A Study of Experimental Fenretinide (LAU-7b) for Cystic Fibrosis in Adults

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Age: 18 years or older
Gender:
Any
Healthy Volunteers: No
Keywords: cystic fibrosis
Type: Drug study, Phase 2
Target:
4 Participants
Investigator:
Description
Cystic fibrosis (CF) is a multi-system disease affecting the respiratory tract. It involves the lungs, nose, and sinuses, pancreas, liver, sweat glands, and reproductive organs. The lungs often experience ongoing infection (usually with bacteria called Pseudomonas aeruginosa). It sustains out-of-control inflammation that results in irreversible damage.

This is a research study of an experimental drug called fenretinide (LAU-7b). Experimental means that this drug has not been approved by the U.S. Food and Drug Administration (FDA).

The study drug (LAU-7b) is a derivative of Vitamin A. The FDA has approved the testing of fenretinide in this study. Researchers hope that treatment with fenretinide will preserve lung function. We hope it will reduce the persistent inflammation in the lung. We also hope it improves the lung's capacity to defend against bacteria such as Pseudomonas aeruginosa.

The main goals of this study are to determine if fenretinide:
Is safe and well tolerated
Improves lung function as measured by spirometry. Spirometry is a routinely performed test to measure lung function.
This study requires

The study team will make sure you qualify to take part in the study. Qualified participants will receive detailed information about the study. This may include a list of study-related tests and procedures.

Who can participate

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 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) or Symdeko® (ivacaftor/tezacaftor), patients must be taking it for a minimum of 3 months prior to screening, and deemed to tolerate it;
  • No change in CF and allowed systemic chronic therapy for a minimum of 1 month 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 1 month 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.
Benefits and risks of participating
BENEFITS:

We cannot promise any benefits to you as a result of taking part in this research study.


RISKS:

The study doctor will discuss the risks associated with taking part in this research study.
Compensation
You will not be paid to take part in this research study.
Resources
Schedule
Study duration and period
You will be in the study for approximately 7 months, including 8 study visits to the study center.
Recruitment period
From May 15, 2018
Location
UC Davis Medical Center
2315 Stockton Boulevard
Sacramento, CA 95817
Contact
John Tushinski
Research Topic
Conditions:
  • Cystic Fibrosis