A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia
This research study, called a clinical trial, is looking at a new medicine named HMB-001 for people with a rare bleeding condition called Glanzmann Thrombasthenia. The main goals are to check if HMB-001 is safe to use and how well people tolerate it. Researchers also want to understand how the medicine works in the body and if it can help prevent or reduce the severity of bleeding episodes. The study is divided into parts, starting with small groups to find the best dose, then expanding to see its effects over longer periods.
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¿De qué trata este estudio?
This study is exploring a new medicine, HMB-001, for individuals living with Glanzmann Thrombasthenia. This is a rare, inherited bleeding disorder where platelets, which are small blood cells crucial for clotting, don't work properly. Because of this, people with Glanzmann Thrombasthenia can experience excessive bleeding, even from minor injuries or surgeries.
The main aim of this research is to thoroughly investigate HMB-001. Firstly, it focuses on safety – making sure the medicine doesn't cause harmful side effects, and how well people can tolerate taking it. Secondly, it looks at how the body handles the medicine (how it's absorbed, distributed, and removed), and how it affects the body on a cellular level. Most importantly, the study wants to find out if HMB-001 can help prevent bleeding episodes or make them less severe for those with Glanzmann Thrombasthenia.
This is a 'Phase 1/2' study. This means it's one of the first times this medicine is being tested in humans, initially focusing heavily on safety and finding the right dose (Phase 1), and then moving on to look more closely at its effectiveness (Phase 2). By taking part, you could contribute valuable information that helps medical science understand and potentially improve treatments for Glanzmann Thrombasthenia.
Puntos clave
- This study is testing a new medicine (HMB-001) for Glanzmann Thrombasthenia.
- The main goals are to check safety, tolerability, and if it reduces bleeding.
- It's an early-stage study (Phase 1/2) that carefully checks doses and effects.
- Participation involves different stages, from single doses to longer-term treatment.
- You must have a confirmed diagnosis of Glanzmann Thrombasthenia and meet other specific health criteria.
- Puede retirarse del estudio en cualquier momento.
¿Quién puede ser elegible?
To join this study, you need to be between 18 and 65 years old. You must have a confirmed diagnosis of Glanzmann Thrombasthenia, either through specific blood tests that look at how your platelets work and what's on their surface, or through a genetic test.
There are also some health checks to make sure it's safe for you to participate. For example, your heart rate and blood pressure need to be within healthy ranges. Your kidneys and liver must be working well, and your haemoglobin (a measure of red blood cells) should be above a certain level. You also need to be able to understand the study information and agree to follow all the study procedures.
If you are a woman who could become pregnant, you'll need a negative pregnancy test and must agree to use highly effective birth control throughout the study and for six months afterwards. Men who could father children will also need to use effective birth control during the study and for six months after their last dose.
¿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 entre 18 y 65 años?
- Do you have a confirmed diagnosis of Glanzmann Thrombasthenia?
- Are your heart rate and blood pressure generally healthy?
- Are your kidney and liver functions within certain healthy limits?
- Are you able to understand and follow study instructions?
- If applicable, are you willing to use effective birth control during and after the study?
¿En qué consiste la participación?
If you decide to take part, the study is divided into several parts. Part A involves taking a single dose of HMB-001, with different small groups receiving different doses. Doctors will carefully monitor your health before increasing the dose for the next group. This part could last about 6 months, including initial checks, a possible observation period, and an 8-week follow-up after your dose.
Part B is similar, testing various doses but you'll receive HMB-001 multiple times over a 3-month period – either weekly, every two weeks, or every four weeks. This involves an initial check, an observation period, the 3-month treatment, and a safety check after your last dose.
If you complete Part B, you might be able to continue into Part C. This would involve taking HMB-001 for a longer period, approximately 18 months, followed by another safety check. Throughout all parts, there will be regular visits and assessments to monitor your health, how the medicine is affecting you, and if it's helping with your bleeding.
Riesgos y beneficios potenciales
Locations (17)
- University of California, San Diego (UCSD) (Part B/C)Código postal verificadoLa Jolla, Estados Unidos
- Tulane University Medical Center (Part B/C)Código postal verificadoNew Orleans, United States
- Mayo Clinic - Rochester (Part B/C)Código postal verificadoRochester, United States
- Hemophilia Center of Western Pennsylvania (HCWP) (Part B/C)Código postal verificadoPittsburgh, Estados Unidos
- Washington Institute for Coagulation (Part B/C)Código postal verificadoSeattle, United States
- University Hospital Leuven - Campus Gasthuisberg (Part B/C)Código postal verificadoLovaina, Bélgica
- AP-HP Hopital Bicetre (Part B/C)Código postal verificadoLe Kremlin-Bicêtre, France
- AP-HM - Hopital de la TimoneCódigo postal verificadoMarsella, Francia
- AP-HP Hopital Necker-Enfants Malades (Part B/C)Código postal verificadoParís, Francia
- Azienda Ospedaliero-Universitaria Careggi (Part B/C)Código postal verificadoFlorence, Italy
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano (Part B/C)Código postal verificadoMilan, Italy
- Universitair Medisch Centrum Utrecht (Part B/C)Código postal verificadoUtrecht, Netherlands
Preguntas comunes
What is Glanzmann Thrombasthenia?
It's a rare inherited bleeding disorder where your platelets, which help stop bleeding, don't work correctly.
What is HMB-001?
HMB-001 is a new medicine being tested in this study to see if it can help people with Glanzmann Thrombasthenia.
What is a 'Phase 1/2' study?
It's an early stage of research. Phase 1 focuses on safety and finding the right dose, while Phase 2 starts to look at how well the medicine works.
¿Cuánto tiempo estaré en el estudio?
Participation can range from about 6 months in Part A, to 3 months for treatment in Part B, and potentially up to 18 months in Part C, plus follow-up times.
¿Puedo abandonar el estudio si cambio de opinión?
Yes, you are free to withdraw from the study at any time without having to give a reason, and it won't affect your medical care.
Cómo obtener más información
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
Discusión
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Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.