NCT07136454

Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome

• Status: Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Oak Hill Bio Ltd · Industry
• Sex: All
• Age: —
• Healthy volunteers: —

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.

Conditions

• Angelman Syndrome

Interventions

Type	Name	Description
DRUG	Rugonersen	Rugonersen is a selective synthetic 20-mer antisense oligonucleotide (ASO).

Timeline

• First posted: 2025-08-22
• Last updated: 2025-08-22