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Virus Therapy
Gene Therapy for Sanfilippo Syndrome
Phase 2 & 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
Age: From birth to 2 years or children older than 2 years with a minimum cognitive Developmental Quotient (DQ) of 60 or above (calculated by Bayley Scales of lnfant and Toddler Development - Third Edition)
Diagnosis of MPS IIIA confirmed by the following methods: No detectable or significantly reduced SGSH enzyme activity by leukocyte assay, and Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to month 24
Awards & highlights
Study Summary
This trial is testing a new gene therapy for a disease called Mucopolysaccharidosis type II (MPS II). The therapy involves injecting a virus carrying a healthy gene into a vein.
Who is the study for?
This trial is for children diagnosed with MPS IIIA, a genetic disorder. Eligible participants are from birth to 2 years old or older than 2 with a cognitive score of at least 60. They must have confirmed mutations in the SGSH gene and reduced enzyme activity. Children can't join if they have uncontrolled seizures, heart issues, previous gene therapy, or conditions that interfere with testing.Check my eligibility
What is being tested?
The study tests ABO-102's effectiveness and safety in treating MPS IIIA alongside adjuvant immunosuppression therapy. It aims to see how well this gene transfer approach works by delivering a functional copy of the SGSH gene into patients.See study design
What are the potential side effects?
Potential side effects may include immune reactions due to the introduction of new genes (gene transfer), complications related to immunosuppression such as increased infection risk, and typical risks associated with lumbar punctures used for treatment administration.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am under 2 years old or older with a cognitive score above 60.
Select...
I have been diagnosed with MPS IIIA through specific genetic and enzyme tests.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to month 24
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to month 24
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change from Baseline in Cognitive Domain Bayley Scales of Infant and Toddler Development Raw Scores-Third edition (BSID-III)
Secondary outcome measures
Change From Baseline in BSID-III: Language Domain
Change From Baseline in BSID-III: Motor Domain
Change From Baseline in Brain Volumes After Treatment
+5 moreOther outcome measures
Number of Participants with Adverse Events, Treatment-emergent Adverse Events, and Serious Adverse Events
Trial Design
3Treatment groups
Experimental Treatment
Group I: Cohort 3 High DoseExperimental Treatment2 Interventions
Dose of 3 X 10^13 vg/kg
Group II: Cohort 2 Mid DoseExperimental Treatment2 Interventions
Dose of 1 X 10^13 vg/kg
Group III: Cohort 1 Low DoseExperimental Treatment2 Interventions
Dose of 0.5 X 10^13 vg/kg
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Who is running the clinical trial?
Ultragenyx Pharmaceutical IncLead Sponsor
89 Previous Clinical Trials
179,046 Total Patients Enrolled
Abeona Therapeutics, IncIndustry Sponsor
11 Previous Clinical Trials
1,073 Total Patients Enrolled
Medical DirectorStudy DirectorUltragenyx Pharmaceutical Inc
2,777 Previous Clinical Trials
8,063,458 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I am not pregnant nor have I tested positive for pregnancy.You have a high level of antibodies against AAV9 in your blood.I have a condition that makes spinal taps unsafe for me.Your vision or hearing problems make it hard for you to do the neurodevelopmental testing.I have seizures that are not controlled by medication.I am under 2 years old or older with a cognitive score above 60.I have been diagnosed with MPS IIIA through specific genetic and enzyme tests.The study leader has decided I can't participate in the trial.My genetic test shows I have the S298P mutation in the SGSH gene.I don't use any items that prevent me from having an MRI.You have a brain condition or behavior that could make it hard to understand the study results.I have a health condition that prevents me from having a lumbar puncture or using anesthetics.You have a current viral infection based on doctor's examination.My genetic test shows two specific changes in the SGSH gene.Your blood tests show signs of being exposed to HIV, or having active hepatitis B or C infection.I haven't had a live vaccine in the last 30 days.My condition is a milder form of MPS IIIA.I have a heart condition like cardiomyopathy or a significant congenital heart abnormality.You have shown a positive response to a specific test for T-cell reactions.You have previously taken part in a clinical trial for gene/cell therapy or enzyme replacement therapy.I have had a stem cell transplant before.My blood tests show abnormal levels in key areas.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 1 Low Dose
- Group 2: Cohort 2 Mid Dose
- Group 3: Cohort 3 High Dose
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What results do researchers hope to observe as a result of this trial?
"This clinical trial will span 24 months, during which time the primary goal is to assess product safety. Secondary objectives involve a comparison between baseline cognitive development and natural history study using either Bayley Scales or Kaufman Assessment Battery; vector shedding analysis in bodily fluids for environmental risk assessment purposes; and changes from baseline on Sanfilippo Behavior Rating Scale at 6, 12, 18 and 24 month intervals."
Answered by AI
Is this study currently recruiting participants?
"According to clinicaltrials.gov, this particular trial is not enrolling patients at the moment; it was initially posted on March 1st 2016 and last revised on November 8th 2022. Nevertheless, there are 28 other trials that have open enrollment now."
Answered by AI
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