FXIPhase 1NCT05844293
Single Ascending Dose of FXI-GalNAc-siRNA in Healthy Subjects
A Phase 1 study testing FXI-GalNAc-siRNA in people with FXI, run by Sirnaomics at 2 sites in 2 countries.
Trial summary
The purpose of this study is to evaluate safety, tolerability, PK, and PD of SAD of FXI-GalNAc-siRNA administered SC to healthy subjects. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- FXI
- What is being tested?
- FXI-GalNAc-siRNA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 16 May 2023to Jun 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Subjects who meet all the following criteria will be eligible to participate in the study:
- Male or female subjects 18 to 55 years of age, inclusive, at the time of signing the informed consent form (ICF).
- Subjects must be in generally reasonable health with clinically insignificant screening and admission results (medical history, 12-lead electrocardiogram (ECG), physical examination, and laboratory tests), as determined by the Investigator.
- Subjects must have a body mass index (BMI) ≥ 18 kg/m2 or ≤ 32 kg/m2 at Screening and Day -1 (Admission).
- Subjects must have aspartate transaminase (AST), alanine transaminase (ALT), total bilirubin (TBIL), alkaline phosphatase (ALP), and glucose within upper limit of normal (ULN) range per reference laboratory reference ranges at Screening and Day -1 (Admission); one repeat laboratory analysis allowed per Investigator discretion.
- Subjects must have baseline Factor XI plasma levels within normal range as per testing laboratory requirements.
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- Subjects must have prothrombin time (PT) and partial thromboplastin time (PTT) within the ULN range per reference laboratory reference ranges at Screening and Day -1 (Admission); one repeat laboratory analysis allowed per Investigator discretion.
- Women of childbearing potential must have a negative serum pregnancy test at Screening and negative urine pregnancy test at Day -1 (Admission), must not be actively breastfeeding, or planning to become pregnant during the study, and if not practicing abstinence from heterosexual activity that could result in conception (if this is the preferred and usual lifestyle of the subject), must agree to use 2 approved methods of birth control, during the study and for at least 140 days after study drug administration from the list below:
- Condom plus diaphragm with spermicide started at least 28 days prior to dosing.
- Condom plus cervical cap or female condom with spermicide started at least 28 days prior to dosing.
- Hormonal contraceptives (stable dose for 28 days [4 weeks] prior to Screening) plus condom.
- Intrauterine device (in place for 28 days [4 weeks] prior to Screening) plus condom.
- Condom plus spermicide.
- Partner vasectomy and use of barrier contraception methods (eg, male condom, diaphragm, or sponge with spermicide).
- Women of nonchildbearing potential must be either surgically sterile (partial/total hysterectomy, bilateral oophorectomy) for at least 6 months confirmed by medical/operative report, or if medical/operative report is not available, confirmed by follicle stimulating hormone [FSH] and 17β-estradiol tests) or > 1 year in the postmenopausal women confirmed by FSH and 17β-estradiol tests.
- Men must agree to use barrier contraception (condom with a female partner of child-bearing potential who is using oral contraceptives, hormonal patch, implant or injection, intrauterine device or diaphragm with spermicide) and refrain from sperm donation from the day of study drug administration until at least 140 days after study drug administration. Post vasectomy, men must agree to use a barrier method (or partner barrier method) from the day of study drug administration until at least 140 days after study drug administration. Abstinence as a method of contraception is acceptable if it is in line with the preferred and usual lifestyle of the study participant.
- Subjects must be informed of the nature of the study and must have agreed to and be able to read, review, and sign the study ICF prior to any study-related procedure being performed.
- NOTE: If laboratory values are abnormal, tests may be repeated once at Investigator discretion.
Who cannot join
- Subjects who meet any of the following criteria will be excluded from participation in the study:
- Patients with known diagnosis of Hemophilia C, Factor XI deficiency or another known bleeding disorder.
- Current or previous cancer except superficial forms of non-melanoma skin cancers that have been resolved with clear histology reporting; diabetes or known diagnosis of prediabetes; or any clinically significant cardiovascular (including ECG disturbances or history of rhythm disturbance/abnormality), endocrine, renal, hepatic, gastrointestinal, hematologic, respiratory, dermatological, neurological, psychiatric disorder that could be deemed by the investigator to interfere with study participation, or other disorders that the investigator deems could interfere with study participation.
- Hypertension, defined as blood pressure > 140/90 mmHg (2 separate readings at least 15 minutes apart if first reading is not within normal range) at Screening and Day -1 (Admission).
- Hyperlipidemia, defined as:
- Cholesterol > 300 mg/dL.
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- Low-density lipoprotein cholesterol > 190 mg/dL.
- And/or triglycerides >500 mg/dL.
- Hemoglobin A1c (HbA1c) > 6.0% at Screening. Note: Abnormal laboratory values may be repeated once at Investigator discretion
- Hemoglobin less than 12 g/dL for females and less than 13 g/dL for males or hematocrit outside upper or lower limits of normal range per reference laboratory range at both Screening and Day -1 (Admission).
- Serum creatinine above ULN per reference laboratory range at both Screening and Day -1 (Admission).
- Estimated glomerular filtration rate (eGFR) < 80 mL/min/1.73 m2. If GFR value is not supplied by local laboratory, it can be calculated using the Chronic Kidney Disease Epidemiology Collaboration equation at Screening and Day -1 (Admission). One retest of the exclusionary eGFR value is allowed at the discretion of the Investigator.
- Unwillingness to abstain from alcohol for 72 hours prior to dosing through the EOS Visit (or ET).
- Use of prescription or nonprescription drugs (excluding hormonal contraceptives), if clinically applicable, including vitamins, supplements, herbal preparations, and medicines that prolong Q wave interval (QT)/ Q wave interval corrected (QTc) within 7 days or 5 times longer than the half-life (whichever is longer) prior to the study drug administration through the EOS Visit (approximately 140 days) or at ET.
- Blood donation within 56 days or plasma donation within 10 days prior to dosing.
- Use of live or non-live vaccine (except SARS-CoV-2 and influenza) within 30 days prior to the first dose of study drug or an intention to receive such a vaccine at any time during the study.
- Received an investigational agent in another clinical study within 30 days prior to the first dose of study drug, or within 10 times longer than the half-life of the compound with which the subject was treated, whichever is longer.
- Positive results of any of the virology tests (human immunodeficiency virus [HIV test], hepatitis B virus [HBsAg test] or hepatitis C virus [hepatitis C antibody test]).
- Diagnosed to be SARS-CoV-2 positive based on study center SARS-CoV-2 testing guidelines on Day -1.
- Personal or family history of vascular aneurysms and disorders.
- Personal or family history of long QT syndrome.
- Corrected QT interval using Fridericia's formula (> 450 msec) based on the average of triplicate ECGs.
- History of active alcoholism or active drug abuse as that would impair the ability of a subject to participate in the study.
- Positive urine drug or breath alcohol screen at Screening or Day -1 (Admission) and at all visits after confinement, verbal confirmation of compliance.
- Any condition that, in the opinion of the Investigator, would complicate or compromise the study data or the well-being of the subject.
- NOTE: If laboratory values are abnormal, tests may be repeated once at Investigator discretion.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: Single-center, Randomized, Double-blind, Placebo-controlled, Sequential Cohort Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SAD of FXI-GalNAc-siRNA When Administered Subcutaneously to Healthy Subjects.
The purpose of this study is to evaluate safety, tolerability, PK, and PD of SAD of FXI-GalNAc-siRNA administered SC to healthy subjects.
Read the detailed description
Phase 1, Single center, randomized, double-blind, sequential cohort study to evaluate safety, tolerability, PK and PD of a single ascending dose of FXI-GalNAc-siRNA when administered subcutaneously in healthy subjects compared with Normal Saline Injection.
The study will include a screening period, treatment period, and a follow-up period.
Five dose cohorts will be evaluated.
- Conditions
- FXI
- Being tested
- FXI-GalNAc-siRNA, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FXI-GalNAc-siRNADrug
Also called FXI-GalNAc-siRNA Solution for Injection
FXI-GalNAc-siRNA solution for injection
PlaceboOther
Saline
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Other | 6 subjects injected with 25 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline Receives: Drug: FXI-GalNAc-siRNA; Other: Placebo |
| Cohort 2 | Other | 6 subjects injected with 50 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline Receives: Drug: FXI-GalNAc-siRNA; Other: Placebo |
| Cohort 3 | Other | 6 subjects injected with 100 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline Receives: Drug: FXI-GalNAc-siRNA; Other: Placebo |
| Cohort 4 | Other | 6 subjects injected with 200 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline Receives: Drug: FXI-GalNAc-siRNA; Other: Placebo |
| Cohort 5 | Other | 6 subjects injected with 400 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline Receives: Drug: FXI-GalNAc-siRNA; Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum peak concentration
Individual plasma concentration data of FXI-GalNAc-siRNA will be used to determine Maximum peak concentration.
Measured: 145 days
Time to maximum concentration.
Individual plasma concentration data of FXI-GalNAc-siRNA will be used to determine time to maximum concentration.
Measured: 145 days
Apparent terminal elimination rate constant.
Individual plasma concentration data of FXI-GalNAc-siRNA will be used to determine apparent terminal elimination rate constant.
Measured: 145 days
Half life
Individual plasma concentration data of FXI-GalNAc-siRNA will be used to determine half life.
Measured: 145 days
Area under the curve
Individual plasma concentration data of FXI-GalNAc-siRNA will be used to determine area under the plasma concentration-time curve from 0 to the last quantifiable plasma concentration.
Measured: 145 days
Area under the curve from 0-infinity
Individual plasma concentration data of FXI-GalNAc-siRNA will be used to determine area under the curve from time 0 to infinity.
Measured: 145 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2023
- Study start16 May 2023
- Primary completionMay 2027 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for FXIStudies 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
- Sequential
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sirnaomics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Francois Lebel, MD, Study director, Chief Medical Officer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05844293
- Sponsor's protocol ID
- SRN-122-001
- Status last verified
- Jul 2026
- First submitted
- 13 Apr 2023
- Last updated
- 29 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single-center, Randomized, Double-blind, Placebo-controlled, Sequential Cohort Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SAD of FXI-GalNAc-siRNA When Administered Subcutaneously to Healthy Subjects.
- ClinicalTrials.gov ID
- NCT05844293
- Acronym / sponsor ID
- SRN-122-001
- Conditions
- FXI
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- SirnaomicsIndustry (pharma, biotech, devices)
- Study start
- 16 May 2023 (actual)
- Primary completion
- May 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 6 May 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.
The purpose of this study is to evaluate safety, tolerability, PK, and PD of SAD of FXI-GalNAc-siRNA administered SC to healthy subjects.
Detailed description
Phase 1, Single center, randomized, double-blind, sequential cohort study to evaluate safety, tolerability, PK and PD of a single ascending dose of FXI-GalNAc-siRNA when administered subcutaneously in healthy subjects compared with Normal Saline Injection.
The study will include a screening period, treatment period, and a follow-up period.
Five dose cohorts will be evaluated.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Other | Drug: FXI-GalNAc-siRNA, Other: Placebo6 subjects injected with 25 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline |
| Cohort 2 | Other | Drug: FXI-GalNAc-siRNA, Other: Placebo6 subjects injected with 50 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline |
| Cohort 3 | Other | Drug: FXI-GalNAc-siRNA, Other: Placebo6 subjects injected with 100 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline |
| Cohort 4 | Other | Drug: FXI-GalNAc-siRNA, Other: Placebo6 subjects injected with 200 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline |
| Cohort 5 | Other | Drug: FXI-GalNAc-siRNA, Other: Placebo6 subjects injected with 400 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (18 listed)
- Subjects who meet all the following criteria will be eligible to participate in the study:
- Male or female subjects 18 to 55 years of age, inclusive, at the time of signing the informed consent form (ICF).
- Subjects must be in generally reasonable health with clinically insignificant screening and admission results (medical history, 12-lead electrocardiogram (ECG), physical examination, and laboratory tests), as determined by the Investigator.
- Subjects must have a body mass index (BMI) ≥ 18 kg/m2 or ≤ 32 kg/m2 at Screening and Day -1 (Admission).
- Subjects must have aspartate transaminase (AST), alanine transaminase (ALT), total bilirubin (TBIL), alkaline phosphatase (ALP), and glucose within upper limit of normal (ULN) range per reference laboratory reference ranges at Screening and Day -1 (Admission); one repeat laboratory analysis allowed per Investigator discretion.
Key exclusion criteria (26 listed)
- Subjects who meet any of the following criteria will be excluded from participation in the study:
- Patients with known diagnosis of Hemophilia C, Factor XI deficiency or another known bleeding disorder.
- Current or previous cancer except superficial forms of non-melanoma skin cancers that have been resolved with clear histology reporting; diabetes or known diagnosis of prediabetes; or any clinically significant cardiovascular (including ECG disturbances or history of rhythm disturbance/abnormality), endocrine, renal, hepatic, gastrointestinal, hematologic, respiratory, dermatological, neurological, psychiatric disorder that could be deemed by the investigator to interfere with study participation, or other disorders that the investigator deems could interfere with study participation.
- Hypertension, defined as blood pressure > 140/90 mmHg (2 separate readings at least 15 minutes apart if first reading is not within normal range) at Screening and Day -1 (Admission).
- Hyperlipidemia, defined as:
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 |
|---|---|---|
| FXI-GalNAc-siRNAAlso: FXI-GalNAc-siRNA Solution for InjectionFXI-GalNAc-siRNA solution for injection | Drug | Cohort 1, Cohort 2, Cohort 3, Cohort 4, Cohort 5 |
| PlaceboSaline | Other | Cohort 1, Cohort 2, Cohort 3, Cohort 4, Cohort 5 |
Primary outcome
Maximum peak concentration
Time frame: 145 days
Individual plasma concentration data of FXI-GalNAc-siRNA will be used to determine Maximum peak concentration.
Time to maximum concentration.
Time frame: 145 days
Individual plasma concentration data of FXI-GalNAc-siRNA will be used to determine time to maximum concentration.
Apparent terminal elimination rate constant.
Time frame: 145 days
Individual plasma concentration data of FXI-GalNAc-siRNA will be used to determine apparent terminal elimination rate constant.
Half life
Time frame: 145 days
Individual plasma concentration data of FXI-GalNAc-siRNA will be used to determine half life.
Area under the curve
Time frame: 145 days
Individual plasma concentration data of FXI-GalNAc-siRNA will be used to determine area under the plasma concentration-time curve from 0 to the last quantifiable plasma concentration.
Area under the curve from 0-infinity
Time frame: 145 days
Individual plasma concentration data of FXI-GalNAc-siRNA will be used to determine area under the curve from time 0 to infinity.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2026.
- 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.
Francois Lebel, MD study director
Chief Medical Officer
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing FXI-GalNAc-siRNA in people with FXI, run by Sirnaomics at 2 sites in 2 countries.
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 18 inclusion and 26 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: under way, not looking for participants, as of the sponsor's last update on 29 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, Canada. 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. 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?
This study is still under way. Results are not available in the source record yet.
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?
Maximum peak concentration (time frame: 145 days). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 29 Jul 2026.