78 Participants Needed

ALN-AGT01 RVR for Healthy Volunteers

AC
Overseen ByAlnylam Clinical Trial Information Line
Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: Alnylam Pharmaceuticals

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, since this study is for healthy volunteers, it's possible that you may not be on any regular medications.

What makes the drug ALN-AGT01 RVR unique compared to other treatments?

ALN-AGT01 RVR, also known as Zilebesiran, is unique because it uses small interfering RNA (siRNA) to target and silence the angiotensinogen gene, which is a novel approach compared to traditional drugs that typically block the effects of angiotensin II. This mechanism allows for a potentially more direct and long-lasting reduction in blood pressure.12345

What is the purpose of this trial?

The purpose of this study is to evaluate the safety, tolerability and PK of single ascending doses of ALN-AGT01 RVR.

Eligibility Criteria

This trial is for healthy adult volunteers who want to participate in a study evaluating the safety of a new medication. Specific eligibility criteria are not provided, but typically participants must meet certain health standards and not be taking conflicting medications.

Inclusion Criteria

Has a body mass index ≥18 kg/m^2 and ≤28 kg/m^2
I am a healthy adult.

Exclusion Criteria

Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > upper limit of normal (ULN)
My kidney function test shows less than normal results.
I have HIV, hepatitis B, or hepatitis C.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single ascending dose of ALN-AGT01 RVR or placebo

1 day
1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 day
1 visit (in-person)

Treatment Details

Interventions

  • ALN-AGT01 RVR
Trial Overview The trial is testing ALN-AGT01 RVR, which is likely a new drug. Participants will receive either this drug or a placebo (a substance with no therapeutic effect) to assess the drug's safety, how well it's tolerated by the body, and its pharmacokinetics (how it's processed by the body).
Participant Groups
2Treatment groups
Experimental Treatment
Placebo Group
Group I: ALN-AGT01 RVRExperimental Treatment1 Intervention
Participants will be administered a single dose of ALN-AGT01 RVR.
Group II: PlaceboPlacebo Group1 Intervention
Participants will be administered a single dose of placebo.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Alnylam Pharmaceuticals

Lead Sponsor

Trials
81
Recruited
16,100+

Dr. Yvonne Greenstreet

Alnylam Pharmaceuticals

Chief Executive Officer since 2021

MD from the University of Leeds, MBA from INSEAD

Dr. Pushkal Garg

Alnylam Pharmaceuticals

Chief Medical Officer since 2016

MD from Columbia University

Findings from Research

The study demonstrated that adeno-associated virus (AAV)-based vectors can effectively transduce the NeoR gene into various human leukemia cell lines, with the highest efficiency observed in K-562 cells at a multiplicity of infection (MOI) of 7, achieving a 27% transduction rate.
Integration of the NeoR gene into the host genome was confirmed, but it occurred outside the expected chromosome 19q13.3 location, indicating that while AAV can deliver genes to leukemia cells, the integration sites may vary significantly.
Recombinant adeno-associated virus-mediated gene transfer into human leukemia cell lines.Itou, T., Miyamura, K., Abe, A., et al.[2019]
Recombinant adeno-associated virus (rAAV) serotypes 5 and 6 effectively transduce human adipose-derived stem cells (ASCs) without reducing cell viability, making them safer options for gene therapy.
Transduction with rAAV6 Y731F mutant further enhances efficiency while maintaining the adipogenic potential of ASCs, suggesting these vectors are promising for genetic manipulation in cellular therapies.
Transduction of human adipose-derived mesenchymal stem cells by recombinant adeno-associated virus vectors.Locke, M., Ussher, JE., Mistry, R., et al.[2018]
The newly developed AAVR-HeLa cell line, which overexpresses the AAVR receptor, significantly enhances the transduction efficiency of various rAAV serotypes, particularly AAV8 and AAV9, making it a valuable tool for detecting neutralizing antibodies (NAbs) in gene therapy trials.
Using AAVR-HeLa cells, the sensitivity for detecting NAbs increased by at least 10 to 20 times for AAV8 and AAV9, respectively, indicating that this cell line can improve the accuracy of assessments for patient eligibility in gene therapy.
Enhanced sensitivity of neutralizing antibody detection for different AAV serotypes using HeLa cells with overexpressed AAVR.Zheng, Z., Ye, J., Leng, M., et al.[2023]

References

Recombinant adeno-associated virus-mediated gene transfer into human leukemia cell lines. [2019]
Transduction of human adipose-derived mesenchymal stem cells by recombinant adeno-associated virus vectors. [2018]
Enhanced sensitivity of neutralizing antibody detection for different AAV serotypes using HeLa cells with overexpressed AAVR. [2023]
Establishment of a cell line carrying single copy of an exogenous mutant reporter gene for assaying the biological activity of ZFNs. [2012]
Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs. [2021]
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