Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Ultragenyx

Status and phase: Invitation-only; Phase 3

Conditions: Sanfilippo A; MPS IIIA; Mucopolysaccharidosis IIIA; Sanfilippo Syndrome

Treatments: Drug: Adjuvant Immunomodulatory (IM) Therapy; Other: No Investigational Product

Study type: Interventional

Funder types: Industry

Identifiers

NCT04360265

LTFU-ABO-102

2023-510392-66-00 (EU Trial (CTIS) Number)

2019-002979-34 (EudraCT Number)

UX111-CL302 (Other Identifier)

Details and patient eligibility

About

The main objective of this study is to evaluate the safety/tolerability and efficacy of UX111 (previously known as ABO-102) in participants with Mucopolysaccharidosis IIIA (MPS IIIA).

Full description

This is a multicenter, long-term follow-up study of patients with MPS IIIA who have participated in a prior clinical trial involving the administration of UX111 (NCT02716246 and NCT04088734). No investigational product will be provided. At approved sites adjuvant immunomodulatory (IM) therapy may be administered to selected participants. 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.

This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.

Enrollment: 41 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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 or current participation in another clinical trial that may confound the safety or efficacy evaluation of UX111 during this study
• Any other situation or medical condition that precludes the participant from undergoing procedures required in this study

Trial design

Primary purpose: Other

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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41 participants in 2 patient groups

Cohort A

Other group

Description:

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.

Treatment:

Other: No Investigational Product

Drug: Adjuvant Immunomodulatory (IM) Therapy

Cohort B

Other group

Description:

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.

Treatment:

Other: No Investigational Product