Angelman Syndrome
Conditions
Brief 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.
Detailed description
The goal of this EAP is to make rugonersen available to patients who have previously consented to participate in the TANGELO Optional Open-Label Extension (OOE). Eligibility reassessment is required for patients who are entering the EAP after they have completed a final visit in the TANGELO study. The purpose of the reassessment is to ensure that the patient continues to meet the eligibility criteria. The relevant assessments to confirm eligibility (i.e., coagulation, platelets, renal function) are part of pre-dosing assessment and can be conducted prior to EAP dosing either on the day before dosing or on the dosing day. An updated medical history for participants should be completed prior to entering the EAP. For the EAP the dose regimen will be 120 mg administered every 16 weeks.
Interventions
DRUGRugonersen
Rugonersen is a selective synthetic 20-mer antisense oligonucleotide (ASO).
Sponsors
Early Access Care LLC
Oak Hill Bio Ltd
Eligibility
Sex/Gender
ALL
Inclusion criteria
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 deemed appropriate. * Will comply with requirements regarding contraception and is confirmed by caregiver consent. * In the opinion and clinical judgement of the Physician, potential benefit outweighs potential risk of continuing rugonersen as an investigational therapy, based on the individual patient's medical history and program eligibility criteria, and judged by the treating physician to still being medically suitable for treatment with rugonersen.
Contacts
Primary ContactAnnette Silvia
Backup ContactSharon Morriss
Outcome results
None listed