Cystic FibrosisPhase 2APPLAUDNCT03265288
Study of LAU-7b in the Treatment of Cystic Fibrosis in Adults
A Phase 2 study testing LAU-7b in people with Cystic Fibrosis, run by Laurent Pharmaceuticals Inc. at 40 sites in 3 countries.
Trial summary
An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF. 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?
- LAU-7b
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 166 participants
- Where
- 40 sitesin 3 countries
- Study dates
- 5 Nov 2018to 15 Sep 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Screening FEV1 between 40% and 100% predicted value for age, gender and height, in patients capable of properly performing the test;
- History of pulmonary exacerbation, defined as at least one (1) pulmonary exacerbation in the year prior to Screening which resulted in documented intravenous or Oral antibiotics;
- Patients are eligible independently of their history of pulmonary Pseudomonas aeruginosa (PsA) infection and their PsA status at screening;
- If taking Kalydeco® (ivacaftor), Orkambi® (ivacaftor/lumacaftor), Symdeko® (ivacaftor/tezacaftor) or other commercially available CFTR modulator products, patients must be taking it for a minimum of 3 months prior to screening if naïve to CFTR modulators and 1 month if switched from another CFTR modulator product and deemed to tolerate it;
- No change in CF and allowed systemic chronic therapy for a minimum of 5 weeks prior to randomization, of which 2 weeks minimum are prior to screening;
- Female patients of child bearing potential should be on highly effective contraceptive methods during the study;
- Male patients with spouse or partner of child bearing potential, or pregnant, are eligible if they use an appropriate method of contraception.
Who cannot join
- Pregnancy: due to the potential teratogenic effects of retinoids, pregnant women are NOT eligible;
- Breast milk feeding by study patient is NOT allowed;
- Clinically abnormal renal function: serum creatinine > 132 μM (1.5 mg/dL);
- Clinically abnormal liver function: Total bilirubin >1.5 x ULN (in the absence of demonstrated Gilbert's syndrome), alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) > 2.5 x ULN;
- Patients with plasma retinol levels below 0.7 µM;
- Presence of nyctalopia or hemeralopia at enrolment, or any other serious retinal, ophthalmological condition;
Show all 11Show fewer
- Presence of serious dermatological conditions at entry, including inflammatory or xerotic skin pathologies such as psoriasis or ichthyosis;
- Intake of chronic systemic steroids in the month prior to screening and during the study;
- History of acute infections (viral/bacterial/fungal) within 5 weeks prior to randomization, of which 2 weeks minimum are prior to screening, whether or not treated and resolved;
- Presence of infection with Burkholderia cepacia (including all species within the Burkholderia cepacia complex group, and Burkholderia gladioli) in the 12 months prior to screening;
- Patients with a confirmed diagnosis (as per the Cystic Fibrosis Foundation diagnostic criteria) of Allergic BronchoPulmonary Aspergillosis (ABPA) and actively being treated with corticosteroids and/or anti fungal agents.
Trial locations and contacts
40 sites in 3 countries.
40 sites in 3 countries
United States27 sites
Albany Medical College
Albany, New York, 12208
University of Michigan Health System
Ann Arbor, Michigan, 48109
St-Luke's CF Center of Idaho
Boise, Idaho, 83712
Medical University of South Carolina
Charleston, South Carolina, 29425
University Hospitals Cleveland Medical Center, Rainbow Babies and Children's Hospital
Cleveland, Ohio, 44106
Nationwide Children's Hospital
Columbus, Ohio, 43205
Wayne State University, Harper University Hospital
Detroit, Michigan, 48201
Division of pulmonary, critical care and sleep medicine, University of Florida
Gainesville, Florida, 32610
Memorial Healthcare System, Joe DiMaggio Children's Hospital Cystic Fibrosis & Pulmonary Center
Hollywood, Florida, 33021
Riley Hospital for Children
Indianapolis, Indiana, 46202
University of Kansas Medical Center
Kansas City, Kansas, 66103
Long Beach Memorial Medical Center
Long Beach, California, 90806
Children's Hospital Los Angeles
Los Angeles, California, 90227
Medical College of Wisconsin, Div of Pulmonary and Critical Care Medicine
Milwaukee, Wisconsin, 53226
The Minnesota Cystic Fibrosis Center, University of Minnesota
Minneapolis, Minnesota, 55455
Morristown Medical Center, NJ Adult Cystic Fibrosis Center
Morristown, New Jersey, 07962
Rutgers University Clinical Research Center, RW Johnson University Hospital
New Brunswick, New Jersey, 08901
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Avanza Medical Research Center
Pensacola, Florida, 32603
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Maine Medical Center Cystic Fibrosis Research
Portland, Maine, 04102
Cystic Fibrosis Center, Doernbecher Children's Hospital, Oregon Health & Science University
Portland, Oregon, 97239
Virginia Commonwealth University
Richmond, Virginia, 23298
UC Davis Medical Center, Division of Pulmonary & Critical Care Medicine
Sacramento, California, 95817
University of Utah
Salt Lake City, Utah, 84132
Washington University Medical School
St Louis, Missouri, 63110
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Australia7 sites
Mater Misericordiae Ltd
Brisbane, Queensland, 4101
Monash Lung and Sleep, Monash Health
Clayton, Victoria, 3168
The Alfred Hospital
Melbourne, Victoria, 3004
Institute of Respiratory Health, Harry Perkins Institute
Nedlands, Western Australia, 6009
Respiratory Medicine, John Hunter Hospital
New Lambton Heights, New South Wales, 2305
Department of Respiratory Medicine, Royal Prince Alfred Hospital
Sydney, New South Wales, 2050
Department of Respiratory and Sleep Medicine, Westmead Hospital
Westmead, New South Wales, 2145
Canada6 sites
Centre d'études cliniques CIUSS SLJ, Hôpital Chicoutimi
Chicoutimi, Quebec, G7H 5H6
Centre Hospitalier de l'Université de Montréal
Montreal, Quebec, H2X2P1
McGill University Health Center
Montreal, Quebec, H4A 3J1
The Ottawa Hospital Center for Practice-Changing Research
Ottawa, Ontario, K1H 8L6
Centre de recherche de l'institut Universitaire de Cardiologie et de Pneumologie de Québec
Québec, Quebec, G1V 4G5
Pacific Lung Research Institute at St. Paul's Hospital
Vancouver, British Columbia, V6Z 1Y6
About this study
Official title: APPLAUD: A Double-Blind, Randomized, Placebo-Controlled, Phase II Study of the Efficacy and Safety of LAU-7b in the Treatment of Cystic Fibrosis in Adults
An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF.
Read the detailed description
An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF. All patients will remain on their CF standard-of-care treatments over the trial duration.
The goal for the treatment with LAU-7b in CF is to preserve lung function by reducing the persistent inflammation in the lung and to improve its capacity to defend against resistant bacteria such as Pseudomonas aeruginosa.
The treatment regimen will consist of 6 consecutive "dosing cycles" of 21 days each, spaced by study drug-free periods of 7 days. A total of 136 eligible adult patients with CF will be randomized to receive 300 mg LAU-7b or placebo in a 1:1 ratio. The participation in the study will last about 7 months.
- Conditions
- Cystic Fibrosis
- Being tested
- LAU-7b, Placebo oral capsule
- Keywords
- Inflammation, Essential Fatty Acids, Inflammation resolution, Docosahexaenoic acid, Lung function
- Medical subject headings
- Cystic Fibrosis, Inflammation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LAU-7bDrug
Also called fenretinide
LAU-7b will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles.
Placebo oral capsuleDrug
Also called Placebo
Placebo will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles.
| Study group | Role | What they receive |
|---|---|---|
| LAU-7b | Experimental | Active drug fenretinide (as LAU-7b capsules) Receives: Drug: LAU-7b |
| Placebo | Placebo comparator | Placebo oral capsule (as inactive capsules identical to active arm) Receives: Drug: Placebo oral capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1%)
Standardized, serial FEV1 measurements were performed during the trial by the clinical sites using standardized spirometers. All spirometry measurements were centrally read and validated by the spirometry provider, Vitalograph. The outcome measure is presented using the Least Squares Mean at the Week 24 time point and the Least Squares Mean of all post-baseline time points through Week 24 averaged.
Measured: From baseline to 24 weeks
Summary of Treatment Emergent Adverse Events With ≥ 10% Incidence
This was assessed through adverse event monitoring at all visits, including spontaneously reported events and those obtained through serial probing of the subjects, and from safety laboratory tests
Measured: From Baseline to 28 weeks
Secondary outcomes (14)
The Proportion of Patients Achieving Normalization of the Arachidonic Acid, Docosahexaenoic Acid and Their Ratio in Phospholipids
Assessed through 4 blood sampling occasions during the trial. Plasma samples were analyzed using a validated LC/MS method and corrected for phospholipid content. Highest proportion of normalization during treatment was determined versus analyte ranges obtained from a group of 20 healthy, non-CF individuals.
Measured: From baseline to 28 weeks
The Absolute Change in FEV1 Percent Predicted at 3, 7, 11, 15, 24 and 28 Weeks Into the Trial
Standardized, serial FEV1 measurements were performed during the trial by the clinical sites using standardized spirometers. All spirometry measurements were centrally read and validated by the spirometry provider, Vitalograph.
Measured: From baseline to 3, 7, 11, 15, 24 and 28 weeks into the trial
The Relative (%) Change in FEV1 Percent Predicted at 3, 7, 11, 15, 24 and 28 Weeks Into the Trial
Standardized, serial FEV1 measurements were performed during the trial by the clinical sites using standardized spirometers. All spirometry measurements were centrally read and validated by the spirometry provider, Vitalograph.
Measured: From baseline to 3, 7, 11, 15, 24 and 28 weeks into the trial
The Time to First Protocol-Defined Pulmonary Exacerbation
Reports of IV antibiotics-treated pulmonary exacerbations during the trial that meet the Fuch's criteria and after the first treatment cycle.
Measured: From baseline to 28 weeks
The Number Per Subject of Protocol-Defined Pulmonary Exacerbations (PEx) During the Trial
The number per subject of Protocol-Defined IV antibiotics-treated pulmonary exacerbations (events) during the trial that meet the Fuch's criteria. Also presented are the number per subject of IV antibiotics-treated pulmonary exacerbations and combined number per subject of IV- or Oral antibiotics-treated pulmonary exacerbations during the trial. Excluded are exacerbations occurring during the first treatment cycle.
Measured: From baseline to 28 weeks
The Time to First Change and Usage of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin)
The time to first change and usage of IV antibiotics to treat pulmonary exacerbations during the trial. Excluded are exacerbations occurring during the first treatment cycle.
Measured: From baseline to 28 weeks
Usage (Number of Antibiotic Treatments) of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin)
Usage (number of antibiotic treatments per subject) of IV antibiotics to treat pulmonary exacerbations during the trial. Excluded are exacerbations occurring during the first treatment cycle.
Measured: From baseline to 28 weeks
Usage (Days) of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin)
Usage (days) of IV antibiotics to treat pulmonary exacerbations during the trial. Excluded are exacerbations occurring during the first treatment cycle.
Measured: From baseline to 28 weeks
The Change From Baseline of Systemic Markers of Inflammation in Blood
This was assessed through scheduled blood sampling during the trial on three occasions. Both ITT and PP populations results presented. Samples were analyzed using validated analytical methods.
Measured: Change from baseline to Week 24
The Change From Screening of the Body Weight
This was assessed through serial weighing during the trial. Measurements performed at clinical sites using calibrated balances.
Measured: From screening to 28 weeks
The Change From Screening of the Body Mass Index (BMI)
This was assessed through serial weighing during the trial and calculation of BMI. Measurements performed at clinical sites using calibrated balances.
Measured: From screening to 28 weeks
The Overall Change From Screening of the Pseudomonas Aeruginosa Density (Colony Forming Units) in the Sputum
This was assessed through induced sputum (and spontaneously obtained during COVID-19 pandemic) on 3 occasions during the trial. Samples were analyzed at a central laboratory. An area under the curve (AUC from baseline to Week 24 inclusive) of colony forming unit/mL is calculated.
Measured: From screening to Week 24
The Impact (From Baseline) on Overall Health, Daily Life, Perceived Well-being and Symptoms Measured With the Cystic Fibrosis Questionnaire-Revised (CFQ-R)
This was assessed through administration of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) at four planned times during the trial. The CFQ-R respiratory sub-score (range 0-100) was extracted and analyzed. The Minimum Clinically Important Difference (MCID) for the respiratory sub-score is 4 units. A higher score means a better outcome.
Measured: From baseline to 24 weeks
The Change in Metabolipidomic Profile and in Markers of Oxidative Stress in Blood
This was assessed through serial blood sampling during the trial. Samples were analyzed using validated methods at specialized laboratories.
Measured: From baseline to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Aug 2017
- Study start5 Nov 2018
- Primary completion15 Sep 2021
- Study completion15 Sep 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Model description
- Double-blind, randomized, parallel groups and placebo-controlled trial.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 166 participants (actual)
Sponsor and investigators
- Lead sponsor
- Laurent Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
- Investigators
- Larry C Lands, MD PhD, Study chair, McGill Uinversity Health CentreMichael W Konstan, MD, Study chair, Rainbow Babies and Children's Hospital/ University Hospitals Cleveland Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03265288
- Sponsor's protocol ID
- LAU-14-01
- Acronym
- APPLAUD
- Status last verified
- Oct 2024
- First submitted
- 21 Feb 2017
- Last updated
- 9 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- 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 9 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- APPLAUD: A Double-Blind, Randomized, Placebo-Controlled, Phase II Study of the Efficacy and Safety of LAU-7b in the Treatment of Cystic Fibrosis in Adults
- ClinicalTrials.gov ID
- NCT03265288
- Acronym / sponsor ID
- APPLAUD / LAU-14-01
- Conditions
- Cystic Fibrosis
- Keywords
- Inflammation, Essential Fatty Acids, Inflammation resolution, Docosahexaenoic acid, Lung function
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 166 participants (actual)
- Lead sponsor
- Laurent Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- 5 Nov 2018 (actual)
- Primary completion
- 15 Sep 2021 (actual)
- Study completion
- 15 Sep 2021 (actual)
- First posted
- 29 Aug 2017 (actual)
- Results
- Posted 9 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF.
Detailed description
An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF. All patients will remain on their CF standard-of-care treatments over the trial duration.
The goal for the treatment with LAU-7b in CF is to preserve lung function by reducing the persistent inflammation in the lung and to improve its capacity to defend against resistant bacteria such as Pseudomonas aeruginosa.
The treatment regimen will consist of 6 consecutive "dosing cycles" of 21 days each, spaced by study drug-free periods of 7 days. A total of 136 eligible adult patients with CF will be randomized to receive 300 mg LAU-7b or placebo in a 1:1 ratio. The participation in the study will last about 7 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Double-blind, randomized, parallel groups and placebo-controlled trial.
| Arm | Type | Receives |
|---|---|---|
| LAU-7b | Experimental | Drug: LAU-7bActive drug fenretinide (as LAU-7b capsules) |
| Placebo | Placebo comparator | Drug: Placebo oral capsulePlacebo oral capsule (as inactive capsules identical to active arm) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Screening FEV1 between 40% and 100% predicted value for age, gender and height, in patients capable of properly performing the test;
- History of pulmonary exacerbation, defined as at least one (1) pulmonary exacerbation in the year prior to Screening which resulted in documented intravenous or Oral antibiotics;
- Patients are eligible independently of their history of pulmonary Pseudomonas aeruginosa (PsA) infection and their PsA status at screening;
- If taking Kalydeco® (ivacaftor), Orkambi® (ivacaftor/lumacaftor), Symdeko® (ivacaftor/tezacaftor) or other commercially available CFTR modulator products, patients must be taking it for a minimum of 3 months prior to screening if naïve to CFTR modulators and 1 month if switched from another CFTR modulator product and deemed to tolerate it;
- No change in CF and allowed systemic chronic therapy for a minimum of 5 weeks prior to randomization, of which 2 weeks minimum are prior to screening;
Key exclusion criteria (11 listed)
- Pregnancy: due to the potential teratogenic effects of retinoids, pregnant women are NOT eligible;
- Breast milk feeding by study patient is NOT allowed;
- Clinically abnormal renal function: serum creatinine > 132 μM (1.5 mg/dL);
- Clinically abnormal liver function: Total bilirubin >1.5 x ULN (in the absence of demonstrated Gilbert's syndrome), alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) > 2.5 x ULN;
- Patients with plasma retinol levels below 0.7 µM;
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 |
|---|---|---|
| LAU-7bAlso: fenretinideLAU-7b will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles. | Drug | LAU-7b |
| Placebo oral capsuleAlso: PlaceboPlacebo will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles. | Drug | Placebo |
Primary outcome
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1%)
Time frame: From baseline to 24 weeks
Standardized, serial FEV1 measurements were performed during the trial by the clinical sites using standardized spirometers. All spirometry measurements were centrally read and validated by the spirometry provider, Vitalograph. The outcome measure is presented using the Least Squares Mean at the Week 24 time point and the Least Squares Mean of all post-baseline time points through Week 24 averaged.
Summary of Treatment Emergent Adverse Events With ≥ 10% Incidence
Time frame: From Baseline to 28 weeks
This was assessed through adverse event monitoring at all visits, including spontaneously reported events and those obtained through serial probing of the subjects, and from safety laboratory tests
14 secondary outcomes
The Proportion of Patients Achieving Normalization of the Arachidonic Acid, Docosahexaenoic Acid and Their Ratio in Phospholipids
Time frame: From baseline to 28 weeks
The Absolute Change in FEV1 Percent Predicted at 3, 7, 11, 15, 24 and 28 Weeks Into the Trial
Time frame: From baseline to 3, 7, 11, 15, 24 and 28 weeks into the trial
The Relative (%) Change in FEV1 Percent Predicted at 3, 7, 11, 15, 24 and 28 Weeks Into the Trial
Time frame: From baseline to 3, 7, 11, 15, 24 and 28 weeks into the trial
The Time to First Protocol-Defined Pulmonary Exacerbation
Time frame: From baseline to 28 weeks
The Number Per Subject of Protocol-Defined Pulmonary Exacerbations (PEx) During the Trial
Time frame: From baseline to 28 weeks
The Time to First Change and Usage of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin)
Time frame: From baseline to 28 weeks
Usage (Number of Antibiotic Treatments) of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin)
Time frame: From baseline to 28 weeks
Usage (Days) of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin)
Time frame: From baseline to 28 weeks
The Change From Baseline of Systemic Markers of Inflammation in Blood
Time frame: Change from baseline to Week 24
The Change From Screening of the Body Weight
Time frame: From screening to 28 weeks
The Change From Screening of the Body Mass Index (BMI)
Time frame: From screening to 28 weeks
The Overall Change From Screening of the Pseudomonas Aeruginosa Density (Colony Forming Units) in the Sputum
Time frame: From screening to Week 24
The Impact (From Baseline) on Overall Health, Daily Life, Perceived Well-being and Symptoms Measured With the Cystic Fibrosis Questionnaire-Revised (CFQ-R)
Time frame: From baseline to 24 weeks
The Change in Metabolipidomic Profile and in Markers of Oxidative Stress in Blood
Time frame: From baseline to 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Larry C Lands, MD PhD study chair
McGill Uinversity Health Centre
Michael W Konstan, MD study chair
Rainbow Babies and Children's Hospital/ University Hospitals Cleveland Medical Center
40 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LAU-7b in people with Cystic Fibrosis, run by Laurent Pharmaceuticals Inc. at 40 sites in 3 countries.
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 7 inclusion and 11 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 9 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 40 sites in United States, Australia, 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. 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?
Yes. Results are posted in the registry (first posted 9 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1%) (time frame: From baseline to 24 weeks). The study lists 2 primary outcomes in all. 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 9 Oct 2024.