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

NCT04360265 — Phase 3, Enrolling by invitation, 41 participants, Ultragenyx Pharmaceutical Inc

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A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)

Registry	NCT04360265
Status	Enrolling by invitation
Phase	Phase 3
Study type	Interventional; non randomized, parallel, masking: none, other
Enrollment	41 (Estimated)
Sponsor	Ultragenyx Pharmaceutical Inc (Industry)
Conditions	Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
Interventions	No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy (Other, drug)
Dates	start 2020-09-28, primary completion 2027-08-01 (estimated), completion 2027-08-01 (estimated), first posted 2020-04-24, last updated 2026-07-17
Locations	4 sites in United States, Australia, Spain

-Summary

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

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.

+Arms and interventions

Arm	Type	Interventions	Description
Cohort A	Other	Other: No Investigational Product; Drug: Adjuvant Immunomodulatory (IM) Therapy	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	Other: No Investigational Product	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.

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.

+Outcome measures

Primary outcomes

Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs) [Up to Year 5]
Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive Raw Score Over Time [Up to Year 5]

Secondary outcomes

Cerebrospinal Fluid (CSF) Heparin Sulfate (HS) (Disaccharide) Exposure [Baseline, Up to Month 36]
Percent Change From Baseline in Prior Trial in CSF HS [Baseline, Up to Month 36]
BSITD-III Receptive Communication Raw Score Over Time [Up to Year 5]
BSITD-III Expressive Communication Raw Score Over Time [Up to Year 5]
Annualized Percentage Change from Baseline in Prior Trial in Total Cortical Volume [Baseline, Up to Month 36]
Survival [Up to Year 5]

+Eligibility

Sex: All

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

+Locations (4)

Facility	City	Country
Nationwide Children's Hospital	Columbus, Ohio	United States
Women's and Children's Hospital	North Adelaide, South Australia	Australia
Vall d'Hebron Barcelona Campus	Barcelona	Spain
Hospital Clínico Universitario de Santiago	Santiago de Compostela	Spain

+Investigators

Medical Director, Study director, Ultragenyx Pharmaceutical Inc

+Funding

NIH grants: 2023, 2019