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

Mucopolysaccharidosis III-A Clinical Trial

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)

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04360265
• Other study ID #: LTFU-ABO-102
• Secondary ID: 2019-002979-34UX
• Status: Enrolling by invitation
• Phase: Phase 3
• Start date: September 28, 2020
• Est. completion date: June 2027

Study information

• Verified date: May 2024
• Source: Ultragenyx Pharmaceutical Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The main objective of this study is to evaluate the long-term safety and tolerability of ABO-102 in participants with MPS IIIA.

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 33
• Est. completion date: June 2027
• Est. primary completion date: June 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis III
• 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.

Locations

Country	Name	City	State
Australia	Women's and Children's Hospital	North Adelaide	South Australia
Spain	Hospital Clínico Universitario de Santiago	Santiago De Compostela
United States	Nationwide Children's Hospital	Columbus	Ohio

Sponsors (2)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc	Abeona Therapeutics, Inc

Countries where clinical trial is conducted

United States,  Australia,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse Events	Long-term Product Safety as Defined by the Incidence, Type, and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).	Up to Year 5
Secondary	Change From Baseline (in Prior Trial) in Cerebrospinal Fluid Heparan Sulfate (CSF HS)		Up to Month 12
Secondary	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		Baseline, Up to Year 5
Secondary	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		Baseline, Up to Year 5
Secondary	Change From Baseline (in Prior Trial), as Assessed by the Parent Report Using the Vineland Adaptive Behavior Scale II - Survey Interview Form (VABS-II)		Baseline, Up to Year 5