Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04360265 | Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA | PHASE3 | ENROLLING BY_INVITATION | 41 | — | — | Sep 28, 2020 | Aug 1, 2027 | Apr 24, 2026 | 4 | United States, Australia +1 |
| Arm | Type | Description |
|---|---|---|
| Cohort A | OTHER | Participants who have participated in a prior clinical trial involving the administration of UX111 (rebisufligene etisparvovec) and are able to comply with onsite scheduled visits and assessments. Select participants may receive adjuvant IM therapy. |
| Cohort B | OTHER | Participants who have participated in a prior clinical trial involving the administration of UX111 (rebisufligene etisparvovec) and who cannot participate in Cohort A. Participants will partake in a reduced number of assessments, performed either onsite or at home via a combination of telehealth and home healthcare visits. |
| Name | Type | Description |
|---|---|---|
| No Investigational Product | OTHER | No investigational product will be administered in this follow-up trial. |
| Adjuvant Immunomodulatory (IM) Therapy | DRUG | The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive IM therapy. |
Inclusion Criteria: * Participants that have participated in a prior clinical trial in which they received UX111 * Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule Exclusion Criteria: * Planned...