ClinicalTrials.gov record NCT07579910

Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

A Phase 3, Randomized, Single-blind, Parallel Group, Controlled, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebroventricular Tralesinidase Alfa Treatment Compared to Standard of Care in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age1 year old to 5 years old
SexAll
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 3 study enrolls young children with MPS IIIB to compare intracerebroventricular (ICV) tralesinidase alfa with a control group receiving standard of care. It is randomized, single-blind, and parallel-group. The main outcome is change in cognitive raw score (BSID-III Cognition) from baseline to Week 260 (about 5 years).

What it is studying. The study tests intracerebroventricular tralesinidase alfa compared with standard of care, measuring effects on cognitive development over approximately five years.

What the researchers hope to learn. To evaluate the safety, tolerability, and efficacy of intracerebroventricular tralesinidase alfa in people with MPS IIIB, with the primary scientific goal of measuring change in cognitive development (BSID-III Cognition) over 260 weeks.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

The primary objectives of this study are to evaluate the effects of Tralesinidase Alfa (TA) on cognition

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Tralesinidase alfa (TA)

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Has a diagnosis of MPS IIIB confirmed by deficient NAGLU enzyme activity during screening.
  • Has nonattenuated severe MPS IIIB by the Genotype and Clinical Assessment Committee.
  • Is ≥1 and ≤5 years of age with BSID-III-C raw score of <70. Is male or female as identified at birth.

Exclusion criteria

  • Genotyped for a known MPS IIIB variant associated with an attenuated phenotype or has a sibling with a known attenuated phenotype.
  • Has another neurological illness that may have caused cognitive decline (e.g., trauma, meningitis, or hemorrhage) before study entry.
  • Has received stem cell, gene therapy, or enzyme replacement therapy for MPS IIIB.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants first have screening to confirm deficient NAGLU enzyme activity. Those who qualify are randomized to receive intracerebroventricular (ICV) tralesinidase alfa or to a control (no intervention) arm. Participants will have repeated cognitive testing with the Bayley Scales of Infant and Toddler Development, Third Edition (BSID‑III‑C), administered by blinded raters and reviewed centrally, with the main comparison through Week 260 (about 5 years). The registry record names the ICV infusion and the BSID‑III‑C assessments but does not provide further details about other visits, tests, or sample collection.

How the study is structured. Phase 3, randomized, single-blind, parallel-group trial. Participants are assigned to either an ICV tralesinidase alfa arm (the study drug is given as a sterile solution for ICV infusion) or a control arm with no intervention. Cognitive assessments (BSID-III-C) are done by trained, blinded raters and reviewed centrally; the primary comparison is change from baseline to Week 260.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry lists this study under MPS IIIB. Does it apply to my diagnosis?
  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • No sites are listed on the registry yet. Where will the study take place?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Sanfilippo syndrome

Clinical trials12 trials listed for Sanfilippo syndromeEvery trial the registry lists for Sanfilippo syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Sanfilippo syndrome researchLatest monthly update: September 2026. Includes: Approved drug shortlist. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
December 2026 (estimated)
Primary completion
August 2033 (estimated)
Study completion
August 2033 (estimated)
Target enrollment
14 participants (estimated)
Last updated on registry
Sep 21, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Spruce Biosciences

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.

Terms used on this page · explanations by MedicalUpdates.ai

during screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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