A Phase 2a, multicenter, randomized, double-blind, placebo-controlled study to evaluate safety, tolerability, pharmacodynamic markers, and pharmacokinetics of AP-101 in patients with familial amyotrophic lateral sclerosis (fALS) and sporadic amyotrophic lateral sclerosis (sALS)
This research study is looking into a new treatment called AP-101 for Amyotrophic Lateral Sclerosis (ALS), sometimes called Motor Neurone Disease. We want to find out if AP-101 is safe for people to take and how well their bodies handle it. Researchers will also check how the medicine affects certain markers in the body related to ALS. For the first part of the study, some participants will receive AP-101, while others will get a placebo (a dummy treatment, like salt water). This helps us see the true effects of AP-101. After this initial stage, everyone who continues in the study will receive AP-101. The study is open to both people with inherited (familial) ALS and those with ALS that isn't inherited (sporadic).
De un vistazo
¿De qué trata este estudio?
This study is focused on a health condition called Amyotrophic Lateral Sclerosis, or ALS. You might know it as Motor Neurone Disease. It's a serious illness that affects nerve cells in the brain and spinal cord, making it harder for messages to get from your brain to your muscles. Over time, this can lead to difficulties with movement, speaking, swallowing, and breathing.
The main goal of this research is to test a new medicine, which we're calling AP-101, for people living with ALS. We want to thoroughly check if it's safe to use and how well people's bodies cope with it. Researchers will also be looking closely at how the medicine moves through the body and if it changes certain disease markers found in the blood and spinal fluid. These markers can give us clues about how ALS is progressing or responding to treatment.
The study involves two main parts. In the first part, some participants will get the actual medicine (AP-101), while others will get a placebo. A placebo looks just like the real medicine but contains no active drug, like a salt water solution. This is a common way to test new medicines and helps us clearly see if any changes are due to AP-101 itself. In the second part, called the 'Open-label Extension', everyone who continues in the study will receive AP-101. This allows us to gather more information on the safety and effects of the medicine over a longer period.
Puntos clave
- This study tests a new medicine (AP-101) for ALS.
- It aims to check safety, side effects, and how the body uses AP-101.
- Participation involves regular clinic visits for checks and medicine administration.
- Some participants will initially receive a placebo (dummy treatment).
- Both inherited and non-inherited forms of ALS are included.
- Puede retirarse del estudio en cualquier momento.
¿Quién puede ser elegible?
To be considered for this study, you need to be an adult, 18 years of age or older. There is no upper age limit for joining.
This study is open to both men and women. We are looking for people who have been diagnosed with ALS, whether it runs in their family (familial ALS) or if it appeared without a family history (sporadic ALS).
There will be other health checks and criteria to make sure the study is right for you and safe. These will be discussed in detail by the study team, but generally, you need to be well enough to take part and attend the study visits.
¿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?
- Have you been diagnosed with ALS (Motor Neurone Disease)?
- Are you able to attend regular clinic appointments?
- Are you generally well enough to take part in a study?
- Do you understand that some people will receive a placebo first?
¿En qué consiste la participación?
If you decide to take part in this study, you will have regular visits to the hospital or clinic. These visits will involve various tests to check your health, such as blood tests, possibly collecting a sample of spinal fluid, checking your heart with an ECG (electrocardiogram), and routine physical and neurological examinations. We will also monitor your weight and vital signs like blood pressure and heart rate.
For the first part of the study, you would either receive the AP-101 medicine or a placebo (salt water solution) through an intravenous drip. You wouldn't know which one you are receiving. After this initial phase, if you continue, you would receive the AP-101 medicine. The total duration of your participation, including all visits and follow-up, will be explained by the study team, but typically, these studies can last several months to a year or more.
Riesgos y beneficios potenciales
Ubicaciones (1)
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Preguntas comunes
What is ALS?
ALS (Amyotrophic Lateral Sclerosis) is a serious illness that affects nerve cells, leading to muscle weakness and difficulty with movement over time. It's also known as Motor Neurone Disease.
What is AP-101?
AP-101 is a new medicine being tested in this study to see if it's safe and how it affects people with ALS.
¿Definitivamente recibiré el nuevo medicamento?
For the first part of the study, some people will get AP-101, and others will get a placebo (a dummy medicine). You won't know which one you're getting. Later, everyone will receive AP-101.
¿Qué es un placebo?
A placebo is a 'dummy' treatment, like a salt water solution, that looks exactly like the real medicine but contains no active drug. It helps researchers understand if the new medicine is truly having an effect.
¿Cuál es el propósito de este estudio?
The main purpose is to test the safety of AP-101 and how the body handles it, along with understanding its effects on certain markers related to ALS.
Cómo obtener más información
Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.
¿Interesado en participar?
Discusión
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