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

• Status: Completed
• Trial end date: 2021-09-15
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Laurent Pharmaceuticals Inc.
• Collaborators: Cystic Fibrosis Foundation; Cystic Fibrosis Foundation Therapeutics
• Treatments: Fenretinide

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.