Ongoing, recruitingHuman Pharmacology (Phase I)- OtherInterventional

An Open-Label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7248824 In Participants with Angelman Syndrome

This study is investigating a new medicine called Rugonersen for individuals with Angelman Syndrome. It's an early-stage study, which means we are mainly focused on safety. Researchers want to understand how the medicine behaves in the body, including how much of it gets into the system and how the body breaks it down. They will also be closely monitoring for any unwanted side effects and how well people tolerate the treatment. This research is crucial for understanding if Rugonersen could be a potential future treatment option for Angelman Syndrome, ensuring the health and safety of participants is the top priority.

At a glance

Status

Ongoing, recruiting

Phase

Human Pharmacology (Phase I)- Other

Sponsor

F. Hoffmann-La Roche AG

Enrolment target

Start

10 Oct 2024

Angelman Syndrome (AS)

What is this study about?

This study is about a new medicine called Rugonersen, which is being investigated for people living with Angelman Syndrome. Angelman Syndrome is a rare genetic condition that affects a person's development, causing issues with communication, movement, and sometimes seizures.

The main goal of this particular study is to understand if Rugonersen is safe to use and how well people's bodies handle it. It's like a first step in testing a new medicine. Researchers will be looking very closely at any side effects that might happen. They also want to see how the medicine moves through the body, how much of it gets into the bloodstream, and how long it stays there. This helps them work out the best way to give the medicine in the future.

By carefully checking these things, scientists can learn a lot about Rugonersen. This information is vital for deciding if the medicine should be studied further in more people. Ultimately, the hope is to find new and better ways to help people with Angelman Syndrome.

Key takeaways

This study is testing a new medicine called Rugonersen for Angelman Syndrome.
The main focus is to check if the medicine is safe and how the body handles it.
Participants will receive the study medicine and have regular health checks.
It's an early-stage study, helping us learn for future treatments.
You can withdraw from the study at any time.

Who may be eligible?

This study is open to both men and women of all ages who have Angelman Syndrome. The research team will carefully review each person's health to make sure the study is a good fit for them. They will need to ensure that taking part is safe and that their health condition allows them to participate without extra risks.

Even though the eligibility mentioned 'any to any' age, a doctor or nurse from the study team will speak with you in detail to confirm if you or your loved one can join. They will check specific health information to make sure it's the right choice.

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.

Do you or your loved one have Angelman Syndrome?
Are you able to attend regular study appointments?
Are you prepared for blood tests and other medical checks?
Have you spoken to your doctor about new treatment options?

Answer every question to see your result.

What does participation involve?

If you or your loved one takes part in this study, you will be given the study medicine, Rugonersen. You'll need to attend several visits to a study clinic or hospital. During these visits, the research team will monitor your health very closely. This will include regular blood tests and possibly tests of your spinal fluid (CSF). They will also check your vital signs, like your temperature, blood pressure, heart rate, and breathing, and perform ECGs, which are heart checks. The total length of time you'll be involved in the study will be explained in full by the study team, along with details about follow-up appointments.

Potential risks and benefits

Taking part in any study has potential benefits and risks. For benefits, you would be helping researchers learn more about Angelman Syndrome and a new medicine, which could potentially help others in the future. However, there's no guarantee the medicine will help you directly. Potential risks include experiencing side effects from the medicine, or discomfort from tests like blood draws or spinal fluid collection. You will be closely monitored, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

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

—
Unverified
Netherlands
—
Unverified
Italy
—
Unverified
Spain

Common questions

What is Angelman Syndrome?

Angelman Syndrome is a rare genetic condition that causes developmental problems, affecting speech, movement, and sometimes leading to seizures.

What is Rugonersen?

Rugonersen is the new medicine being tested in this study for people with Angelman Syndrome. We are trying to understand how it works and if it's safe.

What does 'safety and tolerability' mean?

This means the study will closely watch for any side effects or unwanted reactions to the medicine, and how well people can cope with taking it.

Will I or my loved one receive the medicine?

Yes, if you join this study, participants will receive the study medicine, Rugonersen.

Can I stop being part of the study once I've started?

Yes, you can choose to stop participating at any time, for any reason, without it affecting your medical care.