NCT04360265

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).

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
41

participants targeted

Target at below P25 for phase_3

Timeline
15mo left

Started Sep 2020

Longer than P75 for phase_3

Geographic Reach
3 countries

4 active sites

Status
enrolling by invitation

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress82%
Sep 2020Aug 2027

First Submitted

Initial submission to the registry

April 20, 2020

Completed
4 days until next milestone

First Posted

Study publicly available on registry

April 24, 2020

Completed
5 months until next milestone

Study Start

First participant enrolled

September 28, 2020

Completed
6.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2027

Last Updated

April 24, 2026

Status Verified

April 1, 2026

Enrollment Period

6.8 years

First QC Date

April 20, 2020

Last Update Submit

April 21, 2026

Conditions

Mucopolysaccharidosis IIIAMPS IIIASanfilippo SyndromeSanfilippo A

Keywords

MPS IIIASanfilippoGene Therapy

Outcome Measures

Primary Outcomes (2)

  • 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 (6)

  • 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

  • +1 more secondary outcomes

Study Arms (2)

Cohort A

OTHER

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.

Other: No Investigational ProductDrug: Adjuvant Immunomodulatory (IM) Therapy

Cohort B

OTHER

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.

Other: No Investigational Product

Interventions

No Investigational ProductOTHER

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

Cohort ACohort B
Adjuvant Immunomodulatory (IM) TherapyDRUG

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 A

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Women's and Children's Hospital

North Adelaide, South Australia, Australia

Location

Vall d'Hebron Barcelona Campus

Barcelona, 08035, Spain

Location

Hospital ClĂ­nico Universitario de Santiago

Santiago de Compostela, Spain

Location

Related Links

MeSH Terms

Conditions

Mucopolysaccharidosis III

Interventions

Therapeutics

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Medical Director

    Ultragenyx Pharmaceutical Inc

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
OTHER
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 20, 2020

First Posted

April 24, 2020

Study Start

September 28, 2020

Primary Completion (Estimated)

August 1, 2027

Study Completion (Estimated)

August 1, 2027

Last Updated

April 24, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

Locations

ES(2)AU(1)US(1)