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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

Study Summary

This trial will investigate a possible treatment for a genetic disorder called MPS IIIA, looking at its long-term safety and tolerability.

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.Check my eligibility

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.See study design

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Adverse Events

Secondary outcome measures

Change From Baseline (in Prior Trial) in Cerebrospinal Fluid Heparan Sulfate (CSF HS)

Change From Baseline (in Prior Trial) in the Cognitive Subtest of the Bayley Scales of Infant and Toddler Development -Third Edition (BSID-III) or the Kaufman Assessment Battery for Children - Second Edition (KABC-II), Based on Developmental Age

Change From Baseline (in Prior Trial) in the Language Subtest of the Bayley Scales of Infant and Toddler Development -Third Edition (BSID-III) or the Kaufman Assessment Battery for Children - Second Edition (KABC-II), Based on Developmental Age

+1 more

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.

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Ultragenyx Pharmaceutical IncLead Sponsor

89 Previous Clinical Trials

179,042 Total Patients Enrolled

2 Trials studying Sanfilippo Syndrome

33 Patients Enrolled for Sanfilippo Syndrome

Abeona Therapeutics, IncIndustry Sponsor

11 Previous Clinical Trials

1,068 Total Patients Enrolled

2 Trials studying Sanfilippo Syndrome

33 Patients Enrolled for Sanfilippo Syndrome

Medical DirectorStudy DirectorUltragenyx Pharmaceutical Inc

2,777 Previous Clinical Trials

8,063,831 Total Patients Enrolled

2 Trials studying Sanfilippo Syndrome

33 Patients Enrolled for Sanfilippo Syndrome

Media Library

ABO-102 (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04360265 — Phase 3

Sanfilippo Syndrome Research Study Groups: Cohort 1, Cohort 2

Sanfilippo Syndrome Clinical Trial 2023: ABO-102 Highlights & Side Effects. Trial Name: NCT04360265 — Phase 3

ABO-102 (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04360265 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any current vacancies for participants in this research trial?

"The trial in question has not been recruiting since May 1st, 2023. As per the data hosted on clinicaltrials.gov, this is no longer an active study at present; however, there are 30 other medical trials actively seeking participants right now."

Answered by AI

Does this procedure have the endorsement of governmental health authorities?

"Our team has evaluated this therapeutic intervention to be of a moderate safety level, receiving a rating of 3. This is due to the pre-existing proof-of-concept data and multiple trials highlighting its security profile."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102

2020. "A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04360265.