Mucopolysaccharidosis III-B
Conditions
Keywords
MPS IIIB, Mucopolysaccharidosis IIIB, Sanfilippo B
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.
Interventions
BIOLOGICALABO-101
Gene therapy rAAV9.CMV.hNAGLU
Sponsors
Abeona Therapeutics, Inc
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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). |
| 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) |
| Antibody formation (humoral) against the AAV9 capsid | 60 Months | Long-term immunological responses defined as antibody formation (humoral) against the AAV9 capsid. |
| Viral Load | AII study visits (30, 36, 42, 48, and 60 Months) | Long-term viral load, in applicable cases. |
| 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 |
| 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 |
| 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 |
| 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. |
| 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. |
| 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. |
| 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. |
| T-cell responses against the AAV9 capsid | 60 Months | Long-term immunological responses defined as T-cell responses against the AAV9 capsid |
| 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. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Neutralizing antibody against the AAV9 capsid | 60 Months | Long-term immunological responses defined as neutralizing antibody formation against the AAV9 capsid |
| Sanfilippo Behavior Rating Scale | 30, 36, 42, 48, and 60 Months | Change from baseline in the Sanfilippo Behavior Rating Scale (SBRS) scale after treatment compared to Natural History Study data, as assessed by parent report using the Sanfilippo Behavior Rating Scale form. Minimum value is 0 and maximum value is 6 where higher score indicates a worse outcome |
| Children's Sleep Habits Questionnaire (CSHQ) | 30, 36, 42, 48, and 60 Months | Quality of life based on sleep-pattern evaluation based on modified Children's Sleep Habits Questionnaire (CSHQ).Minimum value is 1 and maximum value is 111 where higher scores indicates a worse outcome |
Countries
France, Germany, United States
Outcome results
None listed