{"id":8883,"date":"2024-07-15T01:01:02","date_gmt":"2024-07-15T08:01:02","guid":{"rendered":"https:\/\/cf-clinical-studies.com\/cf-phase-1b-rct2100\/"},"modified":"2025-12-22T09:58:11","modified_gmt":"2025-12-22T17:58:11","slug":"cf-phase-1b-rct2100","status":"publish","type":"page","link":"https:\/\/cf-clinical-studies.com\/en-gb\/","title":{"rendered":"CF Phase 1b RCT2100"},"content":{"rendered":"\t\t
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A Clinical Trial for People with Cystic Fibrosis (CF)<\/h1>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Who Have Specific Mutations in the CFTR Gene, Which Are the Cause of CF<\/h5>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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This clinical study is exploring a potential new inhaled messenger RNA
(mRNA) treatment called RCT2100 that may improve the function of
the CFTR protein in the lungs and can lead to protein production,
getting at the root cause of CF lung disease rather than just controlling
symptoms.<\/p>

Here, you will find more information about the trial, investigative inhaled therapy, and how you, or someone you know, can participate.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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About Cystic Fibrosis<\/h3>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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CF is a progressive genetic condition that causes persistent lung infections and respiratory failure. It is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and impacts approximately 105,000 people worldwide. <\/p>

The absence or dysfunction of the CFTR protein results in a defect in airway hydration, which leads to excessive mucus buildup in the lungs. It also creates a mucociliary clearance defect, recurrent infections, inflammation, respiratory failure, and other complications. <\/p>

Despite the availability of CFTR modulators that work for 90% of people with CF, many will not be able to take the medication due to side effects, lack of response, or other reasons. About 10% of the CF community have genetic mutations that do not benefit from these therapeutics. <\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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CF impacts approximately 105,000 people worldwide. 90% have responsive genotypes to CFTR modulators, and 10% (13,000 people) do not. <\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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Why this Trial is Important<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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No CFTR modulator therapies are currently approved to treat individuals with certain mutations in the CFTR gene because these mutations prevent the production of any CFTR protein.<\/h4>\n

Without CFTR protein, there is nothing available for the therapy to correct. This study aims to find
out whether the investigational study medicine is safe and may work as a potential new treatment
option for people with CF who are not eligible for, or are eligible but not currently taking, CFTR
modulator therapy. The study is also testing to see if taking the investigational study medicine,\nRCT2100, with the already approved CFTR modulator therapy, ivacaftor, is safe and may provide\ngreater benefit to people with CF who are not eligible for CFTR modulator therapy or who are eligible\nto receive ivacaftor in combination with other CFTR protein correctors. <\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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To learn more, contact:<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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\n\t\t\t\t\t\n\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\tclinicaltrials@recodetx.com<\/span>\n\t\t\t\t\t<\/span>\n\t\t\t\t\t<\/a>\n\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t
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How the Clinical Trial Medicine Works<\/h3>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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ReCode is developing RCT2100, an investigative inhaled therapy for cystic fibrosis (CF) based on messenger RNA (mRNA). This treatment is designed to deliver CFTR mRNA directly to target cells in the lungs, instructing them to produce a functional version of the CFTR protein. <\/p>

Unlike current CFTR modulators that work on existing defective proteins by either correcting or potentiating that protein, RCT2100 is designed to allow the cells to make <\/span>new functional CFTR protein.<\/span><\/p>

By providing CFTR mRNA to individuals with CF who are missing this protein, the therapy aims to address the underlying cause of the condition, potentially improving CFTR protein function in the lungs rather than just managing symptoms.<\/p>

RCT2100 administered with ivacaftor, a CFTR modulator that potentiates existing CFTR protein, may provide more improvement in CFTR protein function in the lungs than RCT2100 alone.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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How the Clinical Trial Medicine is Given<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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The investigative medicine, RCT2100, will be given using a nebuliser \u2014 a medical device that converts liquid medication into a mist for inhalation.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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1<\/span> Screening<\/h4>

There will be a 2\u20134-hour clinic visit to determine your eligibility to participate and an opportunity for you to ask any questions to see if this trial is right for you.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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2<\/span> Treatment<\/h4>\n

If you are eligible, you will begin treatment with ivacaftor alone (taken orally) for 2 weeks followed by inhaled doses of RCT2100 taken in combination with ivacaftor (taken orally) for 4 weeks.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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3<\/span> Follow-up<\/h4>\n

After your last dose, there will be a 24-week follow-up period, which includes a final outpatient visit.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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Tests and procedures<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Throughout the trial, you will have certain exams and tests, including:<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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Physical Exam<\/strong><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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Vital Signs Measurement<\/strong><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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A non-invasive test to record the heart\u2019s electrical activity\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t
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Chest X-Ray<\/strong>
(if you have not had one within six months)<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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\n\t\t\t\t\t\t\t\t\tPulse oximetry\n<\/strong>
A non-invasive test that measures oxygen in the blood\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t
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Spirometry<\/strong>
A non-invasive test to measure how much and how fast you can move air into and out of your lungs and how well your lungs are exchanging gases<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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Blood and Urine Tests<\/strong><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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Full details will be provided for your consideration prior to completing and signing an informed consent which confirms you fully understand the process and implications of taking part in the trial.<\/div>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t
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Eligibility<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t
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