Idiopathic Pulmonary FibrosisPhase 1NCT05984992
The First-in-human Study of SRN-001 in Healthy Participants
A Phase 1 study testing SRN-001 in people with Idiopathic Pulmonary Fibrosis, run by siRNAgen Therapeutics Inc. at 1 site in Australia.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Idiopathic Pulmonary Fibrosis
- What is being tested?
- SRN-001
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 25 participants
- Where
- 1 siteAustralia
- Study dates
- 8 Sep 2023to 25 Sep 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 22Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Double-blinded, Placebo-controlled, Single Ascending Dose Study to Assess the Safety, Tolerability and Pharmacokinetics of SRN-001 in Healthy Participants
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-β (TGF-β) 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.
Read the detailed description
Participants with part in consent will be enrolled in a phase 1a study of SRN-001. Prior to initiation of treatment, participants will undergo several screening test for checking their condition of health. There is no specific test comparing with the general other clinical trial in healthy volunteers. They will be randomized into two groups, active drug and inactive placebo(normal saline) as ratio 2:1. Starting dose is planned 15mg. For confirming maximal tolerable dose, dose will be escalated when no dose-limiting toxicity (DLT) confirmed. Each cohort will take single dose and for 4 weeks, safety observation will be taken. If safety abnormality will be retained in 4 weeks, the participant's safety observation will be prolonged by the end of the adverse event once 2 weeks.
- Conditions
- Idiopathic Pulmonary Fibrosis
- Being tested
- SRN-001, Placebo
- Medical subject headings
- Idiopathic Pulmonary Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SRN-001Drug
siRNA therapeutics, Self Assembled Micelle inhibitory RNA platform utilized
PlaceboDrug
0.9% Sodium Chloride(Normal saline)
| Study group | Role | What they receive |
|---|---|---|
| SRN-001 | Experimental | Not described Receives: Drug: SRN-001 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-emergent adverse events(TEAEs)
Measured: Up to 4 weeks
Number of participants with serious adverse events(SAEs)
Measured: Up to 4 weeks
Secondary outcomes (10)
Cmax
Maximum observed concentration
Measured: Up to 168 hours post-dose
Clast
Observed concentration corresponding to Tlast
Measured: Up to 168 hours post-dose
Tlast
Time of last measurable observed concentration
Measured: Up to 168 hours post-dose
AUClast
Area under the drug concentration-time curve, from time zero to the last measurable concentration
Measured: Up to 168 hours post-dose
AUCinf
Area under the drug concentration-time curve, from time zero to infinity
Measured: Up to 168 hours post-dose
T½
Apparent terminal half-life
Measured: Up to 168 hours post-dose
Kel
Apparent terminal elimination rate constant
Measured: Up to 168 hours post-dose
CL
Total body clearance
Measured: Up to 168 hours post-dose
Vz
Volume of distribution
Measured: Up to 168 hours post-dose
MRT
Mean residence time
Measured: Up to 168 hours post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2023
- Study start8 Sep 2023
- Primary completion15 Mar 2024
- Study completion25 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Idiopathic Pulmonary FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized, Double-blinded, Placebo-controlled, Single Ascending Dose
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- siRNAgen Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05984992
- Sponsor's protocol ID
- SRN-001-C01
- Status last verified
- Oct 2024
- First submitted
- 26 Jul 2023
- Last updated
- 4 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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.
PubMed 26817844
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blinded, Placebo-controlled, Single Ascending Dose Study to Assess the Safety, Tolerability and Pharmacokinetics of SRN-001 in Healthy Participants
- ClinicalTrials.gov ID
- NCT05984992
- Acronym / sponsor ID
- SRN-001-C01
- Conditions
- Idiopathic Pulmonary Fibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- siRNAgen Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 8 Sep 2023 (actual)
- Primary completion
- 15 Mar 2024 (actual)
- Study completion
- 25 Sep 2024 (actual)
- First posted
- 9 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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-β (TGF-β) 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.
Detailed description
Participants with part in consent will be enrolled in a phase 1a study of SRN-001. Prior to initiation of treatment, participants will undergo several screening test for checking their condition of health. There is no specific test comparing with the general other clinical trial in healthy volunteers. They will be randomized into two groups, active drug and inactive placebo(normal saline) as ratio 2:1. Starting dose is planned 15mg. For confirming maximal tolerable dose, dose will be escalated when no dose-limiting toxicity (DLT) confirmed. Each cohort will take single dose and for 4 weeks, safety observation will be taken. If safety abnormality will be retained in 4 weeks, the participant's safety observation will be prolonged by the end of the adverse event once 2 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Randomized, Double-blinded, Placebo-controlled, Single Ascending Dose
| Arm | Type | Receives |
|---|---|---|
| SRN-001 | Experimental | Drug: SRN-001 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (22 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SRN-001siRNA therapeutics, Self Assembled Micelle inhibitory RNA platform utilized | Drug | SRN-001 |
| Placebo0.9% Sodium Chloride(Normal saline) | Drug | Placebo |
Primary outcome
Number of participants with treatment-emergent adverse events(TEAEs)
Time frame: Up to 4 weeks
Number of participants with serious adverse events(SAEs)
Time frame: Up to 4 weeks
10 secondary outcomes
Cmax
Time frame: Up to 168 hours post-dose
Clast
Time frame: Up to 168 hours post-dose
Tlast
Time frame: Up to 168 hours post-dose
AUClast
Time frame: Up to 168 hours post-dose
AUCinf
Time frame: Up to 168 hours post-dose
T½
Time frame: Up to 168 hours post-dose
Kel
Time frame: Up to 168 hours post-dose
CL
Time frame: Up to 168 hours post-dose
Vz
Time frame: Up to 168 hours post-dose
MRT
Time frame: Up to 168 hours post-dose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2024.
- Background: PubMed 26817844Yoon 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.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SRN-001 in people with Idiopathic Pulmonary Fibrosis, run by siRNAgen Therapeutics Inc. at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-emergent adverse events(TEAEs) (time frame: Up to 4 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05984992, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2024.