Long-term safety study of rebisufligene etisparvovec gene therapy in patients previously treated for Mucopolysaccharidosis type IIIA

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What is this study about?

This study focuses on patients with Mucopolysaccharidosis type IIIA, a rare genetic disorder that affects the body’s ability to break down certain complex sugars. The study will evaluate a gene therapy treatment called ABO-102 (also known as rebisufligene etisparvovec), which is given as an injection into the bloodstream. This treatment is designed to help the body produce an important enzyme that is missing in patients with this condition.

The main purpose of this research is to assess the long-term safety and effectiveness of the treatment in patients who have previously received ABO-102 in earlier clinical trials. The treatment works by introducing a corrective gene into the patient’s cells, which should then be able to produce the missing enzyme and help restore normal cellular function.

During the study, participants will be monitored through various tests and examinations to track their progress and any potential side effects. The research team will evaluate different aspects of the participants’ health, including their cognitive development, communication abilities, and certain substances in their spinal fluid. The study will also track changes in brain volume and overall survival of the participants.

1 Initial treatment verification

Confirmation of previous participation in a clinical trial where you received UX111 (also known as ABO-102)

Review and signing of informed consent documents by you or your legal guardian

2 Treatment monitoring

Regular assessment of any side effects that may occur after the treatment

Monitoring of cognitive development using specialized tests (Bayley Scales of Infant and Toddler Development)

Evaluation of communication skills, including both understanding and expressing language

Collection of spinal fluid samples to measure levels of a substance called heparan sulfate

Brain imaging to measure changes in brain volume

3 Long-term follow-up

Continued monitoring until August 2027

Regular medical check-ups to ensure safety and track progress

Assessment of overall health status and survival outcomes

4 Medical supervision

The medication (rebisufligene etisparvovec) was previously administered through intravenous injection

This study focuses on monitoring the long-term effects of that treatment

Your participation helps understand how the treatment affects patients with Mucopolysaccharidosis type IIIA over time

Who Can Join the Study?

Must have previously participated in a clinical trial where they received UX111 (an investigational treatment)
Must be between 2-11 years old
Can be either male or female
Parents or legal guardians must be willing and able to:
- Give informed consent (written permission to participate)
- Follow all study procedures
- Attend all scheduled study visits
Must have been diagnosed with Mucopolysaccharidosis type IIIA (a rare genetic condition affecting the body’s ability to break down certain complex sugars)
Must be able to comply with all study requirements and procedures

Who Cannot Join the Study?

History of hematopoietic stem cell transplantation (a procedure where damaged blood-forming cells are replaced with healthy ones)
Participation in another clinical trial within 30 days before starting this study
Any serious medical condition or abnormal test result that could interfere with the study or put the participant at risk
Known allergies to the study medication or similar compounds
Presence of any contraindications (conditions that make a treatment inadvisable) to the required procedures
Inability to comply with study procedures and follow-up visits
Pregnancy or breastfeeding
Use of prohibited medications within 30 days before starting the study
Major surgery planned during the study period
Significant abnormal findings in laboratory tests or medical examinations
Mental conditions that could affect the ability to provide informed consent or follow study procedures
History of substance abuse within the past year
Any condition that, in the opinion of the study doctor, would make participation unsafe

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Universidade De Santiago De Compostela	Santiago De Compostela	Spain
Hnghpnpr Vgoc dgyvwglm	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Spain	Recruiting	02.05.2020

Trial locations

Investigated drugs:

Rebisufligene Etisparvovec

ABO-102 (also known as UX111) is a gene therapy product designed to treat patients with MPS IIIA (Mucopolysaccharidosis type IIIA), also known as Sanfilippo syndrome type A. This therapy uses a modified virus (AAV9) to deliver a working copy of the SGSH gene to cells. The therapy aims to help the body produce an enzyme that patients with MPS IIIA are missing, which is needed to break down certain complex sugars in the body.

Investigated diseases:

Mucopolysaccharidosis III

Mucopolysaccharidosis type IIIA – A rare genetic disorder that affects the body’s ability to break down complex sugar molecules called heparan sulfate. The condition is characterized by progressive deterioration of the central nervous system. Children with this disorder typically begin showing symptoms between ages 2 and 6, including developmental delays and behavioral problems. As the disease progresses, affected individuals experience a decline in cognitive abilities and language skills. The condition also causes mild physical changes and can affect motor skills over time. The disease is inherited in an autosomal recessive pattern, meaning both parents must pass on the altered gene for a child to be affected.

Trial ID:

2023-510392-66-00

Protocol code:

LTFU-ABO-102

NCT ID:

NCT04360265

Trial Phase:

Therapeutic confirmatory (Phase III)

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Long-term safety study of rebisufligene etisparvovec gene therapy in patients previously treated for Mucopolysaccharidosis type IIIA

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?