Study of LAU-7b in the Treatment of Cystic Fibrosis in Adults

• Register: ClinicalTrials.gov
• Last refreshed on: 14 October 2024
• Main ID: NCT03265288
• Date of registration: 21/02/2017
• Prospective Registration: Yes
• Primary sponsor: Laurent Pharmaceuticals Inc.
• Public title: Study of LAU-7b in the Treatment of Cystic Fibrosis in Adults APPLAUD
• 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
• Date of first enrolment: November 5, 2018
• Target sample size: 166
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03265288
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Australia, Canada, United States

Contacts

• Name: Larry C Lands, MD PhD
• Affiliation: McGill Uinversity Health Centre

• Name: Michael W Konstan, MD
• Affiliation: Rainbow Babies and Children's Hospital/ University Hospitals Cleveland Medical Center

Key inclusion & exclusion criteria

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.

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.


Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Placebo oral capsule

Drug: LAU-7b

Primary Outcome(s)

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1%) [Time Frame: From baseline to 24 weeks]

Summary of Treatment Emergent Adverse Events With = 10% Incidence [Time Frame: From Baseline to 28 weeks]

Secondary Outcome(s)

The Change From Screening of the Body Weight [Time Frame: From screening to 28 weeks]

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]

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 Screening of the Body Mass Index (BMI) [Time Frame: From screening to 28 weeks]

The Number Per Subject of Protocol-Defined Pulmonary Exacerbations (PEx) During the Trial [Time Frame: From baseline to 28 weeks]

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 Overall Change From Screening of the Pseudomonas Aeruginosa Density (Colony Forming Units) in the Sputum [Time Frame: From screening to Week 24]

The Change From Baseline of Systemic Markers of Inflammation in Blood [Time Frame: Change from baseline to Week 24]

The Change in Metabolipidomic Profile and in Markers of Oxidative Stress in Blood [Time Frame: From baseline to 24 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]

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]

The Time to First Protocol-Defined Pulmonary Exacerbation [Time Frame: From baseline to 28 weeks]

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 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]

Secondary ID(s)

LAU-14-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Cystic Fibrosis Foundation

Results

• Results available: Yes
• Date Posted: 09/10/2024
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03265288