study details
start date: September 28, 2020
estimated completion: December 31, 2026
last updated: January 24, 2022
size / enrollment: 50
study description: This is a multicenter, noninterventional, long-term follow-up study of patients with MPS IIIA who have completed a prior clinical trial involving the administration of ABO-102. This study is designed to provide LTFU in accordance with the FDA and 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-102. Participants will have up to 5 scheduled visits with assessments as specified in the schedule of assessments.
primary outcomes:
- Adverse Events
Long-term product safety as defined by the incidence, type, and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
- 60 Months
secondary outcomes:
- Age Equivalent Compared to Natural History Study Data
All study visits (30, 36, 42, 48, and 60 Months)
- Developmental Quotient Compared to Natural History Study Data
All study visits (30, 36, 42, 48, and 60 Months)
- Cognitive Age Equivalent Compared to Natural History Study Data
All study visits (30, 36, 42, 48, and 60 Months)
- Developmental Quotient Compared to Natural History Study Data
All study visits (30, 36, 42, 48, and 60 Months)
- Adaptive Age Equivalent Compared to Natural History Study Data
All study visits (30, 36, 42, 48, and 60 Months)
- Pediatric Quality of Life Inventory (PedsQL™)
All study visits (30, 36, 42, 48, and 60 Months)
- Pediatric Quality of Life Inventory Gastrointestinal Symptom
All study visits (30, 36, 42, 48, and 60 Months)
- Parenting Stress Index, 4th Edition (PSI-4)
All study visits (30, 36, 42, 48, and 60 Months)
- Children's Sleep Habits Questionnaire (CSHQ)
All study visits (30, 36, 42, 48, and 60 Months)
- Parental Global Impression Scale
All study visits (30, 36, 42, 48, and 60 Months)
- Clinical Global Impression Improvement Scale
All study visits (30, 36, 42, 48, and 60 Months)
- Parent Symptom Scale Questionnaire
All study visits (30, 36, 42, 48, and 60 Months)
- T-cell responses against the AAV9 capsid
60 Months
- T-cell responses against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product
60 Months
- Antibody formation (humoral) against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product.
60 Months
- Antibody formation (humoral) against the AAV9 capsid
60 Months
- Viral Load
All study visits (30, 36, 42, 48, and 60 Months)
- Sanfilippo Behavior Rating Scale
All study visits (30, 36, 42, 48, and 60 Months)
- Neutralizing antibody against the AAV9 capsid
60 Months
inclusion criteria:
• Eligible Ages: 0 - 120
• Eligible Sexes: all
Inclusion Criteria:
Participants that have completed a prior clinical trial involving the administration of ABO-102.
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: Criteria:
Planned or current participation in another clinical trial that may confound the safety and efficacy evaluation of ABO-102 during the duration of this study.
Any other situation that precludes the participant from undergoing procedures required in this study.
