Clinical trial NCT04360265: Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
Official title: 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)
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Mucopolysaccharidosis IIIA
MPS IIIA
Sanfilippo Syndrome
Sanfilippo A
Keywords: MPS IIIA
Sanfilippo
Gene Therapy
Lead sponsor: Ultragenyx Pharmaceutical Inc
Sponsor class: INDUSTRY
First posted: 2020-04-24
Enrollment: 41
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

Brief 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).

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs)
timeFrame: Up to Year 5
measure: Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive Raw Score Over Time
timeFrame: Up to Year 5
Secondary outcomes:
measure: Cerebrospinal Fluid (CSF) Heparin Sulfate (HS) (Disaccharide) Exposure
timeFrame: Baseline, Up to Month 36
measure: Percent Change From Baseline in Prior Trial in CSF HS
timeFrame: Baseline, Up to Month 36
measure: BSITD-III Receptive Communication Raw Score Over Time
timeFrame: Up to Year 5
measure: BSITD-III Expressive Communication Raw Score Over Time
timeFrame: Up to Year 5
measure: Annualized Percentage Change from Baseline in Prior Trial in Total Cortical Volume
timeFrame: Baseline, Up to Month 36
measure: Survival
timeFrame: Up to Year 5
Other outcomes:
Locations:
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: North Adelaide
country: Australia
facility: Women's and Children's Hospital
state: South Australia
city: Barcelona
country: Spain
facility: Vall d'Hebron Barcelona Campus
city: Santiago De Compostela
country: Spain
facility: Hospital Clínico Universitario de Santiago
Study officials:
affiliation: Ultragenyx Pharmaceutical Inc
name: Medical Director
role: STUDY_DIRECTOR
References: