A Phase 1 Study Evaluating Safety and Tolerability of RCT2100 in Healthy Participants and in Participants With CF

Launched by RECODE THERAPEUTICS · Jan 23, 2024

Keywords

ClinConnect Summary

This clinical trial is testing a new drug called RCT2100 to see if it is safe and well-tolerated in both healthy adults and those with cystic fibrosis (CF). This is the first time RCT2100 is being given to people, and the results will help researchers understand how it affects the body and prepare for future studies. The trial is currently looking for participants, including healthy adults aged 18 to 55 and individuals with CF who meet specific health criteria.

To be eligible, healthy participants need to be in good overall health, have a certain level of lung function, and meet other basic health requirements. For those with CF, they must have a confirmed diagnosis and specific lung function levels while not currently using certain CF medications. Participants can expect to undergo health screenings to ensure they qualify for the study and will receive close monitoring during the trial. This is an important step in developing new treatments for cystic fibrosis, and taking part could contribute to future advancements in care.

Gender

ALL

Eligibility criteria

• Part 1 Major Inclusion Criteria:
• • 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.
• Part 1 Major Exclusion Criteria:
• • 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 Inclusion Criteria:
• • Confirmed diagnosis of CF
• • Forced expiratory volume in 1 second ≥40% 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
• • Values of AST, ALT, alkaline phosphatase, or gamma-glutamyl transferase (GGT) ≥3×ULN
• • Treatment with a CFTR modulator (Kalydeco, Trikafta, Symdeko, or Orkambi) within 12 weeks of Screening
• • Other protocol defined Inclusion/Exclusion criteria may apply.