A Long-Term Follow-Up Study of Patients With MPS IIIB Treated With ABO-101

study id #: NCT04655911

condition: Mucopolysaccharidosis III-B

status: Recruiting

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).

intervention: ABO-101

results: https://clinicaltrials.gov/ct2/show/results/NCT04655911

last updated: February 26, 2022

start date: October 28, 2020

estimated completion: June 30, 2026

last updated: February 25, 2021

size / enrollment: 24

study description: 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.

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
  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
  AII study visits (30, 36, 42, 48, and 60 Months)
• Cognitive Age Equivalent Compared to Natural History Study Data
  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
  AII study visits (30, 36, 42, 48, and 60 Months)
• Adaptive Age Equivalent Compared to Natural History Study Data
  AII study visits (30, 36, 42, 48, and 60 Months)
• Pediatric Quality of Life lnventory (PedsQL ™)
  AII study visits (30, 36, 42, 48, and 60 Months)
• Parenting Stress lndex, 4th Edition (PSl-4)
  AII study visits (30, 36, 42, 48, and 60 Months)
• Parental Global lmpression Scale
  AII study visits (30, 36, 42, 48, and 60 Months)
• Clinical Global lmpression lmprovement Scale
  AII study visits (30, 36, 42, 48, and 60 Months)
• Parent Symptom Scale Questionnaire
  AII study visits (30, 36, 42, 48, and 60 Months)
• T-cell responses against the AAV9 capsid
  60 Months
• T-cell responses against the α-N- acetylglucosaminidase (NAGLU) transgene product
  60 Months
• Antibody formation (humoral) against the α-N- acetylglucosaminidase (NAGLU) transgene product
  60 Months
• Antibody formation (humoral) against the AAV9 capsid
  60 Months
• Viral Load
  AII study visits (30, 36, 42, 48, and 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-101 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, [email protected]

trial center locations: United States,France,Germany

• United States, Ohio
  Nationwide Children’s Hospital
  Shivani Deshpande, 614-722-2238, [email protected]
  
  
• France
  Armand-Trousseau Hospital
  Bénédicte Héron, MD, +33 (0)1 44 73 74 30, [email protected]
  
  
• Germany
  University Hospital Hamburg-Eppendorf
  Nicole M Muschol, MD, [email protected]