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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

Titles & IDs
Public title
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
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)
Secondary ID [1] 0 0
2019-002979-34
Secondary ID [2] 0 0
LTFU-ABO-102
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mucopolysaccharidosis IIIA 0 0
MPS IIIA 0 0
Sanfilippo Syndrome 0 0
Sanfilippo A 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
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 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 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.
Intervention code [1] 0 0
Other interventions
Intervention code [2] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Timepoint [1] 0 0
Up to Year 5
Primary outcome [2] 0 0
Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive Raw Score Over Time
Timepoint [2] 0 0
Up to Year 5
Secondary outcome [1] 0 0
Cerebrospinal Fluid (CSF) Heparin Sulfate (HS) (Disaccharide) Exposure
Timepoint [1] 0 0
Baseline, Up to Month 36
Secondary outcome [2] 0 0
Percent Change From Baseline in Prior Trial in CSF HS
Timepoint [2] 0 0
Baseline, Up to Month 36
Secondary outcome [3] 0 0
BSITD-III Receptive Communication Raw Score Over Time
Timepoint [3] 0 0
Up to Year 5
Secondary outcome [4] 0 0
BSITD-III Expressive Communication Raw Score Over Time
Timepoint [4] 0 0
Up to Year 5
Secondary outcome [5] 0 0
Annualized Percentage Change from Baseline in Prior Trial in Total Cortical Volume
Timepoint [5] 0 0
Baseline, Up to Month 36
Secondary outcome [6] 0 0
Survival
Timepoint [6] 0 0
Up to Year 5

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
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Other
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/09/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2027
Actual
Sample size
Target
41
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Women's and Children's Hospital - North Adelaide
Recruitment postcode(s) [1] 0 0
- North Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Ohio
Country [2] 0 0
Spain
State/province [2] 0 0
Barcelona
Country [3] 0 0
Spain
State/province [3] 0 0
Santiago De Compostela

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ultragenyx Pharmaceutical Inc
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Ultragenyx Pharmaceutical Inc
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


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