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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04360265




Registration number
NCT04360265
Ethics application status
Date submitted
20/04/2020
Date registered
24/04/2020
Date last updated
30/10/2020

Titles & IDs
Public title
A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102
Scientific title
A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)
Secondary ID [1] 0 0
2019-002979-34
Secondary ID [2] 0 0
LTFU-ABO-102
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mucopolysaccharidosis III-A 0 0
Condition category
Condition code

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - ABO-102

ABO-102 - Participants from prior interventional trials involving the administration of ABO-102.


Other interventions: 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.

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Adverse Events - Long-term product safety as defined by the incidence, type, and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
Timepoint [1] 0 0
60 Months
Secondary outcome [1] 0 0
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 for Children; Second Edition, based on chronological and developmental age
Timepoint [1] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [2] 0 0
Developmental Quotient Compared to Natural History Study Data - 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
Timepoint [2] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [3] 0 0
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 Infant and Toddler Development - Third edition or the Kaufman Assessment Battery for Children. Second Edition, based on developmental age
Timepoint [3] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [4] 0 0
Developmental Quotient Compared to Natural History Study Data - 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
Timepoint [4] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [5] 0 0
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 form
Timepoint [5] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [6] 0 0
Pediatric Quality of Life Inventory (PedsQL™) - Quality of life based on Pediatric Quality of Life Inventory (PedsQL™) applicable for participants who completed these evaluations in the prior clinical trial.
Timepoint [6] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [7] 0 0
Pediatric Quality of Life Inventory Gastrointestinal Symptom - Quality of life based on Pediatric Quality of Life Inventory Gastrointestinal Symptom, applicable for participants who completed these evaluations in the prior clinical trial.
Timepoint [7] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [8] 0 0
Parenting Stress Index, 4th Edition (PSI-4) - Quality of life based on Parenting Stress Index, 4th Edition (PSI-4).
Timepoint [8] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [9] 0 0
Children's Sleep Habits Questionnaire (CSHQ) - Quality of life based on sleep-pattern evaluation based on modified Children's Sleep Habits Questionnaire (CSHQ).
Timepoint [9] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [10] 0 0
Parental Global Impression Scale - Evolution on Parental Global Impression Score, applicable for patients who completed these evaluations in the prior clinical trial.
Timepoint [10] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [11] 0 0
Clinical Global Impression Improvement Scale - Evolution of Clinical Global Impression Improvement Score applicable for patients who completed these evaluations in the prior clinical trial.
Timepoint [11] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [12] 0 0
Parent Symptom Scale Questionnaire - Evolution on Parent Symptom Score Questionnaire applicable for patients who completed these evaluations in the prior clinical trial.
Timepoint [12] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [13] 0 0
T-cell responses against the AAV9 capsid - Long-term immunological responses defined as T-cell responses against the AAV9 capsid.
Timepoint [13] 0 0
60 Months
Secondary outcome [14] 0 0
T-cell responses against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product - Long-term immunological responses defined as T-cell responses against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product.
Timepoint [14] 0 0
60 Months
Secondary outcome [15] 0 0
Antibody formation (humoral) against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product. - Long-term immunological responses defined as antibody formation (humoral) against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product.
Timepoint [15] 0 0
60 Months
Secondary outcome [16] 0 0
Antibody formation (humoral) against the AAV9 capsid - Long-term immunological responses defined as antibody formation (humoral) against the AAV9 capsid.
Timepoint [16] 0 0
60 Months
Secondary outcome [17] 0 0
Viral Load - Long-term viral lod, in aplicable cases.
Timepoint [17] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [18] 0 0
Sanfilippo Behavior Rating Scale - 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
Timepoint [18] 0 0
All study visits (30, 36, 42, 48, and 60 Months)
Secondary outcome [19] 0 0
Neutralizing antibody against the AAV9 capsid - Long-term immunological responses defined as neutralizing antibody formation against the AAV9 capsid.
Timepoint [19] 0 0
60 Months

Eligibility
Key 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.
Minimum age
No limit
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- 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.

- Any other situation that precludes the participant from undergoing procedures required
in this study.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Women's and Children's Hospital - North Adelaide
Recruitment postcode(s) [1] 0 0
- North Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Ohio
Country [2] 0 0
Spain
State/province [2] 0 0
Santiago De Compostela

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Abeona Therapeutics, Inc
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT04360265
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Medical Affairs
Address 0 0
Country 0 0
Phone 0 0
+1 646-813-4701
Fax 0 0
Email 0 0
sanfilippo@abeonatherapeutics.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04360265