A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102

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)

The main objective of this study is to evaluate the long-term safety and tolerability of ABO-102 in participants with MPS IIIA.

Study Overview

• Status: Enrolling by invitation
• Conditions: Mucopolysaccharidosis III-A
• Intervention / Treatment: Other: No Investigational Product; Drug: Adjuvant Immunosuppression (IS) Therapy

Detailed Description

This is a multicenter, long-term follow-up study of patients with MPS IIIA who have completed a prior clinical trial involving the administration of ABO-102 (NCT02716246 and NCT04088734). No investigational product will be provided. At approved sites immunosuppression (IS) 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 IS therapy. Not all participants may receive IS therapy.

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

• Study Type: Interventional
• Enrollment (Estimated): 33
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • South Australia
    • North Adelaide, South Australia, Australia
      • Women's and Children's Hospital
• Spain
  • Santiago De Compostela, Spain
    • Hospital Clinico Universitario de Santiago
• United States
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Study Population

This long-term follow-up study will enroll up to 50 patients with MPS IIIA from prior ABO-102 clinical trials. Participants from the previous trials are of any racial, ethnic, or gender background, and could be in the early, middle or advanced phase of the disease.

Description

Inclusion Criteria:

• Participants that have completed a prior clinical trial involving the administration of ABO-102.
• 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 and efficacy evaluation of ABO-102 during this study.
• Any other situation or medical condition that precludes the participant from undergoing procedures required in this study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Cohort 1; Participants who have completed a prior clinical trial involving the administration of ABO-102 and are able to comply with onsite scheduled visits and assessments. Select participants may receive adjuvant IS therapy.	Other: No Investigational Product; No investigational product will be administered in this follow-up trial.; Drug: Adjuvant Immunosuppression (IS) Therapy; The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS therapy.
Other: Cohort 2; Participants who have completed a prior clinical trial involving the administration of ABO-102 and who cannot participate in Cohort 1. Participants will partake in a reduced number of assessments, performed either onsite or at home via a combination of telehealth and home healthcare visits.	Other: No Investigational Product; No investigational product will be administered in this follow-up trial.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Events	Long-term Product Safety as Defined by the Incidence, Type, and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).	Up to Year 5

Secondary Outcome Measures

Outcome Measure	Time Frame
Change From Baseline (in Prior Trial) in Cerebrospinal Fluid Heparan Sulfate (CSF HS)	Up to Month 12
Change From Baseline (in Prior Trial) in the Cognitive Subtest of the Bayley Scales of Infant and Toddler Development -Third Edition (BSID-III) or the Kaufman Assessment Battery for Children - Second Edition (KABC-II), Based on Developmental Age	Baseline, Up to Year 5
Change From Baseline (in Prior Trial) in the Language Subtest of the Bayley Scales of Infant and Toddler Development -Third Edition (BSID-III) or the Kaufman Assessment Battery for Children - Second Edition (KABC-II), Based on Developmental Age	Baseline, Up to Year 5
Change From Baseline (in Prior Trial), as Assessed by the Parent Report Using the Vineland Adaptive Behavior Scale II - Survey Interview Form (VABS-II)	Baseline, Up to Year 5

Collaborators and Investigators

• Sponsor: Ultragenyx Pharmaceutical Inc
• Collaborators: Abeona Therapeutics, Inc
• Investigators: Study Director: Medical Director, Ultragenyx Pharmaceutical Inc

Study record dates

Study Major Dates

• Study Start (Actual): September 28, 2020
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027

Study Registration Dates

• First Submitted: April 20, 2020
• First Submitted That Met QC Criteria: April 21, 2020
• First Posted (Actual): April 24, 2020

Study Record Updates

• Last Update Posted (Actual): February 7, 2024
• Last Update Submitted That Met QC Criteria: February 5, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: MPS IIIA; Mucopolysaccharidosis IIIA; Sanfilippo A
• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mucopolysaccharidoses; Mucopolysaccharidosis III
• Other Study ID Numbers: LTFU-ABO-102; 2019-002979-34 (EudraCT Number); UX111-CL302 (Other Identifier: Ultragenyx Pharmaceutical Inc)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No