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Focus: Cystic Fibrosis Research

Making CF stand for Cure Found. Every victory, large or small, that makes life better–and longer–for children, teens and adults with cystic fibrosis, makes us that much more dedicated to research ... and even more determined to find a cure.

Cystic Fibrosis (CF) research at Cook Children's is part of the Cook Children's Pulmonary department, which includes the Fort Worth Cystic Fibrosis Center, where both pediatric and adult CF patients are treated in an outpatient setting. The ability to provide CF care for adolescents and adults in one location allows for an easier transition from pediatric to adult care for our CF patients.

Cook Children's Pulmonary department has a dedicated research nurse that oversees the entire research process from registering trial participants to obtaining clinical trial approvals.

Recognized for our commitment to excellence, in January 2009, Cook Children's was honored as a Therapeutics Development Network (TDN) coordinating center by the CF Foundation to participate in clinical trial studies for the CF patient. The TDN is a nationwide network of 82 CF clinical research centers. These centers specialize in conducting clinical trials to evaluate the safety and effectiveness of new CF therapies. The TDN centers work together to promote quality, safety, and efficiency in CF clinical trials by centralizing and standardizing the research process.

Choosing a research facility

As an active research facility, we are at the forefront of treatments from prenatal through adulthood. This lets us work closely with each patient and the families to look at the options and advanced care–from genetics to targeted therapies–that otherwise might not be available to them.

Working together

Not only do we build relationships with a global network of leading researchers, we also partner with our patients and parents starting on day one of the diagnosis; and it is only in the context of that relationship that we introduce the subject of research. Parents do not want to expose their children to risks, but most of them come to understand the concept of clinical trials. We explain how our clinical research has advanced the field of medicine and how it contributes to our cure rates here, as well as advances in cystic fibrosis.

Our approach

The ultimate goal of Children's CF Center is to cure all children, teens and adults with cystic fibrosis, to determine the causes and find ways to prevent it.

Although Cook Children's is not a traditional academic center affiliated with a university, we are academic in how we approach research and practice day in and day out. As a key collaborator with the nation's leading pediatric research organizations, our team offers the best available treatments and contributes to the search for a cure.

Cook Children's is a member institution or has received accreditation from several nationally-known cancer and blood disorder organizations for conducting research trials.

Why participate?

When you take part in a research project, you're not only helping yourself, you are potentially changing the world, at least for people with CF and their families. In fact, much of the advanced care you receive is because someone just like you took an active role in research. Still not sure? Consider this: just 20 years ago, a diagnosis of CF meant you'd be lucky to live into your teens. Today, the outlook is much better, with the average age being 41, and we have people in our CF Center here at Cook Children's who are in their 20s, 30s, even 70s. These are people who live active lives with careers, families and lots to look forward to. And who knows, the trial you participate in might just be the one that changes CF to "Cure Found".

Active research and clinical trials

Participation in the Cystic Fibrosis Patient Registry

Phase 3, Open-Label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With VX-661 in Combination with Ivacaftor in Subjects Aged 12 years and older with Cystic Fibrosis
Interventions: Drug: VX-661; Drug: Ivacaftor

Phase 2, Randomized, Open-label, Assessor-Blind, Non-Inferiority, Active-Comparator Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency
Interventions: Drug: Liprotamase; Drug: Pancreaze

Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of N91115 to Evaluate Efficacy and Safety in Patients with Cystic Fibrosis who are Homozygous for the F508del-CFTR Mutation and Being Treated with lumacaftor/ivacaftor
Interventions: Drug: N91115; Drug: Placebo

Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 Administered Orally Once-Daily for 48 Weeks in Adult Patients with Cystic Fibrosis
Inverventions: Drug: CTX-4430; Drug: Placebo

A Long-term Prospective Observational Safety Study of the Incidence of and Risk Factors for Fibrosing Colonopathy in US Patients with Cystic Fibrosis Treated with Pancreatic Enzyme Replacement Therapy: A Harmonized Protocol across Sponsors

We're here to help.

For more information on the current pulmonary research studies, please contact us at or 682-885-2103.