A Long-Term Follow-Up Study of Patients With MPS IIIA Treated With ABO-102

study id #: NCT04360265

condition: Mucopolysaccharidosis III-A

status: Recruiting

This is a multicenter, non-interventional, long-term follow-up study in participants from prior interventional trials involving the administration of ABO-102. Eligible participants will have up to 5 scheduled visits with assessments as specified in the schedule of assessments.

intervention: ABO-102

results: https://clinicaltrials.gov/ct2/show/results/NCT04360265

last updated: February 26, 2022

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:

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.

sponsor: Abeona Therapeutics, Inc

contacts: Medical Affairs, +1 646-813-4701, sanfilippo@abeonatherapeutics.com

trial center locations: United States,Australia,Spain

• United States, Ohio
  Nationwide Children’s Hospital
  Kristen Brown, 614-722-6918, kristen.brown@nationwidechildrens.org
  
  
• Australia, South Australia
  Women’s and Children’s Hospital
  Louise Jaensch, +61 08 8161 7295, louise.jaensch@sa.gov.au
  
  
• Spain
  Hospital Clínico Universitario de Santiago
  Maria Luz Couce, MD, PhD, maria.luz.couce.pico@sergas.es