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Virus Therapy

ABO-102 for Mucopolysaccharidosis

Phase 1 & 2
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
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, 12 months
Awards & highlights

Study Summary

This trial is testing a new way to deliver a gene therapy to treat a disease called Mucopolysaccharidosis type II, or MPS II.

Eligible Conditions
  • Mucopolysaccharidosis
  • Sanfilippo Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, 12 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, 12 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change From BL in Cerebrospinal Fluid (CSF) Heparan Sulfate Levels After Treatment
Change From Baseline (BL) in Multiples of Normal of Liver and Spleen Volumes After Treatment
Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame
+1 more
Secondary outcome measures
Change From Baseline in Brain Volumes After Treatment
Change From Baseline in Brain Volumes After Treatment: Average Total Cortical Thickness
Change From Baseline in CSF N-Sulfoglucosamine Sulfohydrolase (SGSH) Enzyme Activity Levels After Treatment
+15 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: ABO-102Experimental Treatment1 Intervention
Dose of 3x10^13 vg/kg

Find a Location

Who is running the clinical trial?

Abeona Therapeutics, IncIndustry Sponsor
11 Previous Clinical Trials
1,096 Total Patients Enrolled
Ultragenyx Pharmaceutical IncLead Sponsor
90 Previous Clinical Trials
179,111 Total Patients Enrolled
Medical DirectorStudy DirectorUltragenyx Pharmaceutical Inc
2,785 Previous Clinical Trials
8,065,693 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
Molecular Therapy - Methods & Clinical DevelopmentJournal
Functional Correction of Neurological and Somatic Disorders at Later Stages of Disease in MPS IIIA Mice by Systemic Scaav9-hsgsh Gene Delivery
Fu, Haiyan, Marcela P Cataldi, Tierra A Ware, Kimberly Zaraspe, Aaron S Meadows, Darren A Murrey, and Douglas M McCarty. 2016. “Functional Correction of Neurological and Somatic Disorders at Later Stages of Disease in MPS IIIA Mice by Systemic Scaav9-hsgsh Gene Delivery”. Molecular Therapy - Methods & Clinical Development. Elsevier BV. doi:10.1038/mtm.2016.36.
Human Gene Therapy Clinical DevelopmentJournal
Differential Prevalence of Antibodies Against Adeno-associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for Aav-mediated Gene Therapy
Fu, Haiyan, Aaron S. Meadows, Ricardo J. Pineda, Krista L. Kunkler, Kristen V. Truxal, Kim L. McBride, Kevin M. Flanigan, and Douglas M. McCarty. 2017. “Differential Prevalence of Antibodies Against Adeno-associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for Aav-mediated Gene Therapy”. Human Gene Therapy Clinical Development. Mary Ann Liebert Inc. doi:10.1089/humc.2017.109.
clinicaltrials.govStudy
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
2019. "Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04088734.
~1 spots leftby May 2025
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