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Study on the Safety and Tolerability of Rugonersen in Patients with Angelman Syndrome

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Angelman Syndrome, a rare genetic disorder that affects the nervous system and causes developmental disabilities and neurological problems. The study will use a treatment called Rugonersen, which is administered as a solution for injection. The purpose of the study is to investigate the safety and tolerability of Rugonersen in individuals with Angelman Syndrome.

Participants in the study will receive the treatment through a method called intratracheal use, which involves delivering the medication directly into the windpipe. The study will monitor participants over a period of time to observe any side effects or changes in their health. This will help researchers understand how the body processes the medication and how it affects the participants' health and well-being.

The study will also collect information on the frequency and severity of any adverse events, such as unexpected health issues, and will track changes in vital signs like blood pressure and heart rate. By gathering this data, researchers aim to ensure the treatment is safe and to learn more about its potential benefits for people with Angelman Syndrome.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit

    Upon joining the study, you will attend an initial visit at the clinic. During this visit, a healthcare professional will review your medical history and confirm your diagnosis of Angelman Syndrome.

    You will be asked to provide a blood sample to ensure you can tolerate blood draws. This is important for monitoring your health throughout the study.

  2. Step 2

    Treatment administration

    You will receive the study medication, Rugonersen, which is a solution for injection. The medication will be administered through a procedure called intratracheal use, meaning it will be delivered directly into your windpipe.

    The frequency and dosage of the medication will be determined by the study team based on your specific needs and the study protocol.

  3. Step 3

    Regular monitoring

    Throughout the study, you will have regular clinic visits to monitor your health and the effects of the medication. These visits will include checking your vital signs, such as temperature, blood pressure, heart rate, and respiratory rate.

    Blood and cerebrospinal fluid samples may be taken to assess any changes in your body and to ensure your safety.

  4. Step 4

    Safety assessments

    The study team will closely monitor for any adverse events, which are any unwanted effects you might experience during the study. This includes both minor and serious events.

    If any adverse events occur, the study team will determine if it is necessary to adjust your treatment or discontinue it for your safety.

  5. Step 5

    End of study

    The study is expected to conclude by July 31, 2025. At the end of the study, you will have a final visit to assess your overall health and any changes that have occurred during the study period.

    The study team will discuss the results with you and provide any necessary follow-up care or recommendations.

Who can join the trial?

5 criteria

  • The participant must have a parent, caregiver, or legal representative who is reliable, competent, and at least 18 years old. This person should be willing and able to accompany the participant to clinic visits and be available by phone or email if needed. They should also be knowledgeable about the participant's condition to answer any questions from the study team.
  • The participant must have a clinical diagnosis of Angelman Syndrome (AS), confirmed by a molecular diagnosis. This includes a specific genetic change called a UBE3A mutation on the maternal allele or a deletion on the maternally inherited chromosome 15q11q13 that includes the UBE3A gene and is less than 7 megabases (Mb) in size.
  • The participant should have a stable medical status for at least 4 weeks before the screening and at the time of enrollment in the study.
  • The participant must have adequate supportive psychosocial circumstances, meaning they have a supportive environment and resources to help them participate in the study.
  • The participant must be able to tolerate blood draws, which means they can handle having blood taken for tests.

Who cannot join the trial?

8 criteria

  • Patients who do not have Angelman Syndrome (AS) cannot participate.
  • Patients who are not within the specified age range cannot participate.
  • Patients who are not willing to follow the study procedures cannot participate.
  • Patients who have other medical conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial recently cannot participate.
  • Patients who have a history of allergic reactions to similar medications cannot participate.
  • Patients who are unable to provide informed consent cannot participate.
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Investigated drugs

RO7248824 is a medication being studied for its potential use in treating Angelman Syndrome, a genetic disorder that affects the nervous system. This medication is being tested to see how safe it is for patients and how well it is tolerated by the body. Researchers are also looking at how the medication moves through the body and how it affects the body’s functions. The goal is to understand if this medication can help manage symptoms of Angelman Syndrome and improve the quality of life for those affected by the condition.

What is already known about the treatment

RO7248824 – RO7248824 is administered as an injection and is currently being studied in clinical trials for its safety and tolerability in treating Angelman Syndrome, a rare genetic disorder. This medication is still under investigation, and its full effects and potential uses are being explored in medical research. It is designed to help manage symptoms associated with Angelman Syndrome, although its exact mechanism of action at the molecular level is still being studied. RO7248824 falls under the category of investigational drugs, meaning it is not yet approved for general medical use.

Investigated diseases

Angelman Syndrome – Angelman Syndrome is a genetic disorder that primarily affects the nervous system. It is characterized by developmental delays, intellectual disability, severe speech impairment, and problems with movement and balance. Individuals with this condition often exhibit a happy demeanor, frequent smiling, and laughter. The syndrome is caused by the loss of function of a gene on chromosome 15. As the condition progresses, individuals may experience seizures and sleep disturbances. Motor skills such as walking and coordination are typically affected, leading to a distinct gait.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase ITrial ID2024-514797-45-00Protocol codeBP41674Estimated enrolment53 patientsSponsorF. Hoffmann-La Roche AG

sourced from the EU Clinical Trials Register and site verification

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

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