What side effects are associated with this type of intervention?

Gene therapy treatments for Sanfilippo Syndrome, such as ABO-102, primarily aim to address the underlying genetic mutations causing MPS IIIA. Known side effects of such gene therapies can include immune responses to the viral vector used for gene delivery, which may manifest as fever, inflammation, or other immune-related reactions. Additionally, there may be risks of insertional mutagenesis, where the inserted gene disrupts other genes, potentially leading to unintended effects. Other common side effects can include headaches, nausea, and fatigue. It is crucial to monitor patients closely for any adverse reactions during and after the treatment.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Sanfilippo Syndrome (MPS IIIA) that target the genetic mutation causing the disease, such as gene therapy. ABO-102 is an investigational gene therapy designed to address the underlying genetic cause of MPS IIIA, and its long-term safety and tolerability are currently being evaluated in clinical trials.

What other types of research exist?

Promising novel alternatives to ABO-102 for Sanfilippo Syndrome (MPS IIIA) patients include: 1) Enzyme replacement therapies (ERT) that aim to supplement the deficient enzyme, such as recombinant human heparan-N-sulfatase. 2) Other gene therapies using different vectors or delivery methods to correct the genetic mutation. 3) Substrate reduction therapies (SRT) that reduce the accumulation of glycosaminoglycans (GAGs) by inhibiting their synthesis. 4) Small molecule therapies that enhance the residual activity of the deficient enzyme or stabilize the enzyme to improve its function.

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Enzyme Replacement Therapy

Long-term Follow-up After ABO-102 Treatment for Sanfilippo Syndrome

Phase 3

Waitlist Available

Research Sponsored by Ultragenyx Pharmaceutical Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to year 5

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial aims to evaluate the safety and tolerability of ABO-102 in patients with MPS IIIA who have already participated in a previous trial. ABO-102 is a gene therapy that delivers a healthy gene to improve their condition. ABO-102 is a gene therapy that has been developed to treat MPS IIIA by delivering a healthy gene to correct the enzyme deficiency.

Who is the study for?

This trial is for patients with MPS IIIA who have already received ABO-102 in a previous study. Their parents or guardians must consent and be able to follow the study's procedures and schedule. Those currently in another trial or with conditions that interfere with this study can't participate.

What is being tested?

The focus of this trial is on the long-term safety and effects of ABO-102, a treatment previously given to participants. It also looks at whether additional immune system suppression therapy is needed alongside it.

What are the potential side effects?

While specific side effects are not listed, they may include typical reactions from gene therapies such as immune responses, swelling at injection sites, fever, headaches, nausea, or potential long-term risks which will be monitored.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to year 5

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to year 5

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to year 5 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Adverse Events

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2Experimental Treatment1 Intervention

Participants who have completed a prior clinical trial involving the administration of ABO-102 and who cannot participate in Cohort 1. Participants will partake in a reduced number of assessments, performed either onsite or at home via a combination of telehealth and home healthcare visits.

Group II: Cohort 1Experimental Treatment2 Interventions

Participants who have completed a prior clinical trial involving the administration of ABO-102 and are able to comply with onsite scheduled visits and assessments. Select participants may receive adjuvant IS therapy.

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Gene therapy, such as ABO-102, targets the genetic mutation causing Sanfilippo Syndrome (MPS IIIA) by delivering a functional copy of the defective gene to the patient's cells. This is typically achieved using a viral vector to introduce the correct gene into the patient's DNA, enabling the production of the missing or deficient enzyme. For Sanfilippo Syndrome patients, this approach is crucial as it addresses the root cause of the disease, potentially halting or reversing the progression of symptoms by restoring normal metabolic function.

Limitations of enzyme replacement therapy: current and future.