By InvitationPhase 3

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

United States, Australia, Spain41 participantsStarted 2020-09-28

Plain-language 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).

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs)

Timeframe: Up to Year 5

Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive Raw Score Over Time

Timeframe: Up to Year 5

Trial details

NCT IDNCT04360265

SponsorUltragenyx Pharmaceutical Inc

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-08

View on ClinicalTrials.gov More Mucopolysaccharidosis IIIA trials