Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome (NCT07136454) | Clinical Trial Compass
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Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome
Plain-language summary
For a patient to be considered for this EAP, they must have consented to participate in the TANGELO Optional Open-label Extension (OOE), and continue to meet the inclusion/ exclusion criteria and have not received any other investigational drug for the treatment of AS between the last dose of the TANGELO OOE and the first dose in the EAP.
The Physician making the request for expanded access considers continued treatment via the EAP with rugonersen to be a suitable treatment option for the patient under consideration.
Who can participate
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See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
For a patient to be considered for this EAP, they must have consented to participate in the TANGELO Optional Open-label Extension (OOE) and, haven't deviated from any of the inclusion/ exclusion criteria and have not received any other investigational drug for the treatment of AS between the last dose of the TANGELO OOE and the first dose in the EAP.
The Physician making the request for expanded access considers continued treatment via the EAP with rugonersen to be a suitable treatment option for the patient under consideration.
Inclusion Criteria:
* Signed informed consent must be obtained prior to the initiation of any study-required procedures. The participant has a parent, caregiver, or legal representative (referred to as "caregiver") who is reliable and able to consent for the participant according to ICH and local regulations
* At least 18 years of age
* Willing and able to accompany the participant to clinic visits and be available to the site by phone, email, or other electronic form as needed
* Is and will likely remain sufficiently knowledgeable of the participant's condition in order to respond to queries as requested.
* Patient had previously signed informed consent for the TANGELO Optional OOE and completed a final safety follow-up visit for the TANGELO Study.
* Participant is able to comply with all requirements including blood draws.
* Participant is able to undergo lumbar puncture (LP) and intrathecal (IT) injection under sedation or anesthesia as d…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.