Summary

Eligibility
for people ages 18-65 (full criteria)
Healthy Volunteers
healthy people welcome
Location
at San Diego, California and other locations
Dates
study started
completion around

Description

Summary

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

Official Title

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

Details

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).

Keywords

Cystic Fibrosis, CF, Fibrosis, RCT2100

Eligibility

For people ages 18-65

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% 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, 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.

Locations

  • UCSD not yet accepting patients
    San Diego California 92037 United States
  • University of Arizona not yet accepting patients
    Tucson Arizona 85719 United States
  • UMC Utrecht accepting new patients
    Utrecht Netherlands
  • New Zealand Clinical Research (Part 1 Only) accepting new patients
    Auckland New Zealand

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
ReCode Therapeutics
ID
NCT06237335
Phase
Phase 1 research study
Study Type
Interventional
Participants
Expecting 100 study participants
Last Updated