A Phase 1 Study Evaluating Safety and Tolerability of RCT2100 in Healthy Participants and in Participants With CF

Purpose

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

Condition

  • Cystic Fibrosis

Eligibility

Eligible Ages
Between 18 Years and 65 Years
Eligible Genders
All
Accepts Healthy Volunteers
Yes

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% 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 - Values of AST, ALT, alkaline phosphatase, 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.

Study Design

Phase
Phase 1
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)
Masking Description
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.

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
RCT2100 (Part 1) 		RCT2100 single dose
  • Drug: RCT2100
    RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer
Placebo Comparator
Placebo (Part 1) 		Placebo single dose
  • Other: Placebo
    Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer
Experimental
RCT2100 (Part 2) 4 week 		RCT2100 multiple dose
  • Drug: RCT2100
    RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer for 4 weeks
Experimental
RCT2100 (Part 2) 12 week 		RCT2100 multiple dose
  • Drug: RCT2100
    RCT2100 supplied at a single dose strength administered via oral inhalation using nebulizer for 12 weeks

Recruiting Locations

The University of Alabama at Birmingham
Birmingham, Alabama 35294

More Details

Status
Recruiting
Sponsor
ReCode Therapeutics

Study Contact

Priya Riyali, MBA
650-629-7900
clinicaltrials@recodetx.com

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).