safety, Tolerability and Efficacy of S-1226 in Cystic Fibrosis and Non CF Bronchiectasis

A Phase 2 study testing S-1226 in people with Cystic Fibrosis and Bronchiectasis, run by SolAeroMed Inc. at 3 sites in Canada.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

This is a single center, open label, Phase IIa, multiple-ascending dose study in which subjects with mild to moderate Cystic Fibrosis and non CF bronchiectasis (n≤12) will be enrolled. Read more about this study

Who is it for?
Ages 14 to 50 years
Conditions the study is for
Cystic Fibrosis, Bronchiectasis
What is being tested?
S-1226
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
3 sitesCanada
Study dates
8 Aug 2019to Aug 2022
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 14 to 50 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Confirmed diagnosis of Cystic Fibrosis by sweat chloride concentration over 60 mosm/L and/or genotype analysis identifying two disease causing mutations
  • Male or Female over the age of 14
  • Followed in CF clinic at Foothills Hospital or Alberta Children's Hospital
  • Known lung involvement
  • Recommended airway clearance daily or twice daily
  • FEV1, % predicted, between 40-80%
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  • Evidence of Cystic Fibrosis lung involvement on Imaging, if available
  • Competent at providing reliable and accurate pulmonary function studies
  • Clinically stable - no pulmonary exacerbation of CF for which the patient has been hospitalized or received intravenous antibiotics for 4 weeks, no change in frequency of airway clearance
  • Male or Female including and over the age of 14
  • Known lung involvement
  • FEV1, % predicted, between 40-80%
  • Evidence of bronchiectasis on Imaging
  • Competent at providing reliable and accurate pulmonary function studies
  • Clinically stable - no pulmonary exacerbation for which the patient has been hospitalized or received intravenous antibiotics for 4 weeks

Who cannot join

  • Gross Hemoptysis in previous 4 weeks
  • Pneumothorax in previous 4 weeks
  • Pregnancy or of child bearing age without adequate contraception
  • Inability to produce acceptable and reproducible pulmonary function studies
  • Evidence of elevated PaCO2 in recent 6 months
  • Inability to perform airway clearance twice a day for the duration of the study
Show all 25Show fewer
  • History of anxiety/panic disorders
  • Breast-feeding subject.
  • Positive pregnancy test at screening.
  • Subject, who in the opinion of the Investigator, is mentally or emotionally unsuitable to participate, or unable/unwilling to comply with the study assessments.
  • Gross Hemoptysis in previous 4 weeks Pneumothorax in previous 4 weeks Pregnancy or of child bearing age without adequate contraception Inability to produce acceptable and reproducible pulmonary function studies Evidence of elevated PaCO2 in recent 6 months Inability to perform airway clearance twice a day for the duration of the study History of anxiety/panic disorders Breast-feeding subject. Positive pregnancy test at screening. Subject, who in the opinion of the Investigator, is mentally or emotionally unsuitable to participate, or unable/unwilling to comply with the study assessments.
  • Diagnosis of Cystic Fibrosis
  • Active tuberculosis and/or non-tuberculosis mycobacterial infection
  • Active allergic bronchopulmonary aspergillosis
  • Traction bronchiectasis due to pulmonary fibrosis
  • Gross Hemoptysis in previous 4 weeks
  • Pneumothorax in previous 4 weeks
  • Pregnancy or of child bearing age without adequate contraception
  • Inability to produce acceptable and reproducible pulmonary function studies
  • Evidence of elevated PaCO2 in recent 6 months
  • Inability to perform airway clearance twice a day for the duration of the study
  • History of anxiety/panic disorders
  • Breast-feeding subject.
  • Positive pregnancy test at screening.
  • Subject, who in the opinion of the Investigator, is mentally or emotionally unsuitable to participate, or unable/unwilling to comply with the study assessments.

Trial locations and contacts

3 sites in 1 country.

John Dennis, Phd contact

403-689-5989

jdennis@solaeromed.com

Francis Green, MBChB, MD contact

403-220-4514

fgreen@ucalgary.ca

Canada3 sites, 3 recruiting
  • Adult Cystic Fibrosis Clinic at the Foothills Hospital

    Calgary, Alberta, T2N 2T9

    Grishma Shrestha, MD, 4032203044, grishma.shrestha@ucalgary.ca

    Recruiting
  • Pediatric Cystic Fibrosis Clinic at Alberta Children's Hospital

    Calgary, Alberta, T3B 6A8

    Grishma Shrestha, MD, 4032203044, grishma.shrestha@ucalgary.ca

    Recruiting
  • Alberta Lung Function

    Calgary, Alberta

    Bryce Oslanski, RT, (403) 289-0723, Info@albertalungfunction.ca

    Mark Montgomery, MD

    Not yet recruiting

Other trials in Canada

About this study

Official title: A Phase IIa Open Label Study to Evaluate the Safety, Tolerability and Efficacy of S-1226 Administered by Nebulization in Subjects With Mild to Moderate Cystic Fibrosis and Non CF Bronchiectasis

This is a single center, open label, Phase IIa, multiple-ascending dose study in which subjects with mild to moderate Cystic Fibrosis and non CF bronchiectasis (n≤12) will be enrolled.

The safety and tolerability of S-1226 composed of PFOB with ascending doses of carbon dioxide (4%, 8%, and 12% CO2) administered twice daily in subjects with Cystic Fibrosis and non CF bronchiectasis will be evaluated. This will be followed by 5 day consecutive treatment using the highest tolerated dose of S1226. Participants can choose additional use of a further four weeks (28 days) of S-1226 therapy at home, using same or a lower tolerated dose.

Read the detailed description

This is a single center, open label, Phase IIa, multiple-ascending dose study in which subjects with mild to moderate Cystic Fibrosis and non CF bronchiectasis (n≤12) will be enrolled.

The study will consist of a screening period, a run-in and two dosing and evaluation periods (with a minimum two-day break in between) and a follow-up period.

The dosing and evaluation period of the study is divided into two consecutive components:

  1. Dose escalation study
  • This segment of the treatment period is designed to assess the safety and tolerability of escalating doses of S-1226 (4%, 8% and 12%) in those with mild-moderate CF and non CF bronchiectasis
  1. Daily dosing study - This segment of the treatment period is designed to assess the short term (5 day) safety and tolerability of twice daily administration of a fixed dose of S-1226 in subjects with mild-moderate CF and non CF bronchiectasis.

The primary aim of this study of S-1226 is to capture information to inform follow-up S-1226 clinical studies in CF and non CF bronchiectasis. Important in this regard will be data on safety, tolerability, dosing and efficacy. Specifically, regarding efficacy, the investigators want to examine for optimal dose ranging and magnitude of the effect to determine the power calculations for any follow-up studies and to determine which outcome measures best reflect the effects of the drug. Because the study is investigational in nature, it will be un-blinded and information will be analyzed as it is collected.

During the screening and the run-in period, the subjects will have safety assessments and all eligibility criteria confirmed. The run-in period involves meeting with the study subjects 1 - 2 weeks prior to the start of the treatment for a physical exam, concomitant medication review, spirometry and information regarding the study. The consent process will also take place at this stage.

The dose escalation period will include 3 days of twice daily treatment of the study drug with a washout period in between. The dose of CO2 in S-1226 will be sequentially escalated for the particular subject from the starting dose of 4% CO2. Planned subsequent dose levels are 8% and 12% CO2, although doses may be adjusted within this range based on safety and tolerability data from the completed dosing days. Subjects will not be dosed with a higher CO2 level until drug administration of the preceding dose level has been completed and safety data reviewed by the Safety Committee and a decision taken to proceed or not with the next sequential dose level.

There will be a washout period between the dose escalation and the daily dosing study. The daily dosing study will include 5 days of twice daily treatment of the study drug based on the results of the dose escalation study. Lung function tests will be performed before and after the treatment.

Subjects in the Home Extension Study will receive S-1226 twice daily for 28 consecutive days. Subjects will monitor daily symptom scores. Oxygen saturation values, as determined by pulse oximetry, will be monitored 2 minutes prior to therapy, during therapy and for up to 30 minutes post therapy. Pulmonary function studies will be assessed weekly by the designated respiratory therapist between 4-7 pm.

The follow-up phase will occur 10-14 days after completion of the final dose. This will involve a clinical exam, spirometry and completion of CFQ-R.

Being tested
S-1226
Keywords
Asthma, Carbon dioxide, Perfluorocarbon, Mucolytic
Medical subject headings
Cystic Fibrosis, Bronchiectasis, Asthma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • S-1226Drug

    Also called S-1226 (4%), S-1226 (8%), S-1226 (12%)

    The drug product S-1226 has two components delivered by inhalation. It is a mixture of perfluorooctylbromide (PFOB) nebulized with a medical gas mixture containing CO2. The PFOB component remains the same but the medical gas component contains 4%, 8% or 12% CO2.

Study groups in NCT03903913
Study groupRoleWhat they receive
Dose Escalation StudyExperimentalSubjects will receive up to three inhaled doses of S- 1226. Each dose will be administered over a 2-minute treatment period (with a minimum 2-minute break between treatments) with a nebulizer as follows. Three S-1226 formulations will be tested sequentially: * S-1226(4%) is composed of 3 mL PFOB and 4% CO2 * S-1226(8%) is composed of 3 mL PFOB and 8% CO2 * S-1226(12%) is composed of 3 mL PFOB and 12% CO2 Each formulation will be administered by inhalation for a period of 2 minutes.The nebulizer will be filled with 3 mL of PFOB. The nebulizer is connected to a compressed medical gas mixture consisting of either 4%, 8% or 12%, CO2. A driving pressure of 20 psi will be used, producing a gas flow rate of 9 L/min.
Receives: Drug: S-1226
Daily Dosing StudyExperimentalEligible subjects will receive S-1226 twice daily for 5 consecutive days. Subjects will receive up to three doses of S-1226 in the morning and afternoon, administered over three 2-minute periods with a Circulaire nebulizer, filled with one of the dosages outlined below, depending on the safety and tolerability data gathered from the dose escalation study for that particular subject. * S-1226(4%) is composed of 3 mL PFOB and 4% CO2 * S-1226(8%) is composed of 3 mL PFOB and 8% CO2 * S-1226(12%) is composed of 3 mL PFOB and 12% CO2
Receives: Drug: S-1226

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Treatment-emergent adverse events

    The number and percent of treatment emergent adverse events will be monitored, recorded and graded for severity and assigned attribution. The severity will be assessed in the following manner:

    Mild: Awareness of signs or symptoms, but are easily tolerated and are of minor irritant type, causing no limitations of usual activities. Signs or symptoms may require minor action or additional therapy.

    Moderate: Discomfort severe enough to cause some limitations of usual activities and may require action or additional therapy.

    Severe: Incapacitating with inability to carry out usual activities and requires specific action and/or medical attention. Note: the term severe is not the same as "serious", which is based on subject/event outcome or action criteria usually associated with events that pose a threat to a subject's life or functioning. Seriousness (not severity) serves as a guide for defining regulatory reporting obligations.

    Measured: 60 minutes

Secondary outcomes (2)
  1. Change from baseline in percent of predicted forced expiratory volume in 1 Second

    Change from baseline in percent of predicted forced expiratory volume in 1 Second (FEV1) will be calculated by measuring FEV1 at baseline and after treatment. FEV1 is defined as the volume of air exhaled from the lungs in the first second of a forced expiration.

    Measured: 60 minutes

  2. Change from baseline in the respiratory domain of Cystic Fibrosis Questionnaire -Revised

    Change from baseline in respiratory symptoms scores (subscale) of the Cystic Fibrosis Questionnaire -Revised (CFQ-R) will be measured. CFQ-R is a validated, disease specific, patient reported outcome measure used to evaluate the impact of CF on overall health, daily life, and perceived well-being and symptoms. This sub-scale ranges from scores of 0-100, with higher values representing a better outcome.

    Measured: Up to 4 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Apr 2019
  2. Study start8 Aug 2019
  3. Primary completionFeb 2022 (estimated)
  4. Study completionAug 2022 (estimated)

Resources

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
Non-randomised
Intervention model
Sequential
Model description
Single center, open label, Phase IIa, sequential, multiple-ascending dose study
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03903913
Sponsor's protocol ID
SAMi-03-1-01
Status last verified
Jun 2021
First submitted
15 Feb 2019
Last updated
16 Jun 2021
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Swystun V, Green FHY, Dennis JH, Rampakakis E, Lalli G, Fadayomi M, Chiu A, Shrestha G, El Shahat SG, Nelson DE, El Mays TY, Pieron CA, Leigh R. A phase IIa proof-of-concept, placebo-controlled, randomized, double-blind, crossover, single-dose clinical trial of a new class of bronchodilator for acute asthma. Trials. 2018 Jun 18;19(1):321. doi: 10.1186/s13063-018-2720-6.

    PubMed 29914544
  • Green FH, Leigh R, Fadayomi M, Lalli G, Chiu A, Shrestha G, ElShahat SG, Nelson DE, El Mays TY, Pieron CA, Dennis JH. A phase I, placebo-controlled, randomized, double-blind, single ascending dose-ranging study to evaluate the safety and tolerability of a novel biophysical bronchodilator (S-1226) administered by nebulization in healthy volunteers. Trials. 2016 Jul 28;17:361. doi: 10.1186/s13063-016-1489-8.

    PubMed 27464582
  • El Mays TY, Choudhury P, Leigh R, Koumoundouros E, Van der Velden J, Shrestha G, Pieron CA, Dennis JH, Green FH, Snibson KJ. Nebulized perflubron and carbon dioxide rapidly dilate constricted airways in an ovine model of allergic asthma. Respir Res. 2014 Sep 16;15(1):98. doi: 10.1186/s12931-014-0098-x.

    PubMed 25355286

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing S-1226 in people with Cystic Fibrosis and Bronchiectasis, run by SolAeroMed Inc. at 3 sites in Canada.

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 15 inclusion and 25 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: status not confirmed, as of the sponsor's last update on 16 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Canada. 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. 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?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact John Dennis, Phd (403-689-5989, jdennis@solaeromed.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Treatment-emergent adverse events (time frame: 60 minutes). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03903913, which also lists 3 linked publications.

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 16 Jun 2021.

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