Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05737485




Registration number
NCT05737485
Ethics application status
Date submitted
20/01/2023
Date registered
21/02/2023
Date last updated
8/03/2024

Titles & IDs
Public title
Study Evaluating the Safety and Tolerability of RCT1100 in Healthy and PCD Subjects
Scientific title
A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study, in Healthy Adult Participants and Open-Label Single Ascending Dose Study in Adults With Primary Ciliary Dyskinesia Caused by Pathogenic Mutations in the DNAI1 Gene to Evaluate the Safety and Tolerability of RCT1100
Secondary ID [1] 0 0
RCT1100-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Ciliary Dyskinesia 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Neurological 0 0 0 0
Parkinson's disease
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - RCT1100

Experimental: RCT1100 - Drug: RCT1100 single dose


Treatment: Drugs: RCT1100
RCT1100 mRNA therapy supplied as varying dose strengths administered via oral inhalation using nebulizer
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs).
Timepoint [1] 0 0
From Baseline Through Day 180

Eligibility
Key inclusion criteria
Major

- Healthy, adult, male or female of, 18-65 years of age, inclusive, at screening.

- Participant has pathogenic mutations in the DNAI1 gene

- The participant has a forced expiratory volume in one second (FEV1) of at least 50%
predicted.

Major
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- History or presence of clinically significant medical, surgical, clinical laboratory,
or psychiatric condition or disease.

- History of cancer, with exception of adequately treated basal cell or squamous cell
carcinoma of the skin.

- Medically significant hemoptysis

- Anticoagulation therapy for the treatment of a pulmonary embolus or has had a
pulmonary embolus in the last 6 months of screening.

- Active tuberculosis infection.

- Laboratory abnormalities in clinical laboratory tests at screening:

1. Serum creatinine level

2. Total bilirubin, aspartate aminotransferase or alanine aminotransferase values

3. Hematological or coagulation values outside the normal reference range

- Any medical history of disease that has the potential to cause a rise in total
bilirubin over the ULN

- History of alcohol abuse or drug addiction with the last year of screening.

- Active smoker (vaping included).

Other protocol defined inclusion/exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
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
18/02/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
21/12/2024
Actual
Sample size
Target
9
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Macquarie University - Sydney
Recruitment postcode(s) [1] 0 0
2109 - Sydney
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Nevada
Country [2] 0 0
United States of America
State/province [2] 0 0
Pennsylvania
Country [3] 0 0
New Zealand
State/province [3] 0 0
Auckland
Country [4] 0 0
United Kingdom
State/province [4] 0 0
London

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 RCT1100 and is designed to provide initial safety and
tolerability data for future clinical studies.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05737485
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 Ryali, MBA
Address 0 0
Country 0 0
Phone 0 0
650-629-7900
Fax 0 0
Email 0 0
clinicaltrials@recodetx.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05737485