MPS IIIA, Mucopolysaccharidosis IIIA, Sanfilippo A, Sanfilippo Syndrome
Conditions
Keywords
MPS IIIA, Sanfilippo, Gene Therapy
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).
Detailed description
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.
Interventions
No investigational product will be administered in this follow-up trial.
DRUGAdjuvant 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.
Sponsors
Ultragenyx Pharmaceutical Inc
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 |
| Survival | Up to Year 5 |
Countries
Australia, Spain, United States
Contacts
STUDY_DIRECTORMedical Director
Ultragenyx Pharmaceutical Inc
Outcome results
None listed