A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of SRN001 in Healthy Korean and Caucasian Adult Males

NCT07407543 | Recruiting Phase 1

• 1 other identifier: SRN001-CA-25001A
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of repeated doses of SRN001 in healthy adult volunteers.

Conditions

Idiopathic Pulmonary Fibrosis

Outcome Measures

Primary Outcomes (18)

• Incidence of Treatment-Emergent Adverse Events (TEAEs)

  Number of participants experiencing one or more TEAEs during the study period.

  From first dose through end of study (up to 114 days)

• Number of participants with serious adverse events (SAEs)

  Number of participants with SAEs as defined in protocol.

  From first dose through end of study (up to 114 days)

• Number of participants with clinically significant abnormal laboratory results

  Counts of clinically significant abnormal lab tests during study.

  From first dose through end of study (up to 114 days)

• Maximum Observed Plasma Concentration (Cmax) of SRN001

  Maximum observed plasma concentration (Cmax) following IV administration of SRN001.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose)

• Time to Maximum Plasma Concentration (Tmax) of SRN001

  Time to reach maximum observed plasma concentration following IV administration.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Area Under the Curve from time zero to last measurable concentration (AUClast)

  AUClast of SRN001 plasma concentration versus time curve.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Area Under the Plasma Concentration-Time Curve over the Dosing Interval (AUCtau) of SRN001

  AUCtau will be calculated as the area under the plasma concentration versus time curve over one complete dosing interval following multiple escalating intravenous doses of SRN001.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Terminal Half-Life (t½) of SRN001

  Terminal elimination half-life (t½) will be calculated from the plasma concentration-time profile at steady state following multiple doses.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Clearance (CL) of SRN001

  Systemic clearance (CL) will be determined from non-compartmental analysis of plasma concentrations at steady state after multiple dosing.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Apparent Volume of Distribution (Vz) of SRN001

  Apparent volume of distribution (Vz) will be calculated from plasma concentration data at steady state following multiple doses.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Time to Maximum Observed Plasma Concentration at Steady State (Tmax,ss) of SRN001

  Time to reach maximum observed plasma concentration at steady state following multiple intravenous doses.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Maximum Observed Plasma Concentration at Steady State (Cmax,ss) of SRN001

  Maximum observed plasma concentration at steady state following multiple intravenous doses.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Minimum Observed Plasma Concentration at Steady State (Cmin,ss) of SRN001

  Minimum observed plasma concentration at steady state following multiple doses.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Average Plasma Concentration at Steady State (Cavg,ss) of SRN001

  Average plasma concentration at steady state following multiple intravenous doses.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Trough Plasma Concentration at Steady State (Ctrough) of SRN001

  Plasma concentration just prior to the next dose at steady state following multiple dosing.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Area Under the Plasma Concentration-Time Curve over the Dosing Interval at Steady State (AUCtau,ss) of SRN001

  AUCtau,ss will be calculated over one dosing interval at steady state following multiple intravenous doses.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Peak-to-Trough Fluctuation (PTF) of SRN001 at Steady State Description: Peak-to-trough fluctuation in plasma concentration at steady state, defined as (Cmax,ss - Cmin,ss)/Cavg,ss.

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose)

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

• Accumulation Ratio (R) of SRN001 at Steady State

  Accumulation ratio (R) comparing exposure at steady state with that after the first dose (e.g., based on Cmax or AUC).

  Pre-dose samples: Day 1, Day 15, and Day 29 (pre-dose) Post-dose samples (Day 1 and Day 29): 0 (end of infusion), 10, 20, 30, and 45 minutes, and 1, 1.5, 2, 3, 6, 12, 24, and 48 hours after dosing

Other Outcomes (20)

• Changes in Serum Amphiregulin Concentration

  Changes from baseline in circulating amphiregulin concentrations will be evaluated using 11 blood sampling time points, including pre-dose, early post-dose, and follow-up assessments.

• Changes in PBMC LPS-Stimulated Amphiregulin Levels

  Baseline to Day 29 (pre-dose on Days 1, 15, and 29; post-dose at 2 and 24 hours on Days 1 and 29)

• Changes in Urine Albumin-to-Creatinine Ratio (uACR)

  Day 1 to 48 hours post-dose on Day 29 (pre-dose and 0-3, 3-6, 6-12, 12-24, and 24-48 hours post-dose)

Study Arms (4)

cohort1 Drug: SRN001 45mg

EXPERIMENTAL

cohort1 Drug: SRN001 45mg

Drug: SRN001

cohort2 Drug: SRN001 90mg

EXPERIMENTAL

cohort2 Drug: SRN001 90mg

Drug: SRN001

cohort3 Drug: SRN001 180mg

EXPERIMENTAL

cohort3 Drug: SRN001 180mg

Drug: SRN001

Placebo Comparator

PLACEBO COMPARATOR

Participants receive placebo (0.9% sodium chloride, normal saline)

Drug: 0.9% sodium chloride (normal saline)

Interventions

SRN001 DRUG

SRN001 is an investigational drug administered at doses of 45 mg, 90 mg, or 180 mg depending on cohort.

cohort1 Drug: SRN001 45mg; cohort2 Drug: SRN001 90mg; cohort3 Drug: SRN001 180mg

0.9% sodium chloride (normal saline) DRUG

0.9% sodium chloride solution administered as placebo control.

Placebo Comparator

Eligibility Criteria

• Age: 19 Years - 60 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Healthy Korean or Caucasian male volunteers aged 19 to 60 years at the time of screening.
• Those who weighed 50.0 kg or more at the time of screening and had a body mass index (BMI) between 18.5 kg/m2 and 29.9 kg/m2.
• Body mass index (BMI, kg/m2) = weight (kg) / {height (m2)} 2
• Those whose screening results showed a serum amphiregulin concentration of 100 pg/mL or higher.
• Those who voluntarily agreed to participate in this clinical trial after receiving a thorough explanation and fully understanding the clinical trial. Those who decided to participate and gave written consent to comply with the precautions.

You may not qualify if:

• Those with or have a history of clinically significant diseases of the hepatobiliary system (severe liver failure, viral hepatitis, etc.), kidney (severe renal failure, etc.), nervous system, immune system, respiratory system, digestive system, endocrine system, hematological/oncological system, cardiovascular system (heart failure, torsades de pointes, etc.), urinary system, psychiatric system (mood disorder, obsessive-compulsive disorder, etc.), or sexual dysfunction.
• Those with a history of hypersensitivity to RNA drugs or other drugs (aspirin, antibiotics, etc.) or a history of clinically significant hypersensitivity reactions (atopy, asthma, etc.).
• Those with a positive serum test result (hepatitis B test, hepatitis C test, human immunodeficiency virus (HIV) test, syphilis test).
• Those with a history of drug abuse or a positive urine drug screening test for drugs of abuse.
• Those who were screened in a sitting position after resting for at least 3 minutes. Those who exhibited the following values in measured vital signs:
• Systolic blood pressure \< 80 mmHg or ≥ 140 mmHg
• Diastolic blood pressure \< 45 mmHg or ≥ 90 mmHg
• Pulse \< 45 bpm or \> 100 bpm
• Body temperature \< 35.5 ℃ or \> 37.7 ℃
• Those who exhibited the following values or clinically significant abnormal rhythm findings on the electrocardiogram (12-lead ECG) during the screening test:
• QTcF \> 450 msec
• Those who exhibited one or more of the following results in clinical laboratory tests during the screening test, including additional tests:
• AST (SGOT) or ALT (SGPT) \> 60 IU/L
• Estimated glomerular filtration rate (CKD-EPI equation) \< 60 mL/min/1.73 m2
• Those who have taken any prescription drugs or herbal medicines within two weeks prior to the scheduled first administration of the investigational drug, or have taken any over-the-counter drugs, health functional foods including liver function supplements, or vitamin preparations within one week (however, at the investigator's discretion, subjects may be selected as subjects if other conditions are appropriate) or are expected to take such drugs.

Sponsors & Collaborators

• siRNAgen Therapeutics Inc.: lead

Study Sites (1)

Seoul National University Hospital

Seoul, South Korea

RECRUITING

Related Publications (1)

• 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.

  PMID: 26817844 BACKGROUND

Related Links

• Related Info

MeSH Terms

Conditions

Idiopathic Pulmonary Fibrosis

Interventions

Sodium Chloride; Saline Solution

Condition Hierarchy (Ancestors)

Pulmonary Fibrosis; Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds; Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Central Study Contacts

Eunkyeong Woo

CONTACT

+82-42-930-8654; wek@sirnagen.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 5, 2026
• First Posted: February 12, 2026
• Study Start: January 5, 2026
• Primary Completion (Estimated): October 31, 2026
• Study Completion (Estimated): April 30, 2027
• Last Updated: February 12, 2026

Record last verified: 2026-02

Locations

KR (1)