NCT04655911

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

A Long-term Follow-up Study of Patients With MPS IIIB From Gene Therapy Clinical Trials Involving the Administration of ABO-101 (rAAV9.CMV.hNAGLU)

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 1 (actual)
• Sponsor: Abeona Therapeutics, Inc · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This is a multicenter, non-interventional, long-term follow-up (LTFU) study in participants who have been treated with ABO-101 in a prior trial. Eligible participants will undergo clinical evaluations at prespecified intervals for 3 years from the last visit in the prior clinical trial (up to 5 years post-treatment).

Detailed description

This is a multicenter, noninterventional, long-term follow-up study of patients with (Mucopolysaccharidosis IIIB) MPS IIIB who have completed a prior clinical trial involving the administration of ABO-101. This study is designed to provide LTFU in accordance with the FDA and European Medicines Agency (EMA) guidelines for patients treated with gene therapy products. The duration of the current study is 3 years for a total of up to 5 years post-treatment for these participants who rollover from a prior clinical trial of ABO-101. Participants will have a maximum of 5 annual scheduled visits with assessments as specified in the schedule of assessments.

Conditions

• Mucopolysaccharidosis III-B

Interventions

Type	Name	Description
BIOLOGICAL	ABO-101	Gene therapy rAAV9.CMV.hNAGLU

Timeline

• Start date: 2020-10-28
• Primary completion: 2022-04-07
• Completion: 2022-04-07
• First posted: 2020-12-07
• Last updated: 2022-05-31

Locations

3 sites across 3 countries: United States, France, Germany

Regulatory

• FDA-regulated drug study