Research

Research Studies

At the Toronto Adult CF Clinic we conduct many different types of research studies. Research can be observational or interventional.

Observational research means that you may be invited to fill out a questionnaire, to donate a tube of blood, or provide an extra sputum sample. It could also mean we will ask you for permission to use your clinical data to understand trends in the health of the CF population.

Interventional research generally involves the use of a new drug or device, or the use of an already approved drug or device for a new reason. Interventional trials are also called clinical trials.

What is a Clinical Trial?

Clinical trials are research studies that help to determine if a new drug is safe and effective. There are 4 phases of clinical trials that take place before a drug can be prescribed or used outside of a research study in Canada.

Clinicaltrials.gov is a website where information about all clinical trials is posted. You can use this website to find out more about clinical trials in general, or to search for different clinical trials. Results are also uploaded to clinicaltrials.gov they are available.

The Toronto Adult CF Clinic is part of CF Canada’s Clinical Trials Network (CanACT). The Clinical Trials Finder maintained by CANACT keeps track of which studies are happening at different CF centres across Canada and is another great source of information about current research initiatives in CF.

What are the different types of clinical trials?

There are 4 stages or phases that a drug or device must go through before it becomes available for use outside of a clinical trial setting.

Phase 1 studies usually recruit healthy volunteers and focus on safety. They are usually very short in duration and last from a few days to a few weeks. The goal of phase 1 trials is to find out what the drugs most frequent and serious adverse events are. It also studies how the drug is broken down by the body.

Phase 2 studies are done to determine whether the drug works in people who have a certain disease or medical condition. For example, participants receiving the drug may be compared with similar participants receiving a different treatment, usually an inactive substance (called a placebo) or a different drug. Researchers monitor for safety and short-term adverse events. Phase two trials are generally a month or two long and involve longer and more frequent visits.

Phase 3 studies gather more information about safety and effectiveness in a larger number of participants than phase 2 trials. They can also last for months to years, but will involve fewer visits to clinic. The visits may also be shorter. For many studies, they will align with your regular CF clinic visits. Phase 3 studies may look at different doses of the study drug to help figure out which one is the most effective. Phase 3 studies can start out with a placebo-controlled portion and then move to an open label portion where you and your research team know that you are getting the drug that is being studied.

Phase 4 studies usually take place after Health Canada has approved the drug. These studies continue to monitor and collect long term safety data.

What is observational research?

Observational research studies may involve completing a survey or a questionnaire, providing a tube of blood or a sputum sample, or may involve accessing your clinical information by a chart review. Observational research can be retrospective (look at things that have happened in the past) or prospective (follow up over time to find out what happens.) Observational researches studies help answer different questions. For example, can a questionnaire be used to screen for mental health or sinus disease in CF patients?

Studies at TACFC

What studies are being done at the Toronto Adult CF Center?

You can learn more about research studies happening at the Toronto Adult CF Centre by clicking on the links below. Your CF doctors and the research coordinators will also be happy to answer any questions you may have about research in general or a specific trial.

Clinical Trials Now Recruiting

None at this time.

Observational Trials Now Recruiting

Mapping and Isolation of Genes Influencing Severity of Disease in Cystic Fibrosis

Project Breathe: Analysis of remote monitoring/virtual clinic data in adult patients with Cystic Fibrosis (CF) followed at St. Michael’s Hospital, Toronto, Canada

Pseudomonas aeruginosa and Staphylococcus aureus interactions in CF patients with pulmonary exacerbations

Comprehensive Phenotyping of Exacerbations in CF

The influence of Cytomegalovirus infection on cystic fibrosis (CF) disease progressionCAN-IMPACT: Canadian observational study evaluating the long-term IMPACT of CFTR modulators on people with CF

Studies that are ongoing, but are not recruiting

COVID-19 Antibody Responses in Cystic Fibrosis Patients: (CAR-CF) Study

Study results and completed trials

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial To Evaluate Efficacy And Safety Of Lenabasum In Cystic Fibrosis

Lenabasum (JBT-101) is a novel oral drug designed to reduce inflammation and prevent permanent tissue damage in the lungs. The results from a small Phase 2 clinical trial suggested that Lenabasum treatment showed markers of reduced inflammation. Following this study, a multinational double-blind, randomized, placebo-controlled study was employed to evaluate the safety and efficacy of Lenbasum. However, this large Phase 2b trial did not find a statistically significant reduction in the rate of pulmonary exacerbations in CF patients at high risk for recurrent pulmonary exacerbations between those who took lenabausm and those who took placebo. While, the study failed to meet its primary endpoint, the results from the trial suggest that Lenabasum’s use was safe and well-tolerated.

Clinical Trial Details | Press Release

A Phase 3 Study Of VX-445 Combination Therapy In Cystic Fibrosis (CF) Subjects Heterozygous For F508del And A Gating Or Residual Function Mutation (F/G And F/RF Genotypes)

This study enrolled patients who have DF508 and a mutation that is responsive to either ivacaftor or tezacaftor/ivacaftor to see if there is additional benefit with the elezacaftor/tezacaftor/ivacaftor combination ontop of standard of care. The results of this study will be posted here when they are published.

Clinical Trial Details

Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least one F508del allele: 144-week interim results from a 192-week open-label extension study

The aim of this study was to assess long-term safety and efficacy of ELX/TEZ/IVA (Trikafta) in people with CF who have at least one F508del allele. Participants had increases lung function, respiratory symptoms and body mass index, and had decreases in sweat chloride concentration and pulmonary exacerbation rates that were maintained over the interim analysis period. A favourable safety profile was reported.

Study Results

>> See All Study Results & Completed Trials <<

Papers using registry data

Sleep breathing disorder frequency, risk factors, and treatment among adults with cystic fibrosis

Global disparities in cystic fibrosis outcomes prior to CFTR modulators: A CF registries cohort study in South Africa and Canada

Cystic fibrosis survival outcomes following second lung transplant: The north American experience

Lung function decline is mitigated following liver transplantation in people with cystic fibrosis: A retrospective cohort study

Contemporary cystic fibrosis incidence rates in Canada and the United States

Canadian CF Registry

The Canadian Cystic Fibrosis Registry, or CCFR, was created in the 1970s as a way to monitor important clinical trends in the Canadian CF population. The Canadian CF Registry continues to be an extremely valuable resource of national CF data that is used to improve the quality of patient care, influence research, and bolster advocacy efforts across the country. These data are also leveraged to critically assess health outcomes of new therapies and identify potentially eligible patients for new and emerging treatments.

When you consent to have your clinical data included in the Canadian Cystic Fibrosis Registry, you enable researchers and clinicians to conduct research which allow us to examine patterns of disease within the CF population, respond to emerging health care issues, track clinical outcomes over time, and ultimately improve care and survival of those with CF in Canada. The registry collects important clinical data on virtually all Canadians with CF who are followed at one of the 41 accredited CF centres across the country. Some of the information that is collected includes lung function measurements, nutrition markers (e.g. height and weight), microbiology, and CF-related complications.

You are also able to access your own data within the registry by signing up to MyCFLifePortal. MyCFLifePortal is a secure website and is an extension of the Canadian CF Registry system and is based on the current design of the website used by clinics. A major fundamental difference between the two websites is that MyCFLifePortal will show only patient-level data and is read-only meaning that patients can view but cannot edit/change the information. If you are interested in gaining access to your registry data through the portal, speak to your CF team in clinic and they can get you signed up.

Canadian CF Registry Annual Report

Each year, Cystic Fibrosis Canada publishes an annual report summarizing the characteristics of the Canadian CF population. It is using these data that we can estimate the number of people living with CF in Canada as well as show how dramatically the survival in CF has increased over the decades. In fact, Canada has one of the highest reported median age of survival for CF patients in the world! For more information, read the most recent Canadian CF Registry annual report.