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

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-002936-97-DE

Enrollment

Registered

2020-01-08

Start date

Unknown

Completion date

Unknown

Last updated

2022-04-25

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

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: 20.1 Level: PT Classificat

Interventions

Product Name: rAAV9.CMV.hNAGLU Product Code: ABO-101 Pharmaceutical Form: Suspension for injection INN or Proposed INN: rAAV9.CMV.hNAGLU Current Sponsor code: ABO-101 Other descriptive name: Adeno-ass

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: • 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

Exclusion criteria

Exclusion criteria: • 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.

Design outcomes

Primary

Measure	Time frame
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.	—

Measure

Time frame

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.

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Secondary

Measure	Time frame
Secondary end point(s): • Neurocognitive evolution based on developmental-age appropriate assessments included in the prior clinical trial. • 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). • 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. • Long-term viral load, in applicable cases.;Timepoint(s) of evaluation of this end point: Annually (± 2 months) for three years.	—

Measure

Time frame

Secondary end point(s): • Neurocognitive evolution based on developmental-age appropriate assessments included in the prior clinical trial. • 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). • 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. • Long-term viral load, in applicable cases.;Timepoint(s) of evaluation of this end point: Annually (± 2 months) for three years.

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Countries

France, Germany, Spain, United States

Contacts

Public ContactHead of European Medical Affairs

Abeona Therapeutics Inc

sanfilippo@abeonatherapeutics.com+34689166070

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026