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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04360265
Registration number
NCT04360265
Ethics application status
Date submitted
20/04/2020
Date registered
24/04/2020
Date last updated
13/04/2025
Titles & IDs
Public title
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
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Scientific 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)
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Secondary ID [1]
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2019-002979-34
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Secondary ID [2]
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LTFU-ABO-102
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Mucopolysaccharidosis IIIA
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MPS IIIA
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Sanfilippo Syndrome
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Sanfilippo A
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Condition category
Condition code
Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - No Investigational Product
Treatment: Drugs - Adjuvant Immunomodulatory (IM) Therapy
Other: Cohort A - 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: Cohort B - 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 interventions: No Investigational Product
No investigational product will be administered in this follow-up trial.
Treatment: Drugs: Adjuvant Immunomodulatory (IM) Therapy
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.
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Intervention code [1]
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Other interventions
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Intervention code [2]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs)
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Assessment method [1]
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Timepoint [1]
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Up to Year 5
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Primary outcome [2]
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Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive Raw Score Over Time
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Assessment method [2]
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Timepoint [2]
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Up to Year 5
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Secondary outcome [1]
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Cerebrospinal Fluid (CSF) Heparin Sulfate (HS) (Disaccharide) Exposure
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Assessment method [1]
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Timepoint [1]
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Baseline, Up to Month 36
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Secondary outcome [2]
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Percent Change From Baseline in Prior Trial in CSF HS
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Assessment method [2]
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Timepoint [2]
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Baseline, Up to Month 36
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Secondary outcome [3]
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BSITD-III Receptive Communication Raw Score Over Time
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Assessment method [3]
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Timepoint [3]
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Up to Year 5
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Secondary outcome [4]
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BSITD-III Expressive Communication Raw Score Over Time
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Assessment method [4]
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Timepoint [4]
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Up to Year 5
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Secondary outcome [5]
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Annualized Percentage Change from Baseline in Prior Trial in Total Cortical Volume
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Assessment method [5]
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Timepoint [5]
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Baseline, Up to Month 36
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Secondary outcome [6]
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Survival
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Assessment method [6]
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Timepoint [6]
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Up to Year 5
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Eligibility
Key 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
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Other
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
28/09/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/08/2027
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Actual
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Sample size
Target
41
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
SA
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Recruitment hospital [1]
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Women's and Children's Hospital - North Adelaide
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Recruitment postcode(s) [1]
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- North Adelaide
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Ohio
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Country [2]
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Spain
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State/province [2]
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Barcelona
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Country [3]
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Spain
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State/province [3]
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Santiago De Compostela
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Ultragenyx Pharmaceutical Inc
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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).
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Trial website
https://clinicaltrials.gov/study/NCT04360265
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Medical Director
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Address
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Ultragenyx Pharmaceutical Inc
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT04360265
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