Ongoing, recruitingHuman Pharmacology (Phase I)- First administration to humansInterventional

SAD/MAD study with PTC844

This new study, called the SAD/MAD study with PTC844, is looking into a potential new medicine for autoimmune disorders. Autoimmune disorders happen when your body's defence system mistakenly attacks healthy parts of your own body. This study is the very first time PTC844 is being given to people. Researchers will be carefully checking to see if it's safe and how it behaves in the body. This kind of early study helps us learn if a new medicine has the potential to help people in the future. It's an important step in developing new treatments.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- First administration to humans

Sponsor

PTC Therapeutics Inc.

Enrolment target

Start

23 Dec 2022

Auto immune disorders

What is this study about?

This study is a very early stage trial looking at a new medicine called PTC844. It's designed to see how this potential treatment works in the human body, especially for people who have autoimmune disorders. Autoimmune disorders are conditions where your immune system, which normally fights off infections, starts to attack your own healthy cells and tissues. Examples include conditions like rheumatoid arthritis or lupus, but this study isn't focusing on a specific one yet.

The main goal of this particular study is to understand two key things: how safe the medicine is when given to people, and how the body handles it (for example, how it's absorbed and processed). Because this is the first time humans will receive PTC844, it's called a 'Phase I' or 'Human Pharmacology' study. Researchers will start with very small doses and gradually increase them, all while carefully watching participants for any side effects.

This kind of study is crucial because it helps scientists decide if a new medicine is promising enough to continue testing in larger groups of people. It's a foundational step towards potentially finding new and better ways to treat autoimmune conditions in the future.

Key takeaways

This is a very early study of a new medicine called PTC844.
It's for people with autoimmune disorders.
The main aim is to check the medicine's safety and how it works in the body.
It's the first time this medicine is being given to humans.
Adults aged 18 and over, both men and women, can potentially take part.
Participation could help advance future treatments.

Who may be eligible?

To be considered for this study, you need to be an adult, at least 18 years old. There's no upper age limit specified, so adults of all ages are welcome to inquire.

Both men and women can take part in this study. The researchers want to get a broad understanding of how the medicine works across different people.

Because this is a very early study investigating how a new medicine works in the body, there will be other specific health requirements not mentioned here. These will be carefully checked by the study team to make sure it's safe for you to participate.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Are you comfortable with receiving an experimental new medicine?
Do you have an autoimmune disorder?
Are you able to travel to the study clinic for appointments?
Are you willing to have regular medical checks, including blood tests?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would likely need to visit the study clinic multiple times. During these visits, the study team would give you the new medicine, PTC844, and carefully monitor your health. This might involve having regular blood tests, checks of your heart and other vital signs, and answering questions about how you're feeling. The length of the study and how many visits are needed isn't detailed here but will be fully explained by the study team. You would also have follow-up appointments after you've finished taking the medicine to ensure you remain well.

Potential risks and benefits

Taking part in any study has potential benefits and risks. You might not directly benefit from this early study, as its main purpose is to gather information about the new medicine. However, you would be helping advance medical knowledge and potentially contribute to new treatments for autoimmune disorders in the future. Possible risks could include side effects from the new medicine, which the study team would explain in detail. You are always free to leave the study at any time, for any reason, without it affecting your medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Netherlands

Common questions

What does 'autoimmune disorders' mean?

Autoimmune disorders are conditions where your body's immune system, which normally fights off germs, mistakenly attacks your own healthy body parts instead.

What does 'Phase I' mean for a study?

Phase I means this is one of the very first times a new medicine is being given to people. The main goals are to check if it's safe and how the body handles it.

Will I get better if I join this study?

It's unlikely you'll directly feel better from this early study, as the main goal is to test the medicine's safety and how it works, not primarily to treat your condition yet.

What is PTC844?

PTC844 is the name of the new experimental medicine being tested in this study for autoimmune disorders. It's not yet available as a treatment.

Do I have to pay to be in the study?

No, you would not be required to pay to participate in the study. All study-related care and medicines are typically provided free of charge.