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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/NCT05984992




Registration number
NCT05984992
Ethics application status
Date submitted
26/07/2023
Date registered
9/08/2023
Date last updated
1/04/2024

Titles & IDs
Public title
The First-in-human Study of SRN-001 in Healthy Participants
Scientific title
A Randomized, Double-blinded, Placebo-controlled, Single Ascending Dose Study to Assess the Safety, Tolerability and Pharmacokinetics of SRN-001 in Healthy Participants
Secondary ID [1] 0 0
SRN-001-C01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Idiopathic Pulmonary Fibrosis 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - SRN-001
Treatment: Drugs - Placebo

Experimental: SRN-001 -

Placebo Comparator: Placebo -


Treatment: Drugs: SRN-001
siRNA therapeutics, Self Assembled Micelle inhibitory RNA platform utilized

Treatment: Drugs: Placebo
0.9% Sodium Chloride(Normal saline)
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with treatment-emergent adverse events(TEAEs)
Timepoint [1] 0 0
Up to 4 weeks
Primary outcome [2] 0 0
Number of participants with serious adverse events(SAEs)
Timepoint [2] 0 0
Up to 4 weeks
Secondary outcome [1] 0 0
Cmax
Timepoint [1] 0 0
Up to 168 hours post-dose
Secondary outcome [2] 0 0
Clast
Timepoint [2] 0 0
Up to 168 hours post-dose
Secondary outcome [3] 0 0
Tlast
Timepoint [3] 0 0
Up to 168 hours post-dose
Secondary outcome [4] 0 0
AUClast
Timepoint [4] 0 0
Up to 168 hours post-dose
Secondary outcome [5] 0 0
AUCinf
Timepoint [5] 0 0
Up to 168 hours post-dose
Secondary outcome [6] 0 0
T½
Timepoint [6] 0 0
Up to 168 hours post-dose
Secondary outcome [7] 0 0
Kel
Timepoint [7] 0 0
Up to 168 hours post-dose
Secondary outcome [8] 0 0
CL
Timepoint [8] 0 0
Up to 168 hours post-dose
Secondary outcome [9] 0 0
Vz
Timepoint [9] 0 0
Up to 168 hours post-dose
Secondary outcome [10] 0 0
MRT
Timepoint [10] 0 0
Up to 168 hours post-dose

Eligibility
Key inclusion criteria
- Age 18-70

- BMI =18.0 kg/? and =35 kg/?

- 12-lead triplicate electrocardiogram (ECG) readings within normal limits or with no
clinically significant abnormalities

- systolic blood pressure = 90 mmHg and =160 mmHg; a diastolic blood pressure = 50 mmHg
and =95 mmHg; pulse = 45 bpm and =100 bpm; tympanic temperature = 35.5°C and =37.7°C
and respiratory rate 12rpm to 22rpm

- Negative urinary cotinine

- Compliance to contraception and sperm donation restriction

- Participants who are able and willing to give written informed consent

- Fully vaccinated against SARS-CoV-2
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
- Who has clinically significant history

- Who is with history of multiple drug allergies or history of allergic reaction to an
oligonucleotide or common medicine (eg, aspirin, antibiotics, etc) or clinically
significant hypersensitivity

- No tolerance to IV injections or significant potential of intolerance

- Clinically significant surgical history within 1 year

- History of drug abuse or alcoholism within 2 years, and a restriction of consuming
alcohol during study period

- Pregnant or lactating females

- Liver function test is 1.5 times greater than upper limit of normal (ULN)

- Albumin = 35 g/L and = 50 g/L

- Hb < 115 g/L (female), < 125 g/L (male)

- estimated glomerular filtration rate (eGFR) < 60 mL/min (CKD-EPI), 90 mL/min (MDRD)

- Glucose < 3 mmol/L

- Positive screen for alcohol or drugs of abuse

- HBsAg, Hepatitis B virus (HBV), Hepatitis C virus (HCV), or HIV infection

- QTcF > 450 msec for male, > 470 msec for female

- Inappropriate lab result by physician's discretion

- Who have donated > 500 mL of blood within 3 months

- Who have received an investigational agent within 3 months, or 5 half-lives

- Who have used prescription medication within 4 weeks including vaccines

- Who have used OTC medication within 7 days

- With clinically relevant wounds, following a clinically relevant surgery or have
recently completed any invasive procedures (ie, Endoscopy) within 1 week, or who are
scheduled for an elective surgical procedure

- Who have a significant infection or known inflammatory process ongoing

- Any conditions that, in physician's opinion, would make the participant unsuitable for
enrollment or could interfere with the participant's participation

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 administering the treatment/s
The people assessing the outcomes
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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/09/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
15/08/2024
Actual
Sample size
Target
24
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
CMAX Clinical Research - Adelaide
Recruitment postcode(s) [1] 0 0
5000 - Adelaide

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
siRNAgen Therapeutics Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
SRN-001 is a novel small interfering RNA (siRNA) drug being developed to treat fibrosis using
Self Assembled Micelle inhibitory ribonucleic acid (SAMiRNA™) technology. Amphiregulin (AREG)
is a growth factor involved in fibroblast proliferation and myofibroblast transformation
which is the hallmark of fibrosis in lung and kidney tissues. AREG is a downstream gene
overexpressed by Transforming growth factor-ß (TGF-ß) during fibrosis, promoting fibroblast
to myofibroblast transition (FMT). SRN-001 is designed to downregulate generating
amphiregulin by RNA interference (RNAi). The goal of this clinical trial is to evaluate
safety, tolerability, and pharmacokinetics in healthy participants. This trial is
first-in-human clinical trial to develop SAMiRNA™ to utilize as therapeutic use.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05984992
Trial related presentations / publications
Yoon PO, Park JW, Lee CM, Kim SH, Kim HN, Ko Y, Bae SJ, Yun S, Park JH, Kwon T, Kim WS, Lee J, Lu Q, Kang HR, Cho WK, Elias JA, Yang JS, Park HO, Lee K, Lee CG. Self-assembled Micelle Interfering RNA for Effective and Safe Targeting of Dysregulated Genes in Pulmonary Fibrosis. J Biol Chem. 2016 Mar 18;291(12):6433-46. doi: 10.1074/jbc.M115.693671. Epub 2016 Jan 27.
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries