A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF

NCT06237335 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: RCT2100-1012024-512169-15
• Study Type: interventional
• Target: 192
• Locations: 5 countries
• Sites: 23

Brief Summary

This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; CF; mRNA

Outcome Measures

Primary Outcomes (3)

• Part 1: The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).

  Safety and tolerability as assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

  From Baseline Through Day 29

• Part 2: The number of participants with CF with AEs and SAEs.

  Safety and tolerability of multiple-ascending doses of inhaled RCT2100 administered to participants with CF

  From Day 1 through Safety Follow-up, Week 24

• Part 3: The number of participants with CF with AEs and SAEs.

  To assess the safety and tolerability of RCT2100 co-administered with ivacaftor in participants with CF.

  From Day 1 through Safety Follow-up, Week 24

Study Arms (5)

RCT2100 (Part 1)

EXPERIMENTAL

RCT2100 single dose

Drug: RCT2100

Placebo (Part 1)

PLACEBO COMPARATOR

Placebo single dose

Other: Placebo

RCT2100 (Part 2) 4 week

EXPERIMENTAL

RCT2100 multiple dose

Drug: RCT2100

RCT2100 (Part 2) 12 week

EXPERIMENTAL

RCT2100 multiple dose

Drug: RCT2100

Experimental: RCT2100 (Part 3) 6 week

EXPERIMENTAL

RCT2100 multiple dose

Drug: Ivacaftor; Drug: RCT2100

Interventions

RCT2100 DRUG

RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer

RCT2100 (Part 1)

Placebo OTHER

Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer

Placebo (Part 1)

Ivacaftor DRUG

ivacaftor administered orally for 6 weeks

Experimental: RCT2100 (Part 3) 6 week

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify 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 greater than 10 pack years of smoking history are excluded.
• 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
• 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
• Confirmed diagnosis of CF
• Forced expiratory volume in 1 second ≥50% and ≤100% of predicted mean value for age, sex, and height

Sponsors & Collaborators

• ReCode Therapeutics: lead

Study Sites (23)

The University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

RECRUITING

University of Arizona

Tucson, Arizona, 85719, United States

RECRUITING

Stanford University

Palo Alto, California, 94304, United States

RECRUITING

UCSD

San Diego, California, 92037, United States

RECRUITING

National Jewish Health

Denver, Colorado, 80206, United States

RECRUITING

Emory University

Atlanta, Georgia, 30322, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02215, United States

RECRUITING

New York Medical College

Valhalla, New York, 10595, United States

RECRUITING

The University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

RECRUITING

Oregon Health & Science University

Portland, Oregon, 97239, United States

RECRUITING

University of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

University of Washington

Seattle, Washington, 98195, United States

RECRUITING

Centre Hospitalier Régional Universitaire de Montpellier - Hôpital Arnaud de Villeneuve

Montpellier, France

RECRUITING

Hôpital Necker Enfants Malades

Paris, France

RECRUITING

UMC Utrecht

Utrecht, Netherlands

RECRUITING

New Zealand Clinical Research (Part 1 Only)

Auckland, New Zealand

COMPLETED

University Hospitals Birmingham

Birmingham, United Kingdom

RECRUITING

Royal Papworth Hospital

Cambridge, United Kingdom

RECRUITING

Leeds Teaching Hospitals

Leeds, United Kingdom

RECRUITING

King's College Hospital

London, United Kingdom

RECRUITING

Nottingham University Hospitals

Nottingham, United Kingdom

RECRUITING

University Hospital Southampton

Southampton, United Kingdom

RECRUITING

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

ivacaftor

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• John Matthews, MBBS, MCRP, PhD

  ReCode Therapeutics, Inc.

  STUDY CHAIR

Central Study Contacts

Priya Ryali, MBA

CONTACT

650-629-7900; clinicaltrials@recodetx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Participant and investigator masking only applies to Part 1 which is randomized. For Part 2, there is no masking, and this part is Open Label.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2024
• First Posted: February 1, 2024
• Study Start: February 1, 2024
• Primary Completion (Estimated): August 30, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: January 16, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (13); GB (6); NL (1); FR (2); NZ (1)