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)

Phase 1

• Conditions: MPS IIIA is a devastating lysosomal storage disease, caused by a Nsulfoglucosamine sulfohydrolase gene defect. Infants with MPS IIIA appear normal at birth, but the disease is relentlessly progressive, with deterioration of social and adaptive abilities, neurocognitive decline, and premature death. Death typically occurs by end of the second or beginning of the third decade. Quite importantly, there is no treatmentcurrently available for the disease.; MedDRA version: 20.1Level: PTClassification code 10056890Term: Mucopolysaccharidosis IIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2019-002979-34-ES
• Lead Sponsor: Abeona Therapeutics Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-05-25
• Last Update Posted: 30 November 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 34

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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 34
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the long-term safety/tolerability of ABO-102 in patients with MPS IIIA;Secondary Objective: To evaluate the neurocognitive evolution of patients with MPS IIIA treated with ABO-102;Primary end point(s): Long-term product safety as defined by the incidence, type, and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).;Timepoint(s) of evaluation of this end point: Months: 30, 36, 42 (only for subjects recruited below 3 years of age) , 48, 60. After ABO-102 treatment administered in a prior clinical trial.