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

• Register: ClinicalTrials.gov
• Last refreshed on: 11 October 2021
• 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: October 10, 2018
• Target sample size: 166
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/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: Michael W Konstan, MD
• Affiliation: Rainbow Babies and Children's Hospital/ University Hospitals Cleveland Medical Center

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

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]

The safety and tolerability of LAU-7b will be assessed by the incidence of treatment emergent adverse events compared to placebo [Time Frame: From Baseline to 28 weeks]

Secondary Outcome(s)

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 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 28 weeks]

The change from baseline of systemic markers of inflammation in blood [Time Frame: From baseline to 28 weeks]

The absolute and relative (%) change in FEV1 percent predicted at 3, 7, 11, 15 and 28 weeks into the trial [Time Frame: From baseline to 3, 7, 11, 15 and 28 weeks into the trial]

The incidence of protocol-defined pulmonary exacerbation [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]

The change from screening of the body weight and calculated 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 Weeks 11 and 24]

The time to first protocol-defined pulmonary exacerbation [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