Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
This research study is investigating a new medication, ADX-038, which is being developed for a rare blood disorder called Paroxysmal Nocturnal Hemoglobinuria (PNH). The study is divided into two parts. The first part will involve healthy volunteers to check the safety, how well their body tolerates the medicine, and how it works in their system. The second part will then involve patients with PNH who are already receiving standard treatment but still have anaemia. For these patients, the study will also look at the safety and effects of ADX-038. The main goal is to understand if ADX-038 is safe and how it interacts with the body before it can be studied in more detail.
Em resumo
Sobre o que é este estudo?
This study is about a new potential medicine called ADX-038, which is being developed to help people with a rare and serious blood condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH causes red blood cells to break down too early, leading to symptoms like tiredness (fatigue), breathlessness, and dark urine. While there are existing treatments, some patients still experience problems like anaemia, meaning their body doesn't produce enough healthy red blood cells.
The main purpose of this research is to carefully check if ADX-038 is safe for people to take and how well their bodies tolerate it. Researchers are also interested in understanding how the medicine moves through the body (this is called pharmacokinetics or PK) and what effects it has on the body (this is called pharmacodynamics or PD). This is an early-stage study, meaning it's one of the first times this medicine is being tested in humans.
The study has two main parts. The first part involves healthy volunteers who don't have PNH. This helps researchers understand how the medicine behaves in a healthy person's system before giving it to patients. The second part will involve PNH patients who are already on standard treatment but still suffer from anaemia. This part will check the safety and initial effects of ADX-038 in those who need it most. By gathering this information, scientists can decide if ADX-038 should be studied further as a potential new treatment for PNH.
Pontos principais
- This study is testing a new medicine (ADX-038) for a rare blood condition (PNH).
- It's an early-stage study focused on safety, how well the body tolerates it, and how it works.
- The study involves both healthy volunteers and PNH patients with anaemia.
- Vaccinations are required for safety if you wish to participate.
- Close monitoring will be provided throughout your involvement.
- A participação é voluntária, e você pode se retirar a qualquer momento.
Quem pode ser elegível?
To be considered for this study, you would need to be an adult aged 18 or older. For the first part of the study, we are looking for healthy individuals. This means you should not have any significant ongoing medical conditions.
It's very important that you've recently had, or are willing to have, vaccinations against certain common infections like meningococcal, pneumococcal, and Haemophilus influenzae type B. This is a safety measure because the study medicine might affect your immune system. You would also need to be free from any recent infections and not have a history of frequent infections. We would also check that you are not pregnant or breastfeeding, and that you are not regularly using certain prescription or over-the-counter medications.
For everyone involved, there will be checks to ensure you haven't recently had major surgery, are free from certain allergies, and don't have issues with your kidney function or blood tests. These checks help ensure the study is as safe as possible for all participants.
Este estudo pode ser adequado para você?
Responda a estas perguntas rápidas para ver se você pode ser elegível. Este é apenas um guia — a decisão final é da equipe de pesquisa.
- Você é um adulto com 18 anos ou mais?
- Do you consider yourself generally healthy and free from major ongoing medical conditions (for the healthy volunteer part)?
- Are you up-to-date with meningococcal, pneumococcal, and Haemophilus influenzae type B vaccinations, or willing to get them?
- Are you free from recent infections and do not have a history of frequent infections?
- Você não está grávida ou amamentando?
- Do you understand that you will need to undergo various health checks, including blood tests?
O que envolve a participação?
If you decide to take part in this study, the exact details of your involvement will depend on which part of the study you join. Generally, it will involve several visits to a clinic where medical staff will perform various assessments to monitor your health. These assessments typically include blood tests, checking your vital signs (such as blood pressure and heart rate), and sometimes an electrocardiogram (ECG) to check your heart.
If you are a healthy volunteer, you would receive either the study medicine, ADX-038, or a placebo (a dummy medicine with no active ingredients). This would be given as a single dose. If you are a PNH patient, you would receive ADX-038. All participants will be closely monitored after receiving the medicine. The total duration of your participation, including follow-up, will be explained in full detail by the study team, but typically, early-stage studies like this involve a series of visits over several weeks to ensure all safety data is collected.
Riscos e benefícios potenciais
Localizações (4)
- Nucleus Network BrisbaneCódigo postal verificadoBrisbane, Australia· Concluído
- Peter MacCallum Cancer CentreCódigo postal verificadoMelbourne, Austrália· Concluído
- Royal Melbourne HospitalCódigo postal verificadoParkville, Austrália· Recrutamento
- Richmond Pharmacology LtdCódigo postal verificadoLondres, Reino Unido· Concluído
Perguntas frequentes
What is PNH?
PNH stands for Paroxysmal Nocturnal Hemoglobinuria. It's a rare condition where red blood cells break down too early, leading to symptoms like fatigue and anaemia.
What is ADX-038?
ADX-038 is a new medicine being tested in this study. We don't know much about it yet, but it's being developed to treat PNH.
What does 'placebo' mean?
A placebo is an inactive substance that looks just like the study medicine but contains no active ingredients. It helps researchers compare the effects of the actual medicine.
Will I know if I'm getting ADX-038 or a placebo?
In the first part of the study (with healthy volunteers), neither you nor the study team will know if you're getting ADX-038 or the placebo. This is called 'double-blinded'.
Posso sair do estudo se mudar de ideia?
Yes, absolutely. Your participation is completely voluntary, and you can withdraw from the study at any time without giving a reason, and it won't affect your medical care.
Como saber mais
Stephanie Leyva
Sempre consulte seu médico ou especialista antes de decidir participar de um estudo.
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Desenvolvido pelo nosso fórum em community.patient.info. Por favor, seja respeitoso — isto não é um conselho médico.