A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102

• ClinicalTrials.gov Identifier: NCT04360265
• Recruitment Status: Enrolling by invitation
• First Posted: April 24, 2020
• Last Update Posted: May 14, 2024
• Sponsor: Ultragenyx Pharmaceutical Inc
• Collaborator: Abeona Therapeutics, Inc
• Information provided by (Responsible Party): Ultragenyx Pharmaceutical Inc

Tracking Information

• First Submitted Date: April 20, 2020
• First Posted Date: April 24, 2020
• Last Update Posted Date: May 14, 2024
• Actual Study Start Date: September 28, 2020
• Estimated Primary Completion Date: June 2027 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 1, 2023)

Adverse Events [ Time Frame: Up to Year 5 ]

Long-term Product Safety as Defined by the Incidence, Type, and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).

Original Primary Outcome Measures (submitted: April 21, 2020)

Adverse Events [ Time Frame: up to 60 Months ]

Incidence, type, and severity of Adverse Events (AEs)

Current Secondary Outcome Measures (submitted: May 1, 2023)

• Change From Baseline (in Prior Trial) in Cerebrospinal Fluid Heparan Sulfate (CSF HS) [ Time Frame: Up to Month 12 ]
• Change From Baseline (in Prior Trial) in the Cognitive Subtest of the Bayley Scales of Infant and Toddler Development -Third Edition (BSID-III) or the Kaufman Assessment Battery for Children - Second Edition (KABC-II), Based on Developmental Age [ Time Frame: Baseline, Up to Year 5 ]
• Change From Baseline (in Prior Trial) in the Language Subtest of the Bayley Scales of Infant and Toddler Development -Third Edition (BSID-III) or the Kaufman Assessment Battery for Children - Second Edition (KABC-II), Based on Developmental Age [ Time Frame: Baseline, Up to Year 5 ]
• Change From Baseline (in Prior Trial), as Assessed by the Parent Report Using the Vineland Adaptive Behavior Scale II - Survey Interview Form (VABS-II) [ Time Frame: Baseline, Up to Year 5 ]

Original Secondary Outcome Measures (submitted: April 21, 2020)

• Mullen Scales of Early Learning or the Kaufman Assessment Battery for Children; Second Edition [ Time Frame: 30, 36, 42, 48, and 60 Months ]
  Neurocognitive evolution based on developmental-age appropriate assessments included in the prior clinical trial.
• Pediatric Quality of Life Inventory (PedsQL™) [ Time Frame: 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 [ Time Frame: 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) [ Time Frame: 30, 36, 42, 48, and 60 Months ]
  Quality of life based on Parenting Stress Index, 4th Edition (PSI-4) applicable for participants who completed these evaluations in the prior clinical trial.
• Children's Sleep Habits Questionnaire (CSHQ) [ Time Frame: 30, 36, 42, 48, and 60 Months ]
  Quality of life based on sleep-pattern evaluation based on modified Children's Sleep Habits Questionnaire (CSHQ), applicable for participants who completed these evaluations in the prior clinical trial.
• Parental Global Impression Score [ Time Frame: 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 Score [ Time Frame: 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 Score Questionnaire [ Time Frame: 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.
• Antibody formation (humoral) against the AAV9 capsid [ Time Frame: 60 Months ]
  Long-term immunological responses defined as antibody formation (humoral) and T-cell responses (cellular) against the AAV9 capsid.
• T-cell responses against the AAV9 capsid [ Time Frame: 60 Months ]
  Long-term immunological responses defined as T-cell responses against the AAV9 capsid.
• Antibody formation (humoral) against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product. [ Time Frame: 60 Months ]
  Long-term immunological responses defined as antibody formation (humoral) against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product.
• T-cell responses against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product. [ Time Frame: 60 Months ]
  Long-term immunological responses defined as T-cell responses against the N-sulfoglucosamine sulfohydrolase (SGSH) transgene product.
• Viral load [ Time Frame: 30, 36, 42, 48, and 60 Months ]
  Long-term mentoring of viral load in those participants with positive results from previous trial.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102
• Official 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)
• Brief Summary: The main objective of this study is to evaluate the long-term safety and tolerability of ABO-102 in participants with MPS IIIA.

Detailed Description

This is a multicenter, long-term follow-up study of patients with MPS IIIA who have completed a prior clinical trial involving the administration of ABO-102 (NCT02716246 and NCT04088734). No investigational product will be provided. At approved sites immunosuppression (IS) therapy may be administered to selected participants. The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS therapy. Not all participants may receive IS therapy.

This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.

• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Other
• Condition: Mucopolysaccharidosis III-A

Intervention

• Other: No Investigational Product
  No investigational product will be administered in this follow-up trial.
• Drug: Adjuvant Immunosuppression (IS) Therapy
  The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS therapy.

Study Arms

• Cohort 1
  Participants who have completed a prior clinical trial involving the administration of ABO-102 and are able to comply with onsite scheduled visits and assessments. Select participants may receive adjuvant IS therapy.
  Interventions:

  • Other: No Investigational Product
  • Drug: Adjuvant Immunosuppression (IS) Therapy
• Cohort 2
  Participants who have completed a prior clinical trial involving the administration of ABO-102 and who cannot participate in Cohort 1. Participants will partake in a reduced number of assessments, performed either onsite or at home via a combination of telehealth and home healthcare visits.
  Intervention: Other: No Investigational Product

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Enrolling by invitation
• Estimated Enrollment (submitted: May 1, 2023): 33
• Original Estimated Enrollment (submitted: April 21, 2020): 50
• Estimated Study Completion Date: June 2027
• Estimated Primary Completion Date: June 2027 (Final data collection date for primary outcome measure)

Eligibility 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:

• Planned or current participation in another clinical trial that may confound the safety and efficacy evaluation of ABO-102 during this study.
• Any other situation or medical condition that precludes the participant from undergoing procedures required in this study.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: Child, Adult, Older Adult
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Australia, Spain, United States

Administrative Information

• NCT Number: NCT04360265
• Other Study ID Numbers: LTFU-ABO-102; 2019-002979-34 ( EudraCT Number ); UX111-CL302 ( Other Identifier: Ultragenyx Pharmaceutical Inc )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Ultragenyx Pharmaceutical Inc
• Original Responsible Party: Abeona Therapeutics, Inc
• Current Study Sponsor: Ultragenyx Pharmaceutical Inc
• Original Study Sponsor: Abeona Therapeutics, Inc
• Collaborators: Abeona Therapeutics, Inc

Investigators

• Study Director: Medical Director; Ultragenyx Pharmaceutical Inc

• PRS Account: Ultragenyx Pharmaceutical Inc
• Verification Date: May 2024