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Enrolling by Invitation

NCT Number: NCT04360265

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

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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Key information

Conditions

Mucopolysaccharidosis IIIA Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases MPS IIIA Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis III Nutritional and Metabolic Diseases Sanfilippo A Sanfilippo Syndrome Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Women's and Children's Hospital, North Adelaide, South Australia, Australia

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Primary outcomes

  1. Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs)

    Time frame: Up to Year 5

  2. Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive Raw Score Over Time

    Time frame: Up to Year 5

Secondary outcomes

  1. Cerebrospinal Fluid (CSF) Heparin Sulfate (HS) (Disaccharide) Exposure

    Time frame: Baseline, Up to Month 36

  2. Percent Change From Baseline in Prior Trial in CSF HS

    Time frame: Baseline, Up to Month 36

  3. BSITD-III Receptive Communication Raw Score Over Time

    Time frame: Up to Year 5

  4. BSITD-III Expressive Communication Raw Score Over Time

    Time frame: Up to Year 5

  5. Annualized Percentage Change from Baseline in Prior Trial in Total Cortical Volume

    Time frame: Baseline, Up to Month 36

  6. Survival

    Time frame: Up to Year 5

Sponsors and collaborators

Lead sponsor

Ultragenyx Pharmaceutical Inc

Industry

Registry information

Official study 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)

Important dates

Study start
2020
Primary completion
2027
Study completion
2027
First posted
Apr 24, 2020
Registry last updated
Jul 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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