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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE3

Total Enrollment

41 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-09-28

Study Completion Date

2027-08-31

Brief Summary

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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).

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Detailed Description

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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.

Conditions

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Mucopolysaccharidosis IIIA MPS IIIA Sanfilippo Syndrome Sanfilippo A

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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Cohort A

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.

Group Type OTHER

No Investigational Product

Intervention Type OTHER

No investigational product will be administered in this follow-up trial.

Adjuvant Immunomodulatory (IM) Therapy

Intervention Type 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.

Cohort B

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.

Group Type OTHER

No Investigational Product

Intervention Type OTHER

No investigational product will be administered in this follow-up trial.

Interventions

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No Investigational Product

No investigational product will be administered in this follow-up trial.

Intervention Type OTHER

Adjuvant Immunomodulatory (IM) Therapy

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.

Intervention Type DRUG

Eligibility Criteria

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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

Eligible Sex

ALL

Accepts Healthy Volunteers

No