Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06237335
Registration number
NCT06237335
Ethics application status
Date submitted
23/01/2024
Date registered
1/02/2024
Date last updated
10/07/2025
Titles & IDs
Public title
A Phase 1 Study Evaluating Safety and Tolerability of RCT2100 in Healthy Participants and in Participants With CF
Query!
Scientific 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
Query!
Secondary ID [1]
0
0
2024-512169-15
Query!
Secondary ID [2]
0
0
RCT2100-101
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Cystic fibrosis
Query!
Respiratory
0
0
0
0
Query!
Other respiratory disorders / diseases
Query!
Oral and Gastrointestinal
0
0
0
0
Query!
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Connective tissue diseases
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Other inflammatory or immune system disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - RCT2100
Other interventions - Placebo
Treatment: Drugs - RCT2100
Treatment: Drugs - RCT2100
Experimental: RCT2100 (Part 1) - RCT2100 single dose
Placebo comparator: Placebo (Part 1) - Placebo single dose
Experimental: RCT2100 (Part 2) 4 week - RCT2100 multiple dose
Experimental: RCT2100 (Part 2) 12 week - RCT2100 multiple dose
Treatment: Drugs: RCT2100
RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer
Other interventions: Placebo
Placebo of similar volumes to experimental dose strengths administered via oral inhalation using nebulizer
Treatment: Drugs: RCT2100
RCT2100 supplied as varying dose strengths administered via oral inhalation using nebulizer for 4 weeks
Treatment: Drugs: RCT2100
RCT2100 supplied at a single dose strength administered via oral inhalation using nebulizer for 12 weeks
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Intervention code [2]
0
0
Other interventions
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Part 1: The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).
Query!
Assessment method [1]
0
0
Safety and tolerability as assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Query!
Timepoint [1]
0
0
From Baseline Through Day 29
Query!
Primary outcome [2]
0
0
Part 2: The number of participants with CF with AEs and SAEs.
Query!
Assessment method [2]
0
0
Safety and tolerability of multiple-ascending doses of inhaled RCT2100 administered to participants with CF
Query!
Timepoint [2]
0
0
From Day 1 through Safety Follow-up, Week 24
Query!
Eligibility
Key inclusion criteria
Part 1 Major
* 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
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
65
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
Yes
Query!
Key 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
* 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.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 1
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/02/2024
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
30/03/2026
Query!
Actual
Query!
Sample size
Target
158
Query!
Accrual to date
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Alabama
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Arizona
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
California
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Colorado
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Georgia
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Massachusetts
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
New York
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
North Carolina
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Oregon
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Pennsylvania
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Texas
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
Washington
Query!
Country [13]
0
0
France
Query!
State/province [13]
0
0
Montpellier
Query!
Country [14]
0
0
France
Query!
State/province [14]
0
0
Paris
Query!
Country [15]
0
0
Netherlands
Query!
State/province [15]
0
0
Utrecht
Query!
Country [16]
0
0
New Zealand
Query!
State/province [16]
0
0
Auckland
Query!
Country [17]
0
0
United Kingdom
Query!
State/province [17]
0
0
Birmingham
Query!
Country [18]
0
0
United Kingdom
Query!
State/province [18]
0
0
Cambridge
Query!
Country [19]
0
0
United Kingdom
Query!
State/province [19]
0
0
Leeds
Query!
Country [20]
0
0
United Kingdom
Query!
State/province [20]
0
0
London
Query!
Country [21]
0
0
United Kingdom
Query!
State/province [21]
0
0
Nottingham
Query!
Country [22]
0
0
United Kingdom
Query!
State/province [22]
0
0
Southampton
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
ReCode Therapeutics
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.
Query!
Trial website
https://clinicaltrials.gov/study/NCT06237335
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
John Matthews, MBBS, MCRP, PhD
Query!
Address
0
0
ReCode Therapeutics, Inc.
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Priya Riyali, MBA
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
650-629-7900
Query!
Fax
0
0
Query!
Email
0
0
[email protected]
Query!
Contact person for scientific queries
Data sharing statement
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT06237335
Download to PDF