The Children’s Hospital Colorado has announced that it will part in a Phase 3 clinical trial for an experimental new cystic fibrosis therapy. The full article can be found at the hospital’s website, here.
About Cystic Fibrosis
Cystic fibrosis (CF) is an inherited condition that causes mucus to build up in the lungs and digestive system. An estimated 30,000 people in the US are living with CF.
The sticky mucus that builds up can also prevent organs from functioning normally and cause a range of problems, including breathing difficulties, coughing, frequent lung infections, and issues with digestion. In many cases, the mucus stops digestive enzymes from breaking down food in the gut and this can result in malnutrition. Many people with CF also develop related conditions, such as diabetes, male infertility, and weakened bones.
CF is a genetic condition caused by alterations to the CFTR gene. This means that a protein that the gene codes for doesn’t function normally. Many different alterations to the CFTR gene can result in CF, but the most common is a change called the F508del mutation.
An Experimental New Treatment
Two experimental compounds called VX-659 and VX-445 are being developed for a triple combination modulator therapy for patients with CF. Modulator therapies, such as this one, aim to make the protein produced by the CFTR gene functional to improve the symptoms of CF.
The Children’s Hospital Colorado’s Participation
The Mike McMorris Cystic Fibrosis Research and Care Centre of the Children’s Hospital Colorado will take part in a Phase 3 clinical trial of a combination treatment of VX-659 and SymdekoTMin patients with CF. This is one of two Phase 3 clinical trials evaluating VX-659 and VX-445.
The people taking part in the trial at Children’s Colorado will be patients with CF who are aged 12 and above with a ‘minimal function’ mutation and an F508del alteration. This group doesn’t currently have a CFTR modulator available to it.
Often with CF, a medicine will only be effective for people with certain genetic alterations, rather than all patients. The experimental modulator therapy being investigated in the clinical trial is hoped to benefit a large group of patients since it is expected to work for everyone that has a single F508del alteration.
The Children’s Hospital Colorado has participated in several CF clinical trials before, including for the recently approved drug Symdeko.
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To find out more about CF, check out our partners Cystic Life and Strawfie Challenge.
