Back to resultsA Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102
Primary Purpose
Mucopolysaccharidosis III-A
Status
Enrolling by invitation
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
No Investigational Product
Adjuvant Immunosuppression (IS) Therapy
Sponsored by
About this trial
This is an interventional other trial for Mucopolysaccharidosis III-A focused on measuring MPS IIIA, Mucopolysaccharidosis IIIA, Sanfilippo A
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 the duration of this study.
- Any other situation that precludes the participant from undergoing procedures required in this study.
Sites / Locations
- Nationwide Children's Hospital
- Women's and Children's Hospital
- Hospital Clínico Universitario de Santiago
Arms of the Study
Arm 1
Arm 2
Arm Type
Other
Other
Arm Label
Cohort 1
Cohort 2
Arm Description
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.
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.
Outcomes
Primary Outcome Measures
Adverse Events
Long-term Product Safety as Defined by the Incidence, Type, and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
Secondary Outcome Measures
Change From Baseline (in Prior Trial) in Cerebrospinal Fluid Heparan Sulfate (CSF HS)
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
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
Change From Baseline (in Prior Trial), as Assessed by the Parent Report Using the Vineland Adaptive Behavior Scale II - Survey Interview Form (VABS-II)
Full Information
NCT ID
NCT04360265
First Posted
April 20, 2020
Last Updated
August 2, 2023
Sponsor
Ultragenyx Pharmaceutical Inc
Collaborators
Abeona Therapeutics, Inc
1. Study Identification
Unique Protocol Identification Number
NCT04360265
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)
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
September 28, 2020 (Actual)
Primary Completion Date
June 2027 (Anticipated)
Study Completion Date
June 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ultragenyx Pharmaceutical Inc
Collaborators
Abeona Therapeutics, Inc
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis III-A
Keywords
MPS IIIA, Mucopolysaccharidosis IIIA, Sanfilippo A
7. Study Design
Primary Purpose
Other
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
33 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Cohort 1
Arm Type
Other
Arm Description
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.
Arm Title
Cohort 2
Arm Type
Other
Arm Description
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 Type
Other
Intervention Name(s)
No Investigational Product
Intervention Description
No investigational product will be administered in this follow-up trial.
Intervention Type
Drug
Intervention Name(s)
Adjuvant Immunosuppression (IS) Therapy
Intervention Description
The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS therapy.
Primary Outcome Measure Information:
Title
Adverse Events
Description
Long-term Product Safety as Defined by the Incidence, Type, and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
Time Frame
Up to Year 5
Secondary Outcome Measure Information:
Title
Change From Baseline (in Prior Trial) in Cerebrospinal Fluid Heparan Sulfate (CSF HS)
Time Frame
Up to Month 12
Title
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
Title
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
Title
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
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Ultragenyx Pharmaceutical Inc
Official's Role
Study Director
Facility Information:
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Women's and Children's Hospital
City
North Adelaide
State/Province
South Australia
Country
Australia
Facility Name
Hospital Clínico Universitario de Santiago
City
Santiago De Compostela
Country
Spain
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102
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