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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03265288




Registration number
NCT03265288
Ethics application status
Date submitted
21/02/2017
Date registered
29/08/2017

Titles & IDs
Public title
Study of LAU-7b in the Treatment of Cystic Fibrosis in Adults
Scientific 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
Secondary ID [1] 0 0
LAU-14-01
Universal Trial Number (UTN)
Trial acronym
APPLAUD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - LAU-7b
Treatment: Drugs - Placebo oral capsule

Experimental: LAU-7b - Active drug fenretinide (as LAU-7b capsules)

Placebo comparator: Placebo - Placebo oral capsule (as inactive capsules identical to active arm)


Treatment: Drugs: LAU-7b
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.

Treatment: Drugs: Placebo oral capsule
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1%)
Timepoint [1] 0 0
From baseline to 24 weeks
Primary outcome [2] 0 0
Summary of Treatment Emergent Adverse Events With = 10% Incidence
Timepoint [2] 0 0
From Baseline to 28 weeks
Secondary outcome [1] 0 0
The Proportion of Patients Achieving Normalization of the Arachidonic Acid, Docosahexaenoic Acid and Their Ratio in Phospholipids
Timepoint [1] 0 0
From baseline to 28 weeks
Secondary outcome [2] 0 0
The Absolute Change in FEV1 Percent Predicted at 3, 7, 11, 15, 24 and 28 Weeks Into the Trial
Timepoint [2] 0 0
From baseline to 3, 7, 11, 15, 24 and 28 weeks into the trial
Secondary outcome [3] 0 0
The Relative (%) Change in FEV1 Percent Predicted at 3, 7, 11, 15, 24 and 28 Weeks Into the Trial
Timepoint [3] 0 0
From baseline to 3, 7, 11, 15, 24 and 28 weeks into the trial
Secondary outcome [4] 0 0
The Time to First Protocol-Defined Pulmonary Exacerbation
Timepoint [4] 0 0
From baseline to 28 weeks
Secondary outcome [5] 0 0
The Number Per Subject of Protocol-Defined Pulmonary Exacerbations (PEx) During the Trial
Timepoint [5] 0 0
From baseline to 28 weeks
Secondary outcome [6] 0 0
The Time to First Change and Usage of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin)
Timepoint [6] 0 0
From baseline to 28 weeks
Secondary outcome [7] 0 0
Usage (Number of Antibiotic Treatments) of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin)
Timepoint [7] 0 0
From baseline to 28 weeks
Secondary outcome [8] 0 0
Usage (Days) of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin)
Timepoint [8] 0 0
From baseline to 28 weeks
Secondary outcome [9] 0 0
The Change From Baseline of Systemic Markers of Inflammation in Blood
Timepoint [9] 0 0
Change from baseline to Week 24
Secondary outcome [10] 0 0
The Change From Screening of the Body Weight
Timepoint [10] 0 0
From screening to 28 weeks
Secondary outcome [11] 0 0
The Change From Screening of the Body Mass Index (BMI)
Timepoint [11] 0 0
From screening to 28 weeks
Secondary outcome [12] 0 0
The Overall Change From Screening of the Pseudomonas Aeruginosa Density (Colony Forming Units) in the Sputum
Timepoint [12] 0 0
From screening to Week 24
Secondary outcome [13] 0 0
The Impact (From Baseline) on Overall Health, Daily Life, Perceived Well-being and Symptoms Measured With the Cystic Fibrosis Questionnaire-Revised (CFQ-R)
Timepoint [13] 0 0
From baseline to 24 weeks
Secondary outcome [14] 0 0
The Change in Metabolipidomic Profile and in Markers of Oxidative Stress in Blood
Timepoint [14] 0 0
From baseline to 24 weeks

Eligibility
Key inclusion criteria
* 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 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;
* 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/11/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
15/09/2021
Sample size
Target
Accrual to date
Final
166
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Respiratory Medicine, John Hunter Hospital - New Lambton Heights
Recruitment hospital [2] 0 0
Department of Respiratory Medicine, Royal Prince Alfred Hospital - Sydney
Recruitment hospital [3] 0 0
Department of Respiratory and Sleep Medicine, Westmead Hospital - Westmead
Recruitment hospital [4] 0 0
Mater Misericordiae Ltd - Brisbane
Recruitment hospital [5] 0 0
Monash Lung and Sleep, Monash Health - Clayton
Recruitment hospital [6] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [7] 0 0
Institute of Respiratory Health, Harry Perkins Institute - Nedlands
Recruitment postcode(s) [1] 0 0
2305 - New Lambton Heights
Recruitment postcode(s) [2] 0 0
2050 - Sydney
Recruitment postcode(s) [3] 0 0
2145 - Westmead
Recruitment postcode(s) [4] 0 0
4101 - Brisbane
Recruitment postcode(s) [5] 0 0
3168 - Clayton
Recruitment postcode(s) [6] 0 0
3004 - Melbourne
Recruitment postcode(s) [7] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Idaho
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Kansas
Country [7] 0 0
United States of America
State/province [7] 0 0
Maine
Country [8] 0 0
United States of America
State/province [8] 0 0
Michigan
Country [9] 0 0
United States of America
State/province [9] 0 0
Minnesota
Country [10] 0 0
United States of America
State/province [10] 0 0
Missouri
Country [11] 0 0
United States of America
State/province [11] 0 0
New Jersey
Country [12] 0 0
United States of America
State/province [12] 0 0
New York
Country [13] 0 0
United States of America
State/province [13] 0 0
Ohio
Country [14] 0 0
United States of America
State/province [14] 0 0
Oklahoma
Country [15] 0 0
United States of America
State/province [15] 0 0
Oregon
Country [16] 0 0
United States of America
State/province [16] 0 0
Pennsylvania
Country [17] 0 0
United States of America
State/province [17] 0 0
South Carolina
Country [18] 0 0
United States of America
State/province [18] 0 0
Utah
Country [19] 0 0
United States of America
State/province [19] 0 0
Virginia
Country [20] 0 0
United States of America
State/province [20] 0 0
Wisconsin
Country [21] 0 0
Canada
State/province [21] 0 0
British Columbia
Country [22] 0 0
Canada
State/province [22] 0 0
Ontario
Country [23] 0 0
Canada
State/province [23] 0 0
Quebec

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Laurent Pharmaceuticals Inc.
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Cystic Fibrosis Foundation
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Larry C Lands, MD PhD
Address 0 0
McGill Uinversity Health Centre
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03265288