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

Part of paid clinical trials in Columbus, Ohio.

Sponsor
Ultragenyx Pharmaceutical Inc
Study ID
NCT04360265
Phase
PHASE3
Status
Enrolling By Invitation

Conditions

  • MPS IIIA
  • Mucopolysaccharidosis IIIA
  • Sanfilippo A
  • Sanfilippo Syndrome

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • No Investigational Product — OTHER
    No investigational product will be administered in this follow-up trial.
  • Adjuvant Immunomodulatory (IM) Therapy — DRUG
    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.

Study Details

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).

Key Dates

Start date
Sep 28, 2020
Status verified
Apr 2026
Primary completion
Aug 31, 2027
Completion
Aug 31, 2027

Study Design

Enrollment
41 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER

Arms

  • 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.

Primary Outcome Measure

Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs) [ Time Frame: Up to Year 5 ]

Locations (1)

FacilityCityStateZIPSite coordinators
Nationwide Children's HospitalColumbusOhio43205-

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By research site
Nationwide Children's Hospital· Columbus, OH

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