Actively Recruiting

Phase 2
Age: 18Years - 60Years
All Genders
Healthy Volunteers
NCT06237335

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

Led by ReCode Therapeutics · Updated on 2026-01-16

192

Participants Needed

23

Research Sites

152 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years - 60Years
All Genders
Healthy Volunteers

Eligibility Criteria

Eligible

You may qualify if you...

  • Healthy adults aged 18 to 55 years with body weight ≥50 kg and BMI between 16-32 kg/m2
  • Forced expiratory volume in one second (FEV1) of at least 80% predicted for healthy participants
  • Good general health based on medical history, lab tests, vital signs, ECG, and physical exam
  • Confirmed diagnosis of cystic fibrosis for CF participants
  • FEV1 between 50% and 100% of predicted for CF participants
  • Either not eligible for CFTR modulators due to mutations or eligible but intolerant or contraindicated
  • Willing and able to understand and comply with study procedures
Not Eligible

You will not qualify if you...

  • Significant medical, surgical, or psychiatric conditions
  • Supine blood pressure >150/90 mm Hg after rest for healthy participants
  • Abnormal clinical lab tests at screening
  • Current smokers or recent nicotine users (within 6 weeks) or heavy former smokers (>10 pack years)
  • Hepatic cirrhosis with portal hypertension or moderate to severe hepatic impairment
  • Acute respiratory infection, pulmonary exacerbation, or recent therapy changes within 4 weeks before dosing
  • Lung infections linked to rapid decline in lung function
  • Arterial oxygen saturation on room air less than 94% at screening
  • Treatment with CFTR modulators within 12 weeks of screening

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 23 locations

1

The University of Alabama at Birmingham

Birmingham, Alabama, United States, 35294

Actively Recruiting

2

University of Arizona

Tucson, Arizona, United States, 85719

Actively Recruiting

3

Stanford University

Palo Alto, California, United States, 94304

Actively Recruiting

4

UCSD

San Diego, California, United States, 92037

Actively Recruiting

5

National Jewish Health

Denver, Colorado, United States, 80206

Actively Recruiting

6

Emory University

Atlanta, Georgia, United States, 30322

Actively Recruiting

7

Boston Children's Hospital

Boston, Massachusetts, United States, 02215

Actively Recruiting

8

New York Medical College

Valhalla, New York, United States, 10595

Actively Recruiting

9

The University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States, 27599

Actively Recruiting

10

Oregon Health & Science University

Portland, Oregon, United States, 97239

Actively Recruiting

11

University of Pittsburgh

Pittsburgh, Pennsylvania, United States, 15213

Actively Recruiting

12

UT Southwestern Medical Center

Dallas, Texas, United States, 75390

Actively Recruiting

13

University of Washington

Seattle, Washington, United States, 98195

Actively Recruiting

14

Centre Hospitalier Régional Universitaire de Montpellier - Hôpital Arnaud de Villeneuve

Montpellier, France

Actively Recruiting

15

Hôpital Necker Enfants Malades

Paris, France

Actively Recruiting

16

UMC Utrecht

Utrecht, Netherlands

Actively Recruiting

17

New Zealand Clinical Research (Part 1 Only)

Auckland, New Zealand

Completed

18

University Hospitals Birmingham

Birmingham, United Kingdom

Actively Recruiting

19

Royal Papworth Hospital

Cambridge, United Kingdom

Actively Recruiting

20

Leeds Teaching Hospitals

Leeds, United Kingdom

Actively Recruiting

21

King's College Hospital

London, United Kingdom

Actively Recruiting

22

Nottingham University Hospitals

Nottingham, United Kingdom

Actively Recruiting

23

University Hospital Southampton

Southampton, United Kingdom

Actively Recruiting

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Research Team

P

Priya Ryali, MBA

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

DOUBLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

5

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