RCT2100: RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer
Placebo: Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer
RCT2100: RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer for 4 weeks
RCT2100: RCT2100 supplied at a single dose strength administered via oral inhalation using nebulizer for 12 weeks
Ivacaftor: ivacaftor administered orally for 6 weeks
RCT2100: RCT2100 supplied at varying dose strengths. Co- administered via oral inhalation using nebulizer for 4 weeks with ivacaftor after initial 2 weeks of ivacaftor dosing run in period
Study summary
This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.
Eligibility
Sex
ALL
Min age
18 Years
Max age
60 Years
Healthy volunteers
Accepted
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 ≥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
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
* Treatment with a CFTR modulator (Kalydeco, Trikafta, Symdeko, Orkambi, or Alyftrek) within 12 weeks of Screening
Other protocol defined Inclusion/Exclusion criteria may apply.
Part 3 Major Inclusion Criteria:
* 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 dual or triple CFTR modulators (based on local prescribing information) but not using CFTR modulators due to intolerance or contraindications
Part 3 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
* Treatment with a CFTR modulator (Kalydeco, Trikafta, Symdeko, Orkambi, or Alyftrek) within 12 weeks of Screening
Other protocol defined Inclusion/Exclusion criteria may apply.
Primary outcome measure(s)
Part 1: The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs). — From Baseline Through Day 29 Safety and tolerability as assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Part 2: The number of participants with CF with AEs and SAEs. — From Day 1 through Safety Follow-up, Week 24 Safety and tolerability of multiple-ascending doses of inhaled RCT2100 administered to participants with CF
Part 3: The number of participants with CF with AEs and SAEs. — From Day 1 through Safety Follow-up, Week 24 To assess the safety and tolerability of RCT2100 co-administered with ivacaftor in participants with CF.
Trial sites (23)
Facility
City
Region
Status
The University of Alabama at Birmingham
Birmingham
Alabama
University of Arizona
Tucson
Arizona
Stanford University
Palo Alto
California
UCSD
San Diego
California
National Jewish Health
Denver
Colorado
Emory University
Atlanta
Georgia
Boston Children's Hospital
Boston
Massachusetts
New York Medical College
Valhalla
New York
The University of North Carolina at Chapel Hill
Chapel Hill
North Carolina
Oregon Health & Science University
Portland
Oregon
University of Pittsburgh
Pittsburgh
Pennsylvania
UT Southwestern Medical Center
Dallas
Texas
University of Washington
Seattle
Washington
Centre Hospitalier Régional Universitaire de Montpellier - Hôpital Arnaud de Villeneuve
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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