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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
25/09/2024

Titles & IDs
Public title
A Phase 1 Study Evaluating Safety and Tolerability of RCT2100 in Healthy Participants and in Participants with CF
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
Secondary ID [1] 0 0
2024-512169-15
Secondary ID [2] 0 0
RCT2100-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - RCT2100
Other interventions - Placebo

Experimental: RCT2100 (Part 1) - RCT2100 single dose

Placebo comparator: Placebo (Part 1) - Placebo single dose

Experimental: RCT2100 (Part 2) - 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
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part 1: The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).
Timepoint [1] 0 0
From Baseline Through Day 29
Primary outcome [2] 0 0
Part 2: The number of participants with CF with AEs and SAEs.
Timepoint [2] 0 0
From Day 1 through Safety Follow-up, Week 24

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
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
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% 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, 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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/02/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
2/12/2025
Actual
Sample size
Target
100
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Colorado
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
North Carolina
Country [9] 0 0
United States of America
State/province [9] 0 0
Oregon
Country [10] 0 0
United States of America
State/province [10] 0 0
Pennsylvania
Country [11] 0 0
United States of America
State/province [11] 0 0
Texas
Country [12] 0 0
United States of America
State/province [12] 0 0
Washington
Country [13] 0 0
France
State/province [13] 0 0
Montpellier
Country [14] 0 0
France
State/province [14] 0 0
Paris
Country [15] 0 0
France
State/province [15] 0 0
Toulouse
Country [16] 0 0
Netherlands
State/province [16] 0 0
Utrecht
Country [17] 0 0
New Zealand
State/province [17] 0 0
Auckland
Country [18] 0 0
United Kingdom
State/province [18] 0 0
Birmingham
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Cambridge
Country [20] 0 0
United Kingdom
State/province [20] 0 0
Leeds
Country [21] 0 0
United Kingdom
State/province [21] 0 0
London
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Nottingham
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
ReCode Therapeutics
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT06237335
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
John Matthews, MBBS, MCRP, PhD
Address 0 0
ReCode Therapeutics, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Priya Riyali, MBA
Address 0 0
Country 0 0
Phone 0 0
650-629-7900
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06237335