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

Phase 1

• Conditions: Cystic Fibrosis; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: CTIS2024-512169-15-00
• Lead Sponsor: Recode Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-03-29
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 78

Inclusion Criteria

Males or females aged 18 years or older on the date of informed consent, Confirmed diagnosis of CF, 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) Not using CFTR modulators due to intolerance or contraindications, Forced expiratory volume in 1 second =40% of predicted mean value for age, sex, and height (equations of the Global Lung Function Initiative [GLI]) at the Screening Visit. Spirometry measurements must meet American Thoracic Society/European Respiratory Society criteria for acceptability and repeatability

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 (Day 1), Lung infection with organisms associated with a more rapid decline in pulmonary status (including, but not limited to, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus). For participants who have had a history of a positive culture, the Investigator will apply the following criteria to establish whether the participant is free of infection from such organisms: • The participant has not had a respiratory tract culture positive for these organisms within the 12 months before the date of informed consent • The participant has had at least two respiratory tract cultures negative for such organisms within the 12 months before the date of informed consent, with the first and last of these separated by at least 3 months, and the most recent one within the 6 months before the date of informed consent, Arterial oxygen saturation on room air less than 94% at screening, Values of AST, ALT, or gamma-glutamyl transferase (GGT) =3×ULN, Treatment with a CFTR modulator (Kalydeco, Trikafta, Symdeko, or Orkambi) within 12 weeks of Screening

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified