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Tests treatment safety and results for Cystic Fibrosis
Official title A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
ClinicalTrials.gov ID: NCT06237335
What this study is testing
What is RCT2100?
RCT2100 is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for cystic fibrosis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 60. Healthy volunteers may be eligible.
You may be able to join if
- Part 1 Major
- Healthy, adult, male or female, 18-55 years of age, inclusive, at screening.
- Body weight greater than or equal to 50 kg and body mass index (BMI) between 16-32 kg/m2, inclusive
- The participant has a forced expiratory volume in one second (FEV1) of at least 80% predicted
- The participant is considered by the investigator to be in good general health as determined by medical history, clinical laboratory test results...
You likely can't join if
- History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or disease.
- The participant has supine blood pressure (BP) \>150 mm Hg (systolic) or \>90 mm Hg (diastolic), following at least 5 minutes of supine rest.
- The participant has abnormal clinical laboratory tests at screening, as assessed by the study-specific laboratory.
- The participant is a smoker or has used nicotine or nicotine-containing products 6 weeks before the first dose of study drug. Former smokers with...
- Confirmed diagnosis of CF
- Forced expiratory volume in 1 second ≥50% and ≤100% of predicted mean value for age, sex, and height
See the full eligibility criteria
- Part 1 Major
- Healthy, adult, male or female, 18-55 years of age, inclusive, at screening.
- Body weight greater than or equal to 50 kg and body mass index (BMI) between 16-32 kg/m2, inclusive
- The participant has a forced expiratory volume in one second (FEV1) of at least 80% predicted
- The participant is considered by the investigator to be in good general health as determined by medical history, clinical laboratory test results, vital sign measurements, 12-lead ECG results, and physical examination...
- Understands the study procedures in the informed consent form (ICF), and is willing and able to comply with the protocol. Part 1 Major
- History or presence of clinically significant medical, surgical, clinical laboratory, or psychiatric condition or disease.
- The participant has supine blood pressure (BP) \>150 mm Hg (systolic) or \>90 mm Hg (diastolic), following at least 5 minutes of supine rest.
- The participant has abnormal clinical laboratory tests at screening, as assessed by the study-specific laboratory.
- The participant is a smoker or has used nicotine or nicotine-containing products 6 weeks before the first dose of study drug. Former smokers with greater than 10 pack years of smoking history are excluded. Part 2 Major...
- Confirmed diagnosis of CF
- Forced expiratory volume in 1 second ≥50% and ≤100% of predicted mean value for age, sex, and height
- a) Not eligible for CFTR modulators based on having mutations of CFTR gene on both alleles that are not responsive to CFTR modulator therapy OR
- b) Eligible for CFTR modulators (based on local prescribing information) but not using CFTR modulators due to intolerance or contraindications Part 2 Major Exclusion Criteria:
- Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for sinopulmonary disease within 4 weeks before the first dose of study drug
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Arterial oxygen saturation on room air less than 94% at screening
- Treatment with a CFTR modulator (Kalydeco, Trikafta, Symdeko, Orkambi, or Alyftrek) within 12 weeks of Screening Other protocol defined Inclusion/Exclusion criteria may apply. Part 3 Major Inclusion Criteria:
- Confirmed diagnosis of CF
- Forced expiratory volume in 1 second ≥50% and ≤100% of predicted mean value for age, sex, and height
- a) Not eligible for CFTR modulators based on having mutations of CFTR gene on both alleles that are not responsive to CFTR modulator therapy OR
- b) Eligible for dual or triple CFTR modulators (based on local prescribing information) but not using CFTR modulators due to intolerance or contraindications Part 3 Major Exclusion Criteria:
- Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for sinopulmonary disease within 4 weeks before the first dose of study drug
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Arterial oxygen saturation on room air less than 94% at screening
- Treatment with a CFTR modulator (Kalydeco, Trikafta, Symdeko, Orkambi, or Alyftrek) within 12 weeks of Screening Other protocol defined Inclusion/Exclusion criteria may apply.
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Tucson, Arizona, United States
- Palo Alto, California, United States
- San Diego, California, United States
- Denver, Colorado, United States
- Atlanta, Georgia, United States
- Boston, Massachusetts, United States
- Valhalla, New York, United States
- Chapel Hill, North Carolina, United States
- Portland, Oregon, United States
- Pittsburgh, Pennsylvania, United States
- Dallas, Texas, United States
- Seattle, Washington, United States
- Montpellier, France
- Paris, France
- Utrecht, Netherlands
- Auckland, New Zealand
- Birmingham, United Kingdom
- Cambridge, United Kingdom
- Leeds, United Kingdom
+ 3 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 60 years. Healthy volunteers may be eligible. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Tucson, Arizona, United States; Palo Alto, California, United States; San Diego, California, United States; Denver, Colorado, United States; Atlanta, Georgia, United States and 17 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.