Clinical trial NCT06237335: A Phase 1 Study Evaluating Safety and Tolerability of RCT2100 in Healthy Participants and in Participants With CF
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis
CF
Lead sponsor: ReCode Therapeutics
Sponsor class: INDUSTRY
First posted: 2024-02-01
Enrollment: 158
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

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).

Eligibility criteria: 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% 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.

Primary outcomes:
measure: Part 1: The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).
timeFrame: From Baseline Through Day 29
measure: Part 2: The number of participants with CF with AEs and SAEs.
timeFrame: From Day 1 through Safety Follow-up, Week 24
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: The University of Alabama at Birmingham
state: Alabama
city: Tucson
country: United States
facility: University of Arizona
state: Arizona
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: San Diego
country: United States
facility: UCSD
state: California
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Valhalla
country: United States
facility: New York Medical College
state: New York
city: Chapel Hill
country: United States
facility: The University of North Carolina at Chapel Hill
state: North Carolina
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Seattle
country: United States
facility: University of Washington
state: Washington
city: Montpellier
country: France
facility: Centre Hospitalier Régional Universitaire de Montpellier - Hôpital Arnaud de Villeneuve
city: Paris
country: France
facility: Hôpital Necker Enfants Malades
city: Utrecht
country: Netherlands
facility: UMC Utrecht
city: Auckland
country: New Zealand
facility: New Zealand Clinical Research (Part 1 Only)
city: Birmingham
country: United Kingdom
facility: University Hospitals Birmingham
city: Cambridge
country: United Kingdom
facility: Royal Papworth Hospital
city: Leeds
country: United Kingdom
facility: Leeds Teaching Hospitals
city: London
country: United Kingdom
facility: King's College Hospital
city: Nottingham
country: United Kingdom
facility: Nottingham University Hospitals
city: Southampton
country: United Kingdom
facility: University Hospital Southampton
Study officials:
affiliation: ReCode Therapeutics, Inc.
name: John Matthews, MBBS, MCRP, PhD
role: STUDY_CHAIR
References: