This is a multicenter, non-interventional, long-term follow-up (LTFU) study in participants who have been treated with ABO-101 in a prior trial. Eligible participants will undergo clinical evaluations at prespecified intervals for 3 years from the last visit in the prior clinical trial (up to 5 years post-treatment).
This is a multicenter, noninterventional, long-term follow-up study of patients with (Mucopolysaccharidosis IIIB) MPS IIIB who have completed a prior clinical trial involving the administration of ABO-101. This study is designed to provide LTFU in accordance with the FDA and European Medicines Agency (EMA) guidelines for patients treated with gene therapy products. The duration of the current study is 3 years for a total of up to 5 years post-treatment for these participants who rollover from a prior clinical trial of ABO-101. Participants will have a maximum of 5 annual scheduled visits with assessments as specified in the schedule of assessments.
Study Type
OBSERVATIONAL
Enrollment
1
Gene therapy rAAV9.CMV.hNAGLU
Nationwide Children's Hospital
Columbus, Ohio, United States
Armand-Trousseau Hospital
Paris, France
University Hospital Hamburg-Eppendorf
Hamburg, Germany
Adverse Events
Long-term product safety as defined by the incidence, type, and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: 60 months
Age Equivalent Compared to Natural History Study Data
Change from baseline in the Age Equivalent after treatment compared to Natural History Study data calculated by the Mullen Scales of Early Learning or the Kaufman Assessment Battery far Children; Second Edition, based on chronological and developmental age
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Developmental Quotient Compared to Natural History Study Data calculated by the Mullen Scales of Early Learning or the Kaufman Assessment Battery for Children
Change from baseline in the Developmental Quotient (DQ) after treatment compared to Natural History Study data calculated by the Mullen Scales of Early Learning or the Kaufman Assessment Battery for Children; Second Edition, based on chronological and developmental age
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Cognitive Age Equivalent Compared to Natural History Study Data
Change from baseline in the Cognitive Age Equivalent after treatment compared to Natural History Study, calculated using the Bayley Scales of lnfant and Toddler Development - Third edition or the Kaufman Assessment Battery far Children. Second Edition, based on developmental age
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Developmental Quotient Compared to Natural History Study Data calculated using the Bayley Scales of lnfant and Toddler Development or the Kaufman Assessment Battery for Children
Change from baseline in the Developmental Quotient after treatment compared to Natural History Study, calculated using the Bayley Scales of lnfant and Toddler Development - Third edition or the Kaufman Assessment Battery far Children. Second Edition, based on developmental age
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Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Adaptive Age Equivalent Compared to Natural History Study Data
Change from baseline in the Adaptive Age Equivalent score after treatment compared to Natural History Study data, as assessed by parent report using the Vineland Adaptive Behavior Scale II Survey farm
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Pediatric Quality of Life lnventory (PedsQL ™)
Quality of life based on Pediatric Quality of Life lnventory (PedsQL ™) applicable for participants who completed these evaluations in the prior clinical trial.
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Parenting Stress lndex, 4th Edition (PSl-4)
Quality of life based on Parenting Stress lndex, 4th Edition (PSl-4).
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Parental Global lmpression Scale
Evolution on Parental Global lmpression Score, applicable for patients who completed these evaluations in the prior clinical trial.
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Clinical Global lmpression lmprovement Scale
Evolution of Clinical Global lmpression lmprovement Score applicable for patients who completed these evaluations in the prior clinical trial.
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
Parent Symptom Scale Questionnaire
Evolution on Parent Symptom Score Questionnaire applicable for patients who completed these evaluations in the prior clinical trial.
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)
T-cell responses against the AAV9 capsid
Long-term immunological responses defined as T-cell responses against the AAV9 capsid
Time frame: 60 Months
T-cell responses against the α-N- acetylglucosaminidase (NAGLU) transgene product
Long-term immunological responses defined as T-cell responses against the α-N- acetylglucosaminidase transgene product.
Time frame: 60 Months
Antibody formation (humoral) against the α-N- acetylglucosaminidase (NAGLU) transgene product
Long-term immunological responses defined as antibody formation (humoral) against the α-N- acetylglucosaminidase (NAGLU)
Time frame: 60 Months
Antibody formation (humoral) against the AAV9 capsid
Long-term immunological responses defined as antibody formation (humoral) against the AAV9 capsid.
Time frame: 60 Months
Viral Load
Long-term viral load, in applicable cases.
Time frame: AII study visits (30, 36, 42, 48, and 60 Months)