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)
- Conditions
- MPS IIIB is a devastating lysosomal storage disease, caused by a N-a-acetylglucosaminidase (NAGLU) gene defect. Infants with MPS IIIB 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 treatment currently available for the disease.MedDRA version: 20.1Level: PTClassification code 10056890Term: Mucopolysaccharidosis IIISystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2019-002936-97-DE
- Lead Sponsor
- Abeona Therapeutics Europe SL.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 24
• Participants that have completed a prior clinical trial involving the administration of ABO-101.
• 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: 24
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
• Planned or current participation in another clinical trial that may confound the safety or efficacy evaluation of ABO-101 during 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: The primary objective of the study is to evaluate the long-term safety/tolerability of ABO-101 in patients with MPS IIIB.;Secondary Objective: The secondary objective is to evaluate the neurocognitive evolution of patients with MPS IIIB treated with ABO-101.;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: Safety will be evaluated along the entire trial for 3 years.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): • Neurocognitive evolution based on developmental-age appropriate assessments included in the prior clinical trial.<br>• Quality of life based on Pediatric Quality of Life Inventory (PedsQL™ and PedsQL Grastrointestinal Symptom scales-this last one, only applicable for participants who completed these evaluations in the prior clinical trial), Parenting Stress Index, 4th Edition (PSI-4), and sleep-pattern evaluation based on modified Children’s Sleep Habits Questionnaire (CSHQ).<br>• Long-term immunological responses defined as antibody formation (humoral) and T-cell responses (cellular) against the AAV9 capsid and against the a-N-acetylglucosaminidase (NAGLU) transgene product.<br>• Long-term viral load, in applicable cases.;Timepoint(s) of evaluation of this end point: Annually (± 2 months) for three years.