NCT06659640 · Alnylam Pharmaceuticals
A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
(InsigHHT)
What this study is about
The purpose of this study is to: * evaluate the safety, tolerability, how the drug moves through the body (PK) and how the drug affects the body (PD) of single ascending doses of ALN-6400 in healthy volunteers * evaluate the effectiveness, safety, tolerability and PD of multiple doses of ALN-6400 in adult patients with HHT
View original scientific description
The purpose of this study is to: * evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in healthy volunteers * evaluate the efficacy, safety, tolerability and PD of multiple doses of ALN-6400 in adult patients with HHT
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Is a healthy adult volunteer Part B:
- Is an adult patient with a clinical diagnosis of HHT
Exclusion criteria
- Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> upper limit of normal (ULN)
- Has known human immunodeficiency virus (HIV) infection; or known current or chronic hepatitis C virus or hepatitis B virus infection
- Has an estimated glomerular filtration (eGFR) of \<90 mL/min/1.73m\^2 at screening Part B:
- Has ALT or AST \>2×ULN
- Has total bilirubin \>1.5×ULN
- Has eGFR of \<30 mL/min/1.73m\^2 at screening Parts A and B:
- Is not willing to comply with the contraceptive requirements during the study period Note: other protocol defined inclusion / exclusion criteria apply
Where
- Birmingham, Alabama
- Cypress, California
- Gainesville, Florida
- Indianapolis, Indiana
- Boston, Massachusetts
- Rochester, Minnesota
- St Louis, Missouri
- Chapel Hill, North Carolina
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 12, 2026 · Source of record for eligibility and locations