Cystic Fibrosis Clinical Trials 2023

What Are Cystic Fibrosis BPH Clinical Trials?

According to Mayo Clinic, cystic fibrosis is an inherited genetic condition. This condition causes mucus to build up in a cystic fibrosis patient's lungs and digestive system. Symptoms of this condition begin in childhood and get progressively worse over time. Cystic fibrosis causes various complications, including lung infections and digestive issues.

Clinical Trials for cystic fibrosis aim to collect research data on those with this condition and improve the level of care and treatment options for those with cystic fibrosis. One of the current trials for cystic fibrosis is the repeated application of gene therapy to combat this disease.

Why Is Cystic Fibrosis Being Studied Through Clinical Trials?

Cystic fibrosis is studied through clinical trials to develop new therapies to treat this condition. Trials for cystic fibrosis also focus on developing new treatments to treat health complications caused by this condition. Clinical trials also work towards improving the lives of patients diagnosed with cystic fibrosis.

Cystic Fibrosis is only inherited if both parents carry the gene that causes this condition. The Cystic Fibrosis Foundation states that there are 40,000 people living with cystic fibrosis in the U.S, and 1 in 30 Americans is a carrier of this condition. Worldwide cystic fibrosis affects 70,000 people.

Those with cystic fibrosis are prone to developing severe infections that could lead to respiratory failure. In addition, intestinal secretions in the digestive tract of those with cystic fibrosis can become abnormal and cause blockages that may require surgery.

Cystic fibrosis is a condition that needs to be studied to find better treatments and a possible cure. Because this is a genetic condition, it cannot be prevented but can be improved through treatment.

What Are The Types Of Treatments Available For Cystic Fibrosis?

There are a few treatments currently being used to treat cystic fibrosis. These include:

1. Medication. Medications are the most common treatment for cystic fibrosis. Medications for treating this condition include antibiotics, anti-inflammatories, mucus thinners, CTFR modulators, and Bronchodilators.
2. Surgery. Those who have an advanced case of cystic fibrosis may need a liver or lung transplant.
3. Techniques and devices for clearing blockages. Methods and devices are used to clear mucus and blockages from the lungs in patients with cystic fibrosis. This treatment is used in conjunction with medications.
4. Vitamins. Patients with cystic fibrosis are more prone to becoming malnourished due to the digestive complications of this condition. Therefore, vitamins are usually prescribed to help prevent malnourishment.

A few current clinical trials for cystic fibrosis include:

1. Trikafta. This clinical trial focuses on the effectiveness of the treatment of Trikafta, a drug that helps patients 12 years and older by improving their CFTR function and bringing it as close as possible to normal functioning. CTFR is the cystic transmembrane conductance regulator protein responsible for balancing the salt and water on the surface of the lungs.
2. IV gallium. This clinical trial aims to study IV gallium as a treatment for those diagnosed with cystic fibrosis who have nontuberculous mycobacteria infections in their lungs.

What Are Some Recent Breakthrough Clinical Trials For Cystic Fibrosis?

Multiple clinical trials have been conducted over the past several years on cystic fibrosis. Some of the most notable breakthrough clinical trials are:

2020: Trikafta for treating cystic fibrosis. Trikafta is an oral medication that works at targeting a gene defect that causes 90% of cystic fibrosis cases. According to recent clinical trials, this treatment reduced lung flare-ups, decreased salt loss and improved the quality of life, and improved respiratory symptoms in cystic fibrosis patients.

2023: Kaftrio for treating cystic fibrosis. Kaftrio is a treatment that works towards getting to the underlying cause of cystic fibrosis and also helps patients breathe better. This treatment is solely focused on helping children diagnosed with cystic fibrosis.

Who Are Some Of The Key Opinion Leaders / Researchers Conducting Cystic Fibrosis Clinical Trial Research?

Dr. Juan Lanowski is an affiliate researcher at the Respiratory Research Center. Lanowski and his associate Julian Tam are researching cystic fibrosis and working on the development of new treatments like gene therapy. Their research also is aimed at helping 10% of cystic fibrosis patients who can't be helped with existing forms of treatment.

James Finklea is a pulmonologist focusing on cystic fibrosis, critical care medicine, and general pulmonology. He is a specialist in the field of cystic fibrosis and has written numerous publications on the topic of cystic fibrosis.