A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
This research study, called 'A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)', is investigating a new potential treatment, ALN-6400. The main goal is to understand if ALN-6400 is safe and how the body handles it in healthy volunteers. It will then look at whether it's effective, safe, and well-tolerated in adults who have Hereditary Hemorrhagic Telangiectasia (HHT). This study involves two main parts: one for healthy adults and another for adults diagnosed with HHT. Researchers hope this study will provide important information about a new way to manage HHT.
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¿De qué trata este estudio?
This study is investigating a new medication called ALN-6400, which is being developed as a potential treatment for Hereditary Hemorrhagic Telangiectasia, often shortened to HHT. HHT is a genetic condition that causes abnormal blood vessel formation, which can lead to frequent nosebleeds, bleeding in other parts of the body, and other health issues.
The study has two main goals. The first is to learn about the safety of ALN-6400 and how the body processes it in healthy people. This helps researchers understand if the new medication is generally well-tolerated at different doses before giving it to people who are unwell. The second goal is to see how effective ALN-6400 is, as well as its safety and how well people with HHT tolerate it when they take multiple doses.
By taking part in this study, you could help doctors understand more about ALN-6400 and whether it could become a useful treatment for people with HHT in the future. Finding new and better ways to manage HHT is very important for improving the quality of life for those affected by this condition.
Puntos clave
- This study is testing a new medication (ALN-6400) for Hereditary Hemorrhagic Telangiectasia (HHT).
- It's looking at safety in healthy people and effectiveness in people with HHT.
- Participation involves clinic visits, examinations, and blood tests.
- There are specific health requirements to join the study.
- You have the right to leave the study at any time.
- Your involvement could help advance treatment for HHT.
¿Quién puede ser elegible?
To join this study, you'll need to be an adult, aged 18 or over. The study is split into two parts. In Part A, we're looking for healthy adult volunteers. In Part B, we're looking for adults who have a confirmed diagnosis of Hereditary Hemorrhagic Telangiectasia (HHT).
There are also some health conditions that would prevent you from taking part. For healthy volunteers, this includes having certain liver or kidney issues, or known HIV, hepatitis C, or hepatitis B infections. For people with HHT, it includes more significant liver or kidney problems. Everyone involved must also be willing to follow strict contraception guidelines if they are able to get pregnant or get someone pregnant. Your study doctor will discuss all these details with you thoroughly.
¿Podría este estudio ser adecuado para ti?
Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.
- ¿Tienes 18 años o más?
- If you have HHT, has a doctor officially diagnosed you with it?
- Are you generally healthy, or if you have HHT, are your liver and kidney functions within certain limits?
- Are you willing to discuss and follow strict contraception rules if needed?
¿En qué consiste la participación?
Taking part in this study would involve regular visits to the clinic for medical check-ups and to receive the study medication, which might be ALN-6400 or a placebo (a dummy treatment with no active medicine). You'll have blood tests, physical examinations, and other assessments to monitor your health and see how your body responds to the treatment. The total number of visits and the overall length of your participation will depend on which part of the study you are in, and whether you are a healthy volunteer or a person with HHT. The study team will explain the full schedule and what each visit involves before you decide to take part.
Riesgos y beneficios potenciales
Locations (16)
- Clinical Trial SiteCódigo postal verificadoBirmingham, United States· Reclutamiento
- Clinical Trial SiteCódigo postal verificadoCypress, United States· Reclutamiento
- Clinical Trial SiteCódigo postal verificadoGainesville, Estados Unidos· Reclutamiento
- Clinical Trial SiteCódigo postal verificadoIndianápolis, Estados Unidos· Reclutamiento
- Clinical Trial SiteCódigo postal verificadoBoston, Estados Unidos· Reclutamiento
- Clinical Trial SiteCódigo postal verificadoRochester, United States· Reclutamiento
- Clinical Trial SiteCódigo postal verificadoSan Luis, Estados Unidos· Reclutamiento
- Clinical Trial SiteCódigo postal verificadoChapel Hill, United States· Reclutamiento
- Clinical Trial SiteCódigo postal verificadoCamperdown, Australia· Reclutamiento
- Clinical Trial SiteCódigo postal verificadoParkville, Australia· Reclutamiento
- Clinical Trial SiteCódigo postal verificadoMount Royal, Canada· Reclutamiento
- Clinical Trial SiteCódigo postal verificadoToronto, Canadá· Reclutamiento
Preguntas comunes
What is HHT?
HHT, or Hereditary Hemorrhagic Telangiectasia, is a genetic condition that causes blood vessels to develop abnormally, often leading to bleeding, especially nosebleeds.
What is ALN-6400?
ALN-6400 is a new medication being tested in this study to see if it can be a safe and effective treatment for HHT.
¿Qué es un placebo?
A placebo is a 'dummy' treatment that looks like the real medication but doesn't contain any active drug. It helps researchers compare the effects of the active medication.
¿Quién puede participar en este estudio?
Adults aged 18 and over, either healthy volunteers or those diagnosed with HHT, might be able to participate, depending on specific health checks.
Will I get the actual medicine or a placebo?
Some participants will receive ALN-6400 and others will receive a placebo. The study team will explain how this is decided.
Cómo obtener más información
Alnylam Clinical Trial Information Line
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
¿Interesado en participar?
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Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.