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

Recruiting at 3 trial locations

Overseen By Medical Affairs

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Ultragenyx Pharmaceutical Inc

Disqualifiers: Other trials, Medical conditions, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

41 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It is best to discuss this with the trial coordinators or your doctor.

Research Team

Medical Director

Principal Investigator

Ultragenyx Pharmaceutical Inc

Eligibility Criteria

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.

Inclusion Criteria

You have participated in a previous study where you were given ABO-102.

Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule.

Exclusion Criteria

Planned or current participation in another clinical trial that may confound the safety and efficacy evaluation of ABO-102 during this study

Any other situation or medical condition that precludes the participant from undergoing procedures required in this study.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Long-term Follow-up

Participants are monitored for safety/tolerability and efficacy of UX111 after previous gene therapy trials

Long-term

Onsite scheduled visits and assessments; telehealth and home healthcare visits for some participants

Adjuvant Immunomodulatory Therapy

Selected participants may receive adjuvant immunomodulatory therapy based on consultation with the medical monitor

Treatment Details

Interventions

• ABO-102

Trial Overview 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.

Participant Groups

Group I: Cohort B Experimental Treatment 1 Intervention

Participants who have participated in a prior clinical trial involving the administration of UX111 (rebisufligene etisparvovec) and who cannot participate in Cohort A. 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 A Experimental Treatment 2 Interventions

Participants who have participated in a prior clinical trial involving the administration of UX111 (rebisufligene etisparvovec) and are able to comply with onsite scheduled visits and assessments. Select participants may receive adjuvant IM therapy.

Who Is Running the Clinical Trial?

Ultragenyx Pharmaceutical Inc

Lead Sponsor

Trials

94

Recruited

104,000+

Dr. Emil D. Kakkis

Ultragenyx Pharmaceutical Inc

Chief Executive Officer since 2010

MD/PhD in Biological Chemistry from UCLA

Dr. Eric Crombez

Ultragenyx Pharmaceutical Inc

Chief Medical Officer since 2023

MD from Wayne State University School of Medicine

Abeona Therapeutics, Inc

Industry Sponsor

Trials

12

Recruited

1,100+