MPS IIIA Disease Monitoring Program
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorUltragenyx Pharmaceutical Inc
About this trial
The main objective of this observational study is to evaluate the long-term safety and effectiveness of rebisufligene etisparvovec for 10 years after rebisufligene etisparvovec administration.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Confirmed diagnosis of MPS IIIA
Group 1: Received rebisufligene etisparvovec in a prior interventional clinical trial.
Group 2: Received (or plans to receive) rebisufligene etisparvovec in a postmarketing setting.
Provide informed consent after the nature of the study has been explained, and prior to any research-related procedures. If a minor or an adult with cognitive limitations, willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related procedures.
Disqualifiers
Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results.
Trial population
The DMP population comprises patients with MPS IIIA who received rebisufligene etisparvovec in a clinical trial or have been prescribed (or plan to receive) rebisufligene etisparvovec in a post marketing setting.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
No Intervention
Other interventionNo investigational/study product will be administered in this DMP.
Treatment groups
55 Participants
are divided into 2 treatment groupsGroup A: Group 1: Rebisufligene Etisparvovec in Prior Clinical Study1 intervention
Group B: Group 2: Rebisufligene Etisparvovec in Post-Marketing Setting1 intervention
Trial outcomes
Primary outcomes
1
Number of Participants with Serious Adverse Events (SAEs) Assessed as Related to Rebisufligene Etisparvovec by the Investigator and Targeted Adverse Events (Targeted AEs) Associated with Identified and Potential Risks of UX111 or AAV8 Gene Therapy
Time frame
10 Years2
Bayley Scales of Infant and Toddler Development Cognitive Raw Score Over Time
Time frame
10 YearsLocations
Sponsors and contacts
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Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: