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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-002936-97 | EudraCT Number |
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Abeona has decided to discontinue development activities for Product AB0-101 due to a lack of drug supply and for business reasons unrelated to the product safety profile and/or signs of efficacy
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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ABO-101 | Participants from prior interventional trials involving the administration of ABO-101. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ABO-101 | Biological | Gene therapy rAAV9.CMV.hNAGLU |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Age Equivalent Compared to Natural History Study Data | 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 | AII study visits (30, 36, 42, 48, and 60 Months) |
| Measure | Description | Time Frame |
|---|---|---|
| Sanfilippo Behavior Rating Scale | 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 | 30, 36, 42, 48, and 60 Months |
Inclusion Criteria:
Exclusion Criteria:
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This long-term follow-up study will enroll up to 24patients with MPS IIIB from prior ABO-101 clinical trials. Participants from the previous trials are of any racial, ethnic, or gender background, and could be in the early, middle or advanced phase of the disease
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States | ||
| Armand-Trousseau Hospital |
There is no plan to share data
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| ID | Term |
|---|---|
| D009084 | Mucopolysaccharidosis III |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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| Developmental Quotient Compared to Natural History Study Data calculated by the Mullen Scales of Early Learning or the Kaufman Assessment Battery for Children | 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 | AII study visits (30, 36, 42, 48, and 60 Months) |
| Cognitive Age Equivalent Compared to Natural History Study Data | 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 | 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 | 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 | AII study visits (30, 36, 42, 48, and 60 Months) |
| Adaptive Age Equivalent Compared to Natural History Study Data | 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 | AII study visits (30, 36, 42, 48, and 60 Months) |
| Pediatric Quality of Life lnventory (PedsQL ™) | Quality of life based on Pediatric Quality of Life lnventory (PedsQL ™) applicable for participants who completed these evaluations in the prior clinical trial. | AII study visits (30, 36, 42, 48, and 60 Months) |
| Parenting Stress lndex, 4th Edition (PSl-4) | Quality of life based on Parenting Stress lndex, 4th Edition (PSl-4). | AII study visits (30, 36, 42, 48, and 60 Months) |
| Parental Global lmpression Scale | Evolution on Parental Global lmpression Score, applicable for patients who completed these evaluations in the prior clinical trial. | AII study visits (30, 36, 42, 48, and 60 Months) |
| Clinical Global lmpression lmprovement Scale | Evolution of Clinical Global lmpression lmprovement Score applicable for patients who completed these evaluations in the prior clinical trial. | AII study visits (30, 36, 42, 48, and 60 Months) |
| Parent Symptom Scale Questionnaire | Evolution on Parent Symptom Score Questionnaire applicable for patients who completed these evaluations in the prior clinical trial. | AII study visits (30, 36, 42, 48, and 60 Months) |
| T-cell responses against the AAV9 capsid | Long-term immunological responses defined as T-cell responses against the AAV9 capsid | 60 Months |
| T-cell responses against the α-N- acetylglucosaminidase (NAGLU) transgene product | Long-term immunological responses defined as T-cell responses against the α-N- acetylglucosaminidase transgene product. | 60 Months |
| Antibody formation (humoral) against the α-N- acetylglucosaminidase (NAGLU) transgene product | Long-term immunological responses defined as antibody formation (humoral) against the α-N- acetylglucosaminidase (NAGLU) | 60 Months |
| Antibody formation (humoral) against the AAV9 capsid | Long-term immunological responses defined as antibody formation (humoral) against the AAV9 capsid. | 60 Months |
| Viral Load | Long-term viral load, in applicable cases. | AII study visits (30, 36, 42, 48, and 60 Months) |
| Children's Sleep Habits Questionnaire (CSHQ) | 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 | 30, 36, 42, 48, and 60 Months |
| Neutralizing antibody against the AAV9 capsid | Long-term immunological responses defined as neutralizing antibody formation against the AAV9 capsid | 60 Months |
| Paris |
| 75571 |
| France |
| University Hospital Hamburg-Eppendorf | Hamburg | 20246 | Germany |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |