A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102
Study Details
Study Description
Brief Summary
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Detailed 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.
This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| ABO-102 Participants from prior interventional trials involving the administration of ABO-102. |
Biological: ABO-102
Study drug is not administered in this trial. Participants who received ABO-102 in a previous trial will be evaluated for long-term safety and efficacy.
Other Names:
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Outcome Measures
Primary Outcome Measures
- 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
- Age Equivalent Compared to Natural History Study Data [All 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 for Children; Second Edition, based on chronological and developmental age
- Developmental Quotient Compared to Natural History Study Data [All 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 [All 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 Infant and Toddler Development - Third edition or the Kaufman Assessment Battery for Children. Second Edition, based on developmental age
- Developmental Quotient Compared to Natural History Study Data [All 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 Infant and Toddler Development - Third edition or the Kaufman Assessment Battery for Children. Second Edition, based on developmental age
- Adaptive Age Equivalent Compared to Natural History Study Data [All 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 form
- Pediatric Quality of Life Inventory (PedsQL™) [All study visits (30, 36, 42, 48, and 60 Months)]
Quality of life based on Pediatric Quality of Life Inventory (PedsQL™) applicable for participants who completed these evaluations in the prior clinical trial.
- Pediatric Quality of Life Inventory Gastrointestinal Symptom [All study visits (30, 36, 42, 48, and 60 Months)]
Quality of life based on Pediatric Quality of Life Inventory Gastrointestinal Symptom, applicable for participants who completed these evaluations in the prior clinical trial.
- Parenting Stress Index, 4th Edition (PSI-4) [All study visits (30, 36, 42, 48, and 60 Months)]
Quality of life based on Parenting Stress Index, 4th Edition (PSI-4).
- Children's Sleep Habits Questionnaire (CSHQ) [All study visits (30, 36, 42, 48, and 60 Months)]
Quality of life based on sleep-pattern evaluation based on modified Children's Sleep Habits Questionnaire (CSHQ).
- Parental Global Impression Scale [All study visits (30, 36, 42, 48, and 60 Months)]
Evolution on Parental Global Impression Score, applicable for patients who completed these evaluations in the prior clinical trial.
- Clinical Global Impression Improvement Scale [All study visits (30, 36, 42, 48, and 60 Months)]
Evolution of Clinical Global Impression Improvement Score applicable for patients who completed these evaluations in the prior clinical trial.
- Parent Symptom Scale Questionnaire [All 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 N-sulfoglucosamine sulfohydrolase (SGSH) transgene product [60 Months]
Long-term immunological responses defined as T-cell responses against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product.
- Antibody formation (humoral) against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product. [60 Months]
Long-term immunological responses defined as antibody formation (humoral) against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product.
- Antibody formation (humoral) against the AAV9 capsid [60 Months]
Long-term immunological responses defined as antibody formation (humoral) against the AAV9 capsid.
- Viral Load [All study visits (30, 36, 42, 48, and 60 Months)]
Long-term viral lod, in aplicable cases.
- Sanfilippo Behavior Rating Scale [All study visits (30, 36, 42, 48, and 60 Months)]
Change from baseline in the SBRS scale after treatment compared to Natural History Study data, as assessed by parent report using the Sanfilippo Behavior Rating Scale form
- Neutralizing antibody against the AAV9 capsid [60 Months]
Long-term immunological responses defined as neutralizing antibody formation against the AAV9 capsid.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Participants that have completed a prior clinical trial involving the administration of ABO-102.
-
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:
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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.
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Any other situation that precludes the participant from undergoing procedures required in this study.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Nationwide Children's Hospital | Columbus | Ohio | United States | 43205 |
| 2 | Women's and Children's Hospital | North Adelaide | South Australia | Australia | |
| 3 | Hospital Clínico Universitario de Santiago | Santiago De Compostela | Spain |
Sponsors and Collaborators
- Ultragenyx Pharmaceutical Inc
- Abeona Therapeutics, Inc
Investigators
- Study Director: Medical Director, Ultragenyx Pharmaceutical Inc
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- LTFU-ABO-102
- 2019-002979-34
- UX111-CL302