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 1/2

Recruiting

• Conditions: Cystic Fibrosis

• Registration Number: 2024-512169-15-00
• Lead Sponsor: Recode Therapeutics Inc.

Brief Summary

To assess the safety and tolerability of multiple-ascending doses of inhaled RCT2100 administered via nebulizer to participants with CF

Detailed Description

This is a multi-part study to assess the safety, tolerability, and biodistribution of a single ascending dose of inhaled RCT2100 administered via nebulizer to healthy participants (Part 1) and multiple-ascending doses of inhaled RCT2100 administered to participants with CF (Part 2).

Study Timeline

• Study First Submitted: 2024-01-23
• Study First Submitted QC: 2024-01-23
• Study Start: 2024-02-01
• Study First Posted: 2024-02-01
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-29
• Last Update Posted: 2025-07-30
• Primary Completion: 2025-12-31
• Study Completion: 2026-03-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 180

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 ≥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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
Safety and tolerability as assessed by number of participants with AEs and SAEs		Safety and tolerability as assessed by number of participants with AEs and SAEs

Secondary Outcome Measures

Name	Time	Method
Biodistribution parameters may be derived from concentrations of RCT2100 components in blood (where feasible). Parameters may include but are not limited to the following: AUC, Cmax, Tmax, and t1/2		Biodistribution parameters may be derived from concentrations of RCT2100 components in blood (where feasible). Parameters may include but are not limited to the following: AUC, Cmax, Tmax, and t1/2
Absolute change in percent predicted FEV1 (ppFEV1) from baseline to Week 4 (escalation cohorts 1 to 3) or Week 12 (expansion cohort)		Absolute change in percent predicted FEV1 (ppFEV1) from baseline to Week 4 (escalation cohorts 1 to 3) or Week 12 (expansion cohort)
Change from baseline in CF questionnaire-revised (CFQ-R) respiratory domain score through Week 4 (escalation cohorts 1 to 3) or Week 12 (expansion cohort)		Change from baseline in CF questionnaire-revised (CFQ-R) respiratory domain score through Week 4 (escalation cohorts 1 to 3) or Week 12 (expansion cohort)
Incidence and titer of anti-CFTR binding antibodies		Incidence and titer of anti-CFTR binding antibodies
Based on safety and tolerability at projected efficacious doses		Based on safety and tolerability at projected efficacious doses

Trial Locations

Locations (23)

The University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

University of Arizona; 🇺🇸 Tucson, Arizona, United States

Stanford University; 🇺🇸 Palo Alto, California, United States

UCSD; 🇺🇸 San Diego, California, United States

National Jewish Health; 🇺🇸 Denver, Colorado, United States

Emory University; 🇺🇸 Atlanta, Georgia, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

New York Medical College; 🇺🇸 Valhalla, New York, United States

The University of North Carolina at Chapel Hill; 🇺🇸 Chapel Hill, North Carolina, United States

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States

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