FXI-GalNAc-siRNA Safety and Tolerability in Healthy Subjects
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial evaluates the safety and tolerability of a new treatment called FXI-GalNAc-siRNA in healthy individuals. Different groups will receive various doses of the treatment, while some will receive a placebo, a harmless substance used as a control. The trial aims to understand how the body processes the treatment and its effects. Ideal participants are those in good health, without bleeding disorders, and willing to follow specific lifestyle and medication guidelines. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive it.
Is there any evidence suggesting that FXI-GalNAc-siRNA is likely to be safe for humans?
Research has shown that FXI-GalNAc-siRNA has been safe in previous studies. One study found that a similar treatment was well tolerated, with no major side effects, and it successfully lowered FXI levels in the body without causing problems. Another report confirmed that patients did not experience any serious safety issues, suggesting this treatment is generally safe for people. Although this is a new study, these earlier results suggest that FXI-GalNAc-siRNA might be well-tolerated by participants.12345
Why do researchers think this study treatment might be promising?
Researchers are excited about FXI-GalNAc-siRNA because it represents a new approach to treating conditions related to blood clotting. Unlike traditional blood thinners that work by targeting different parts of the coagulation process, FXI-GalNAc-siRNA specifically targets and silences the FXI gene, which plays a crucial role in thrombosis. This targeted mechanism may reduce the risk of bleeding compared to current options. Additionally, the treatment is delivered using a GalNAc conjugation, designed to enhance delivery specifically to the liver, where FXI is produced, potentially improving effectiveness and safety.
What evidence suggests that FXI-GalNAc-siRNA could be effective?
Research has shown that FXI-GalNAc-siRNA can lower the activity of Factor XI (FXI), which plays a role in blood clotting. In animal studies, this treatment significantly reduced FXI levels. Earlier research found it could decrease FXI by over 90%, potentially helping to prevent unwanted blood clots. Early human trials reported that the treatment was well tolerated, with few serious side effects. This trial will assess the safety and tolerability of FXI-GalNAc-siRNA in healthy subjects, suggesting it could effectively manage conditions requiring blood clot prevention.12356
Who Is on the Research Team?
Francois Lebel, MD
Principal Investigator
Chief Medical Officer
Are You a Good Fit for This Trial?
Healthy men and women aged 18-55, with a BMI between 18 and 32 kg/m2, normal liver function tests, normal Factor XI levels, and no history of significant health issues. Women must not be pregnant or breastfeeding and agree to use two forms of contraception if they can become pregnant. Men also need to use barrier contraception.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single ascending dose of FXI-GalNAc-siRNA or saline
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- FXI-GalNAc-siRNA
Trial Overview
The trial is testing the safety, how well tolerated it is, the pharmacokinetics (how the drug moves through the body), and pharmacodynamics (the effects of the drug) of a single subcutaneous dose of FXI-GalNAc-siRNA compared to a placebo in healthy participants.
How Is the Trial Designed?
5
Treatment groups
Experimental Treatment
6 subjects injected with 400 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline
6 subjects injected with 200 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline
6 subjects injected with 100 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline
6 subjects injected with 50 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline
6 subjects injected with 25 mg dose of FXI-GalNAc-siRNA 2 subjects with Saline
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sirnaomics
Lead Sponsor
Citations
Inhibition of Factor XI Using RBD4059 - PMC - NIH
In the present study, the GalNAc-siRNA molecule RBD4059 showed promising reduction of FXI activity in preclinical animal models and ...
First-in-human study to evaluate the safety, pharmacokinetics ...
GalNAc-siRNA drug targeting coagulation factor XI (FXI), in healthy subjects. Here we report on the safety, PK and PD effects of the GalNAc- ...
NCT05844293 | Single Ascending Dose of FXI-GalNAc- ...
The purpose of this study is to evaluate safety, tolerability, PK, and PD of SAD of FXI-GalNAc-siRNA administered SC to healthy subjects.
FXI-GalNAc-siRNA - Drug Targets, Indications, Patents
... Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SAD of FXI-GalNAc-siRNA When Administered Subcutaneously to Healthy Subjects.
5.
prnewswire.com
prnewswire.com/apac/news-releases/sirnaomics-launches-phase-i-clinical-trial-for-galnac-factor-xi-program-301795342.htmlSirnaomics Launches Phase I Clinical Trial for GalNAc ...
By targeting FXI, the Group has the potential to target multiple diseases that require anticoagulation such as atrial fibrillation, pulmonary ...
6.
ashpublications.org
ashpublications.org/bloodadvances/article/10/7/2541/565939/RNA-interference-therapy-targeting-coagulationa first-in-human trial of RBD4059 (vortosiran) | Blood Advances
Key PointsRBD4059, a novel siRNA targeting FXI, was well tolerated with a durable, dose-dependent suppression of FXI up to >90%.
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