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

NCT ID: NCT03265288

Last Updated: 2024-10-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 166 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-11-05
• Study Completion Date: 2021-09-15

Brief 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.

Detailed Description

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. All patients will remain on their CF standard-of-care treatments over the trial duration.

The goal for the treatment with LAU-7b in CF is to preserve lung function by reducing the persistent inflammation in the lung and to improve its capacity to defend against resistant bacteria such as Pseudomonas aeruginosa.

The treatment regimen will consist of 6 consecutive "dosing cycles" of 21 days each, spaced by study drug-free periods of 7 days. A total of 136 eligible adult patients with CF will be randomized to receive 300 mg LAU-7b or placebo in a 1:1 ratio. The participation in the study will last about 7 months.

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

LAU-7b

Active drug fenretinide (as LAU-7b capsules)

Group Type: EXPERIMENTAL

LAU-7b

Intervention Type: DRUG

LAU-7b will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles.

Placebo

Placebo oral capsule (as inactive capsules identical to active arm)

Group Type: PLACEBO_COMPARATOR

Placebo oral capsule

Intervention Type: DRUG

Placebo will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles.

Interventions

LAU-7b

LAU-7b will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles.

Intervention Type: DRUG

Placebo oral capsule

Placebo will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles.

Intervention Type: DRUG

Other Intervention Names

fenretinide; Placebo

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cystic Fibrosis Foundation

OTHER

Sponsor Role: collaborator

Laurent Pharmaceuticals Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Larry C Lands, MD PhD

Role: STUDY_CHAIR

McGill Uinversity Health Centre

Michael W Konstan, MD

Role: STUDY_CHAIR

Rainbow Babies and Children's Hospital/ University Hospitals Cleveland Medical Center

Locations

Long Beach Memorial Medical Center

Long Beach, California, United States

Children's Hospital Los Angeles

Los Angeles, California, United States

UC Davis Medical Center, Division of Pulmonary & Critical Care Medicine

Sacramento, California, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Division of pulmonary, critical care and sleep medicine, University of Florida

Gainesville, Florida, United States

Memorial Healthcare System, Joe DiMaggio Children's Hospital Cystic Fibrosis & Pulmonary Center

Hollywood, Florida, United States

Avanza Medical Research Center

Pensacola, Florida, United States

St-Luke's CF Center of Idaho

Boise, Idaho, United States

Riley Hospital for Children

Indianapolis, Indiana, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

Maine Medical Center Cystic Fibrosis Research

Portland, Maine, United States

University of Michigan Health System

Ann Arbor, Michigan, United States

Wayne State University, Harper University Hospital

Detroit, Michigan, United States

The Minnesota Cystic Fibrosis Center, University of Minnesota

Minneapolis, Minnesota, United States

Washington University Medical School

St Louis, Missouri, United States

Morristown Medical Center, NJ Adult Cystic Fibrosis Center

Morristown, New Jersey, United States

Rutgers University Clinical Research Center, RW Johnson University Hospital

New Brunswick, New Jersey, United States

Albany Medical College

Albany, New York, United States

University Hospitals Cleveland Medical Center, Rainbow Babies and Children's Hospital

Cleveland, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States

Cystic Fibrosis Center, Doernbecher Children's Hospital, Oregon Health & Science University

Portland, Oregon, United States

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Medical University of South Carolina

Charleston, South Carolina, United States

University of Utah

Salt Lake City, Utah, United States

Virginia Commonwealth University

Richmond, Virginia, United States

Medical College of Wisconsin, Div of Pulmonary and Critical Care Medicine

Milwaukee, Wisconsin, United States

Respiratory Medicine, John Hunter Hospital

New Lambton Heights, New South Wales, Australia

Department of Respiratory Medicine, Royal Prince Alfred Hospital

Sydney, New South Wales, Australia

Department of Respiratory and Sleep Medicine, Westmead Hospital

Westmead, New South Wales, Australia

Mater Misericordiae Ltd

Brisbane, Queensland, Australia

Monash Lung and Sleep, Monash Health

Clayton, Victoria, Australia

The Alfred Hospital

Melbourne, Victoria, Australia

Institute of Respiratory Health, Harry Perkins Institute

Nedlands, Western Australia, Australia

Pacific Lung Research Institute at St. Paul's Hospital

Vancouver, British Columbia, Canada

The Ottawa Hospital Center for Practice-Changing Research

Ottawa, Ontario, Canada

Centre d'études cliniques CIUSS SLJ, Hôpital Chicoutimi

Chicoutimi, Quebec, Canada

Centre Hospitalier de l'Université de Montréal

Montreal, Quebec, Canada

McGill University Health Center

Montreal, Quebec, Canada

Centre de recherche de l'institut Universitaire de Cardiologie et de Pneumologie de Québec

Québec, Quebec, Canada

Countries

United States; Australia; Canada

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

LAU-14-01

Identifier Type: -

Identifier Source: org_study_id