Overview
Study of LAU-7b in the Treatment of Cystic Fibrosis in Adults
Status:
Completed
Completed
Trial end date:
2021-09-15
2021-09-15
Target enrollment:
0
0
Participant gender:
All
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 2Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
Laurent Pharmaceuticals Inc.Collaborators:
Cystic Fibrosis Foundation
Cystic Fibrosis Foundation TherapeuticsTreatments:
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.