A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101
- Conditions
- Mucopolysaccharidosis III-B
- Interventions
- Biological: ABO-101
- Registration Number
- NCT04655911
- Lead Sponsor
- Abeona Therapeutics, Inc
- Brief Summary
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).
- Detailed 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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 1
- Participants that have completed a prior clinical trial involving the administration of ABO-101
- Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule
- 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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description ABO-101 ABO-101 Participants from prior interventional trials involving the administration of ABO-101.
- Primary Outcome Measures
Name Time Method Adverse Events 60 months Long-term product safety as defined by the incidence, type, and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
- Secondary Outcome Measures
Name Time Method Age Equivalent Compared to Natural History Study Data AII study visits (30, 36, 42, 48, and 60 Months) 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
Parenting Stress lndex, 4th Edition (PSl-4) AII study visits (30, 36, 42, 48, and 60 Months) Quality of life based on Parenting Stress lndex, 4th Edition (PSl-4).
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) 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
Pediatric Quality of Life lnventory (PedsQL ™) AII study visits (30, 36, 42, 48, and 60 Months) Quality of life based on Pediatric Quality of Life lnventory (PedsQL ™) applicable for participants who completed these evaluations in the prior clinical trial.
Cognitive Age Equivalent Compared to Natural History Study Data AII study visits (30, 36, 42, 48, and 60 Months) 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
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) 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
Adaptive Age Equivalent Compared to Natural History Study Data AII study visits (30, 36, 42, 48, and 60 Months) 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
Parent Symptom Scale Questionnaire AII study visits (30, 36, 42, 48, and 60 Months) Evolution on Parent Symptom Score Questionnaire applicable for patients who completed these evaluations in the prior clinical trial.
Parental Global lmpression Scale AII study visits (30, 36, 42, 48, and 60 Months) Evolution on Parental Global lmpression Score, applicable for patients who completed these evaluations in the prior clinical trial.
T-cell responses against the AAV9 capsid 60 Months Long-term immunological responses defined as T-cell responses against the AAV9 capsid
Antibody formation (humoral) against the AAV9 capsid 60 Months Long-term immunological responses defined as antibody formation (humoral) against the AAV9 capsid.
Clinical Global lmpression lmprovement Scale AII study visits (30, 36, 42, 48, and 60 Months) Evolution of Clinical Global lmpression lmprovement Score applicable for patients who completed these evaluations in the prior clinical trial.
Viral Load AII study visits (30, 36, 42, 48, and 60 Months) Long-term viral load, in applicable cases.
T-cell responses against the α-N- acetylglucosaminidase (NAGLU) transgene product 60 Months Long-term immunological responses defined as T-cell responses against the α-N- acetylglucosaminidase transgene product.
Antibody formation (humoral) against the α-N- acetylglucosaminidase (NAGLU) transgene product 60 Months Long-term immunological responses defined as antibody formation (humoral) against the α-N- acetylglucosaminidase (NAGLU)
Trial Locations
- Locations (3)
Armand-Trousseau Hospital
🇫🇷Paris, France
University Hospital Hamburg-Eppendorf
🇩🇪Hamburg, Germany
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States