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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04360265




Registration number
NCT04360265
Ethics application status
Date submitted
20/04/2020
Date registered
24/04/2020
Date last updated
14/05/2024

Titles & IDs
Public title
A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102
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 III-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 Immunosuppression (IS) Therapy

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: 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 interventions: No Investigational Product
No investigational product will be administered in this follow-up trial.

Treatment: Drugs: Adjuvant Immunosuppression (IS) Therapy
The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS 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
Adverse Events
Timepoint [1] 0 0
Up to Year 5
Secondary outcome [1] 0 0
Change From Baseline (in Prior Trial) in Cerebrospinal Fluid Heparan Sulfate (CSF HS)
Timepoint [1] 0 0
Up to Month 12
Secondary outcome [2] 0 0
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
Timepoint [2] 0 0
Baseline, Up to Year 5
Secondary outcome [3] 0 0
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
Timepoint [3] 0 0
Baseline, Up to Year 5
Secondary outcome [4] 0 0
Change From Baseline (in Prior Trial), as Assessed by the Parent Report Using the Vineland Adaptive Behavior Scale II - Survey Interview Form (VABS-II)
Timepoint [4] 0 0
Baseline, Up to Year 5

Eligibility
Key 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.
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 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 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
1/06/2027
Actual
Sample size
Target
33
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
Santiago De Compostela

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Ultragenyx Pharmaceutical Inc
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Abeona Therapeutics, Inc
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The main objective of this study is to evaluate the long-term safety and tolerability of
ABO-102 in participants with MPS IIIA.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04360265
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04360265