MPS IIIANCT07855562
MPS IIIA Disease Monitoring Program
An observational study of MPS IIIA and Sanfilippo Syndrome, run by Ultragenyx Pharmaceutical Inc.
Trial summary
The main objective of this observational study is to evaluate the long-term safety and effectiveness of rebisufligene etisparvovec for 10 years after rebisufligene etisparvovec administration. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
- How many people
- 55 participants
- Where
- No sites listed yet
- Study dates
- Nov 2026to Oct 2038
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The DMP population comprises patients with MPS IIIA who received rebisufligene etisparvovec in a clinical trial or have been prescribed (or plan to receive) rebisufligene etisparvovec in a post marketing setting.
Who may be able to join
- Confirmed diagnosis of MPS IIIA
- Patient who:
- Group 1: Received rebisufligene etisparvovec in a prior interventional clinical trial.
- May be enrolled upon completion of Study ABT-001 (Cohort 4) or end of study (EOS) or early termination (ET) visit from the prior clinical trial (Study ABT-003 or Study LTFU-ABO-102). OR
- Group 2: Received (or plans to receive) rebisufligene etisparvovec in a postmarketing setting.
- Provide informed consent after the nature of the study has been explained, and prior to any research-related procedures. If a minor or an adult with cognitive limitations, willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related procedures.
Who cannot join
- Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Mucopolysaccharidosis Type IIIA (MPS IIIA) Disease Monitoring Program
The main objective of this observational study is to evaluate the long-term safety and effectiveness of rebisufligene etisparvovec for 10 years after rebisufligene etisparvovec administration.
Read the detailed description
The UX111-CL401 MPS IIIA Disease Monitoring Program is a prospective, multicenter, long term outcomes study to obtain safety and clinical outcomes data for 10 years after the administration of rebisufligene etisparvovec in either a clinical trial or post-marketing setting.
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Being tested
- No Intervention
- Keywords
- MPS IIIA, Sanfilippo, Gene Therapy
- Medical subject headings
- Mucopolysaccharidosis III
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
No investigational/study product will be administered in this DMP.
| Study group | Role | What they receive |
|---|---|---|
| Group 1: Rebisufligene Etisparvovec in Prior Clinical Study | Participants administered rebisufligene etisparvovec (Fayuvi, UX111) in prior clinical study involving rebisufligene etisparvovec Receives: Other: No Intervention | |
| Group 2: Rebisufligene Etisparvovec in Post-Marketing Setting | Participants administered rebisufligene etisparvovec (Fayuvi, UX111) in a post-marketing setting Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Serious Adverse Events (SAEs) Assessed as Related to Rebisufligene Etisparvovec by the Investigator and Targeted Adverse Events (Targeted AEs) Associated with Identified and Potential Risks of UX111 or AAV8 Gene Therapy
Measured: 10 Years
Bayley Scales of Infant and Toddler Development Cognitive Raw Score Over Time
Measured: 10 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2026
- Study startNov 2026 (estimated)
- Primary completionOct 2038 (estimated)
- Study completionOct 2038 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MPS IIIAStudies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 55 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceuticals Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07855562
- Sponsor's protocol ID
- UX111-CL401
- Status last verified
- Sep 2026
- First submitted
- 25 Sep 2026
- Last updated
- 2 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mucopolysaccharidosis Type IIIA (MPS IIIA) Disease Monitoring Program
- ClinicalTrials.gov ID
- NCT07855562
- Acronym / sponsor ID
- UX111-CL401
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Keywords
- MPS IIIA, Sanfilippo, Gene Therapy
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 55 participants (estimated)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- Nov 2026 (estimated)
- Primary completion
- Oct 2038 (estimated)
- Study completion
- Oct 2038 (estimated)
- First posted
- 2 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main objective of this observational study is to evaluate the long-term safety and effectiveness of rebisufligene etisparvovec for 10 years after rebisufligene etisparvovec administration.
Detailed description
The UX111-CL401 MPS IIIA Disease Monitoring Program is a prospective, multicenter, long term outcomes study to obtain safety and clinical outcomes data for 10 years after the administration of rebisufligene etisparvovec in either a clinical trial or post-marketing setting.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1: Rebisufligene Etisparvovec in Prior Clinical Study | – | Other: No InterventionParticipants administered rebisufligene etisparvovec (Fayuvi, UX111) in prior clinical study involving rebisufligene etisparvovec |
| Group 2: Rebisufligene Etisparvovec in Post-Marketing Setting | – | Other: No InterventionParticipants administered rebisufligene etisparvovec (Fayuvi, UX111) in a post-marketing setting |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The DMP population comprises patients with MPS IIIA who received rebisufligene etisparvovec in a clinical trial or have been prescribed (or plan to receive) rebisufligene etisparvovec in a post marketing setting.
Key inclusion criteria (6 listed)
- Confirmed diagnosis of MPS IIIA
- Patient who:
- May be enrolled upon completion of Study ABT-001 (Cohort 4) or end of study (EOS) or early termination (ET) visit from the prior clinical trial (Study ABT-003 or Study LTFU-ABO-102). OR
- Provide informed consent after the nature of the study has been explained, and prior to any research-related procedures. If a minor or an adult with cognitive limitations, willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related procedures.
Key exclusion criteria (1 listed)
- Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No InterventionNo investigational/study product will be administered in this DMP. | Other | Group 1: Rebisufligene Etisparvovec in Prior Clinical Study, Group 2: Rebisufligene Etisparvovec in Post-Marketing Setting |
Primary outcome
Number of Participants with Serious Adverse Events (SAEs) Assessed as Related to Rebisufligene Etisparvovec by the Investigator and Targeted Adverse Events (Targeted AEs) Associated with Identified and Potential Risks of UX111 or AAV8 Gene Therapy
Time frame: 10 Years
Bayley Scales of Infant and Toddler Development Cognitive Raw Score Over Time
Time frame: 10 Years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2026.
- Our Clinical Trial Transparency Commitment-UltragenyxLink provided by the sponsor.
- Ultragenyx Patient Advocacy WebsiteLink provided by the sponsor.
Related trials for MPS IIIA
RecruitingPhase 1/2
Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
Mucopolysaccharidosis Type IIIA
RecruitingPhase 2/3
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
MPS IIIA, Sanfilippo Syndrome
Not yet recruitingPhase 1/2
Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB
Sanfilippo Syndrome (MPS III)
Active, not recruitingPhase 1
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Sanfilippo Syndrome Type A
Active, not recruitingPhase 1/2
Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
Mucopolysaccharidosis III-A
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director study director
Ultragenyx Pharmaceuticals Inc.
Questions about this trial
What is this clinical trial studying?
An observational study of MPS IIIA and Sanfilippo Syndrome, run by Ultragenyx Pharmaceutical Inc.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 2 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Patients Contact: Trial Recruitment (1-888-756-8657, trialrecruitment@ultragenyx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Serious Adverse Events (SAEs) Assessed as Related to Rebisufligene Etisparvovec by the Investigator and Targeted Adverse Events (Targeted AEs) Associated with Identified and Potential Risks of UX111 or AAV8 Gene Therapy (time frame: 10 Years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2026.