A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
A Phase 1/2, Multicenter Study Evaluating the Safety, Tolerability, and Biodistribution of RCT2100 With Single-Ascending Doses in Healthy Participants and Multiple-Ascending Doses and Proof-of-Concept in Participants With Cystic Fibrosis
ReCode Therapeutics
192 participants
Feb 1, 2024
INTERVENTIONAL
Conditions
Summary
This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.
Eligibility
Inclusion Criteria13
- 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 findings at screening.
- Understands the study procedures in the informed consent form (ICF), and is willing and able to comply with the protocol.
- 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
- 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
Exclusion Criteria14
- 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.
- 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
- 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
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Interventions
RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer
Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer
RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer for 4 weeks
RCT2100 supplied at a single dose strength administered via oral inhalation using nebulizer for 12 weeks
ivacaftor administered orally for 6 weeks
RCT2100 supplied at varying dose strengths. Co- administered via oral inhalation using nebulizer for 4 weeks with ivacaftor after initial 2 weeks of ivacaftor dosing run in period
Locations(23)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06237335