Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome
ConditionAngelman Syndrome
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
AgeNot listed
SponsorOak Hill Bio Ltd
About this trial
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.
Eligibility criteria
Qualifiers
None
Disqualifiers
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.
Trial design
Treatments tested in this trial
- Rugonersen
Treatment groups
No treatment groups listed
Locations
Sponsors and collaborators
Oak Hill Bio Ltd
Lead sponsor
Early Access Care LLC
Collaborator
Source ClinicalTrials.gov